Ignite Creation Date:
2025-12-24 @ 11:37 PM
Ignite Modification Date:
2025-12-25 @ 9:27 PM
Study NCT ID:
NCT03763656
Status:
COMPLETED
Last Update Posted:
2024-10-28
First Post:
2018-12-03
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Pharmacokinetics of Oral Hydroxyurea Solution
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D006450', 'term': 'Hemoglobin SC Disease'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D006918', 'term': 'Hydroxyurea'}, {'id': 'D012996', 'term': 'Solutions'}], 'ancestors': [{'id': 'D014508', 'term': 'Urea'}, {'id': 'D000577', 'term': 'Amides'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D004364', 'term': 'Pharmaceutical Preparations'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'hussain.mulla@novalabs.co.uk', 'phone': '0116 223 0101', 'title': 'Dr Hussain Mulla, Head of Clinical Development', 'organization': 'Nova Laboratories Ltd'}, 'certainAgreement': {'otherDetails': 'Any publication or sub-set analysis of data based on the results obtained shall not be made before the first publication by the Sponsor, in which the Investigators may be named as authors. After initial publication material for public dissemination will be submitted to the Sponsor for review at least 60 days prior to submission, where all reasonable comments by the Sponsor will be incorporated. Sponsor may request additional delay for up to 6 months to protect proprietary information/IP.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Screening to safety follow up call at 64 weeks (maximum 15 months on treatment).', 'description': 'Protocol definition of SAE for SCA associated complications, AEs only became reportable SAEs if the hospital admission was prolonged \\>7days. SCA related hospitalisations \\<7days recorded as AEs due to the frequency of admissions within population and chronic nature of the condition. Attendance of emergency room without admission or for elective procedures were not considered SAEs unless prolonged. All SAEs unrelated to SCA condition were reportable in line with standard definitions.', 'eventGroups': [{'id': 'EG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution', 'otherNumAtRisk': 32, 'deathsNumAtRisk': 32, 'otherNumAffected': 31, 'seriousNumAtRisk': 32, 'deathsNumAffected': 0, 'seriousNumAffected': 6}], 'otherEvents': [{'term': 'Anemia', 'notes': '\\*Related Treatment Emergent Adverse Event (TEAE) in Safety population (n=32). Where relatedness is defined as anything at least possibly related to the IMP.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Neutropenia/Low ANC Count', 'notes': 'Haematological toxicity (ANC \\<1.0x10\\^9/L). n=3 Grade 3 events in n=3 participants; n=4 events in n=4 participants at least possibly related. In n=3 in the 6month-1.99 year age group, associated with viral/other illness or dose titration.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Thrombocytopenia/Low Platelet Count', 'notes': 'Haematological toxicity (Platelet \\<80x10\\^9/L) were \\</= Grade 2. Of which n=6 events were considered possibly related (in 3 participants), with n=5 events in the 6 month-1.99 year age group, associated with viral/other illness or dose titration.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Haemoglobin (Hb) Decreased', 'notes': 'Haematological toxicity (Hb \\<4.5g/dL or 20% drop). n=1 event Grade 3 in n=1 participant; n=2 events at least possibly related in n=1 participant in the 6month-1.99 year age group.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Absolute Reticulocyte Count (ARC) Decreased', 'notes': 'Haematological toxicity (ARC \\<80x10\\^9/L; unless Hb \\>9.0g/dL). All events \\</= Grade 2; n=2 events at least possibly related in n=1 participant in the 6month-1.99 year age group.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Red Blood Cell (RBC) Count Decreased', 'notes': '\\*Related Treatment Emergent Adverse Event (TEAE) in Safety population (n=32). Where relatedness is defined as anything at least possibly related to the IMP.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Gamma-Glutamyltransferase (GGT) Increased', 'notes': '\\*Related Treatment Emergent Adverse Event (TEAE) in Safety population (n=32). Where relatedness is defined as anything at least possibly related to the IMP.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Hematocrit (HCT) Decreased', 'notes': '\\*Related Treatment Emergent Adverse Event (TEAE) in Safety population (n=32). Where relatedness is defined as anything at least possibly related to the IMP.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Diarrhoea', 'notes': 'Diarrhoea; n=1 event at least possibly related in n=1 participant; n=4 unrelated event of mild/moderate severity in n=3 participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Alopecia', 'notes': '\\*Related Treatment Emergent Adverse Event (TEAE) in Safety population (n=32). Where relatedness is defined as anything at least possibly related to the IMP.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Nail Discolouration', 'notes': '\\*Related Treatment Emergent Adverse Event (TEAE) in Safety population (n=32). Where relatedness is defined as anything at least possibly related to the IMP.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Rash', 'notes': 'Rash; n=1 at least possibly related event in n= 1 participant; n=2 unrelated events in n=2 participants of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Rash Papular', 'notes': '\\*Related Treatment Emergent Adverse Event (TEAE) in Safety population (n=32). Where relatedness is defined as anything at least possibly related to the IMP.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Sickle Cell Anemia with Crisis/Vaso-Occlusive Crisis (VOC)', 'notes': "'SCA with crisis' interchangeable with 'Vaso-Occlusive crisis (VOC).' n=7 events were Grade 3 or above in n=4 participants and did not meet the protocol definition of SAEs; Remainder n=46 in n=14 participants were mild/moderate severity.", 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 53, 'numAffected': 18}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Viral Upper Respiratory Tract Infection', 'notes': 'Unrelated viral URTI; n=1 Grade 3 event in 1 participant (6months -2.99years); n=2 events in n=2 participants of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Pain in extremity', 'notes': 'Unrelated pain in extremity; n=1 event Grade 3 in n=1 participant; n=4 events Grade \\</=2 in 4 participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Acute Chest Syndrome (ACS)', 'notes': 'ACS events of mild/moderate severity unrelated to IMP that did not meet the protocol definition of SAE.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Hypereosinophilic Syndrome', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Splenomegaly', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Ear Pain', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Abdominal Discomfort', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Abdominal Pain', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Constipation', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Gastritis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Gastrointestinal Haemorrhage', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Mouth Ulceration', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Salivary Gland Enlargement', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Teething', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Vomiting', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Feeling Abnormal', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Influenza Like Illness', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Injection Site Pain', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Injection Site Swelling', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Peripheral Swelling', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Pyrexia', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 16, 'numAffected': 9}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Swelling Face', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Ulcer', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Cholecystitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Hepatomegaly', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Drug Hypersensitivity', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Body Tinea', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Chronic Sinusitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Conjunctivitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Conjunctivitis Viral', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'COVID-19', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Croup Infectious', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Ear Infection', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Fungal Skin Infection', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Gastroenteritis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Gingival Abscess', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Gingivitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Hand-Foot-And-Mouth Disease', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Localised Infection', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Lower Respiratory Tract Infection', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Nasopharyngitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Otitis Media', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Pharyngitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Pharyngotonsillitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Rhinitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Sinusitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Tinea Capitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Tonsilitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Upper Respiratory Tract Infection', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 43, 'numAffected': 17}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Urinary Tract Infection', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Viral Infection', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Viral Pharyngitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Viral Rash', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Arthropod Bite', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Contusion', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Forearm Fracture', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Lip Injury', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Procedural Pain', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Skin Abrasion', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Skin Laceration', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Soft Tissue Injury', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Wound Complication', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Alanine Aminotransferase Increased', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Aspartate Aminotransferase Increased', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Blood Iron Decreased', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Body Temperature Increased', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Platelet Count Increased', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Vitamin D Decreased', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Decreased Appetite', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Vitamin D Deficiency', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Arthralgia', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Back Pain', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Bone Pain', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Muscle Tightness', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Soft Tissue Swelling', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Trismus', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Benign Rolandic Epilepsy', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Headache', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 8, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Lethargy', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Pica', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Balanoposthitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Priapism', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Allergic Respiratory Disease', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Allergic Sinusitis', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Asthma', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 7, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Bronchial Hyperreactivity', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Bronchospasm', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Cough', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Rhinitis Allergic', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Rhinorrhoea', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Sneezing', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Wheezing', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 4, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Eczema', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Pruritus', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Skin Erosion', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Urticaria Papular', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Tooth Extraction', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Toothache', 'notes': 'Unrelated Treatment Emergent (TEAEs) of mild/moderate severity.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}], 'seriousEvents': [{'term': 'Acute Chest Syndrome (ACS) Grade 3', 'notes': 'Grade 3, requiring hospitalisation \\>7days (n=3 participants)', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Acute Chest Syndrome (ACS) Grade 2', 'notes': 'Grade 2, requiring hospitalisation \\>7days', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Vaso-occlusive Crisis (VOC)', 'notes': 'Grade 3, requiring hospitalisation \\>7days (n=2 participants)', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}, {'term': 'Anemia secondary to SCD', 'notes': 'Grade 3, requiring hospitalisation for oxygen therapy and blood transfusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 32, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.1)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Clearance (CL/F)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'categories': [{'measurements': [{'value': '3.61', 'groupId': 'OG000', 'lowerLimit': '3.29', 'upperLimit': '3.92'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).', 'unitOfMeasure': 'L/hr', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Clearance value for patient of 15.05 kg'}, {'type': 'PRIMARY', 'title': 'Volume of Distribution (V/F)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'categories': [{'measurements': [{'value': '11.4', 'groupId': 'OG000', 'lowerLimit': '9.52', 'upperLimit': '13.2'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).', 'unitOfMeasure': 'L', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Volume of distribution for central and peripheral compartments for patient of 15.05 kg'}, {'type': 'PRIMARY', 'title': 'Time to Maximum Concentration (Tmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Tmax (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.253', 'spread': '0.412', 'groupId': 'OG000'}]}]}, {'title': 'Tmax (Age Group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.265', 'spread': '0.322', 'groupId': 'OG000'}]}]}, {'title': 'Tmax (Age Group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.238', 'spread': '0.455', 'groupId': 'OG000'}]}]}, {'title': 'Tmax (Age Group 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.269', 'spread': '0.423', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean Tmax (h) pharmacokinetic parameter derived using the final population PK model.', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK population = 32 participants, who have received at least one dose at 15 mg/kg and have at least one sample of PK plasma above the lower limit of quantification'}, {'type': 'PRIMARY', 'title': 'Maximum Plasma Concentration Cmax (ug/mL)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Cmax (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.124', 'spread': '1.742', 'groupId': 'OG000'}]}]}, {'title': 'Cmax (Age Group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12.807', 'spread': '1.399', 'groupId': 'OG000'}]}]}, {'title': 'Cmax (Age Group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.068', 'spread': '1.939', 'groupId': 'OG000'}]}]}, {'title': 'Cmax (Age Group 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.404', 'spread': '1.714', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean Cmax (ug/mL) pharmacokinetic parameter derived using the final population PK model.', 'unitOfMeasure': 'ug/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK population: n=32 safety population, having received at least one dose of study drug at 15mg/kg and one sample of PK plasma above the lower limit of quantification.'}, {'type': 'PRIMARY', 'title': 'Area Under Plasma Concentration Time Curve (AUC 0-Inf)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'AUC Infinity (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '64.721', 'spread': '9.339', 'groupId': 'OG000'}]}]}, {'title': 'AUC Infinity (Age Group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '62.537', 'spread': '9.016', 'groupId': 'OG000'}]}]}, {'title': 'AUC Infinity (Age Group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '62.899', 'spread': '7.373', 'groupId': 'OG000'}]}]}, {'title': 'AUC Infinity (Age Group 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '68.948', 'spread': '11.649', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean AUC 0-Infinity (hr\\*ug/mL) pharmacokinetic parameters derived using the final population PK model.', 'unitOfMeasure': 'hr*ug/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK population = 32 participants receiving at least one dose of 15mg/kg hydroxyurea and have at least one PK plasma sample above lower limit of quantification.'}, {'type': 'PRIMARY', 'title': 'Terminal Half-life (Hours)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Terminal Half-life (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.880', 'spread': '0.3004', 'groupId': 'OG000'}]}]}, {'title': 'Terminal Half-life (Age Group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.107', 'spread': '0.187', 'groupId': 'OG000'}]}]}, {'title': 'Terminal Half-life (Age Group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.949', 'spread': '0.257', 'groupId': 'OG000'}]}]}, {'title': 'Terminal Half-life (Age 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.634', 'spread': '0.268', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean Terminal Half-life (hours) pharmacokinetic parameter derived using the final population PK model.', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Population n=32 where all subjects received at least one dose of 15 mg/kg hydroxyurea and have at least one sample of PK plasma above the lower limit of quantification.'}, {'type': 'SECONDARY', 'title': 'Adverse Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Any Adverse Events (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '339', 'groupId': 'OG000'}]}]}, {'title': 'Treatment Emergent Adverse Events (TEAEs) Overall', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '339', 'groupId': 'OG000'}]}]}, {'title': 'Serious AEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Grade >or=3 TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Related TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring unrelated TEAE: SCA with Crisis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '53', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring unrelated TEAE: Upper Respiratory Tract Infection', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '43', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring unrelated TEAE: Pyrexia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring unrelated TEAE: Cough', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring unrelated TEAE: Vomiting', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring unrelated TEAE: Abdominal Pain', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring possibly related TEAE: Neutropenia/ANC Count Decreased', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Commonly occurring possibly related TEAE: Thrombocytopenia/Platelet Count Decreased', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Screening Up to Week 64', 'description': "Incidence of Adverse events during the course of the trial from screening to final follow up phone call at Week 64.\n\nRelatedness refers to 'at least possibly related to the IMP', with severity/toxicity assessed using the CTCAE Toxicity Grade and seriousness based on the standard definition for SAE in accordance with GCP. With the exception of SCA related events, requiring \\>7day hospitalisation, or extension of hospitalisation before being reported as an SAE due to their frequency within the disease population. All other non-SCA related SAEs were reportable.", 'unitOfMeasure': 'events', 'reportingStatus': 'POSTED', 'populationDescription': "Safety population n=32. Overall 339 AEs in 31 participants recorded. 7 adverse events included in this list of 339 were defined as 'Serious' as per the protocol definition (i.e. SCA related and \\>7days hospitalisation). A separate SAE report was made at that time with a clearly defined start and end date to be able to denote a resolution to the 'seriousness' criteria as this is an ongoing chronic condition."}, {'type': 'SECONDARY', 'title': 'Absolute Neutrophil Count (ANC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.90', 'spread': '1.825', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.30', 'spread': '1.608', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.38', 'spread': '2.087', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.43', 'spread': '1.354', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.70', 'spread': '1.033', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.27', 'spread': '0.671', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.46', 'spread': '0.837', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.48', 'spread': '1.255', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 60 (or final visit); max 15 months on treatment', 'description': 'Safety review for haematological toxicity (mild myelosuppression target: 1-3x10\\^9/L)', 'unitOfMeasure': 'x10^9 neutrophils/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'White Blood Cell Count (Leukocytes)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.01', 'spread': '3.514', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12.24', 'spread': '2.019', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14.59', 'spread': '3.856', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.88', 'spread': '2.188', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.48', 'spread': '2.286', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.90', 'spread': '2.466', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.83', 'spread': '2.244', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.41', 'spread': '2.108', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 60 or Final Visit; max 15 months on treatment', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': '10^9 leukocytes/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Leukocytes Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Platelets', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '409.06', 'spread': '131.542', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '383.80', 'spread': '52.756', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '381.31', 'spread': '104.305', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '466.10', 'spread': '181.577', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '315.10', 'spread': '134.057', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '333.67', 'spread': '98.699', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '258.00', 'spread': '141.782', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '408.25', 'spread': '86.742', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 60 (or Final Visit), max 15 months on treatment', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': '10^9 platelets/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Mean Corpuscular Hemoglobin (MCH)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '27.57', 'spread': '3.741', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '23.80', 'spread': '3.291', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28.36', 'spread': '4.665', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28.53', 'spread': '1.015', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '33.36', 'spread': '4.590', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28.95', 'spread': '8.126', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '34.02', 'spread': '3.971', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '34.31', 'spread': '2.307', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 60 (or Final Visit), max 15 months on treatment', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': 'pg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Hematocrit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '25.02', 'spread': '4.4', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '30.60', 'spread': '3.161', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '23.24', 'spread': '3.378', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '24.30', 'spread': '4.081', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '27.74', 'spread': '3.995', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '30.38', 'spread': '3.035', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28.45', 'spread': '4.001', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '25.10', 'spread': '3.208', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': '% by volume of red blood cells in blood', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Bilirubin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '37.43', 'spread': '19.649', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14.78', 'spread': '9.763', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '39.60', 'spread': '19.777', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '47.53', 'spread': '12.702', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.12', 'spread': '16.191', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.42', 'spread': '1.941', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.25', 'spread': '13.331', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '39.89', 'spread': '13.191', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': 'umol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Elevation in Liver Function Tests (LFTs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'ALP Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.55', 'spread': '0.846', 'groupId': 'OG000'}]}]}, {'title': 'ALP Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.19', 'spread': '0.983', 'groupId': 'OG000'}]}]}, {'title': 'ALP Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.60', 'spread': '0.666', 'groupId': 'OG000'}]}]}, {'title': 'ALP Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.10', 'spread': '0.827', 'groupId': 'OG000'}]}]}, {'title': 'ALP Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.57', 'spread': '0.858', 'groupId': 'OG000'}]}]}, {'title': 'ALP Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.06', 'spread': '0.820', 'groupId': 'OG000'}]}]}, {'title': 'ALP Final Visit (2 months - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.41', 'spread': '0.578', 'groupId': 'OG000'}]}]}, {'title': 'ALP Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.52', 'spread': '1.237', 'groupId': 'OG000'}]}]}, {'title': 'GGT Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.38', 'spread': '0.333', 'groupId': 'OG000'}]}]}, {'title': 'GGT Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.16', 'spread': '0.034', 'groupId': 'OG000'}]}]}, {'title': 'GGT Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.32', 'spread': '0.231', 'groupId': 'OG000'}]}]}, {'title': 'GGT Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.62', 'spread': '0.437', 'groupId': 'OG000'}]}]}, {'title': 'GGT Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.32', 'spread': '0.233', 'groupId': 'OG000'}]}]}, {'title': 'GGT Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.16', 'spread': '0.040', 'groupId': 'OG000'}]}]}, {'title': 'GGT Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.29', 'spread': '0.174', 'groupId': 'OG000'}]}]}, {'title': 'GGT Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.49', 'spread': '0.316', 'groupId': 'OG000'}]}]}, {'title': 'AST Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.85', 'spread': '0.221', 'groupId': 'OG000'}]}]}, {'title': 'AST Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.72', 'spread': '0.178', 'groupId': 'OG000'}]}]}, {'title': 'AST Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.84', 'spread': '0.212', 'groupId': 'OG000'}]}]}, {'title': 'AST Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.92', 'spread': '0.244', 'groupId': 'OG000'}]}]}, {'title': 'AST Final Visit (Overallk)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.67', 'spread': '0.165', 'groupId': 'OG000'}]}]}, {'title': 'AST Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.61', 'spread': '0.066', 'groupId': 'OG000'}]}]}, {'title': 'AST Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.68', 'spread': '0.185', 'groupId': 'OG000'}]}]}, {'title': 'AST Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.71', 'spread': '0.181', 'groupId': 'OG000'}]}]}, {'title': 'ALT Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.33', 'spread': '0.128', 'groupId': 'OG000'}]}]}, {'title': 'ALT Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.34', 'spread': '0.053', 'groupId': 'OG000'}]}]}, {'title': 'ALT Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.30', 'spread': '0.129', 'groupId': 'OG000'}]}]}, {'title': 'ALT Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.35', 'spread': '0.158', 'groupId': 'OG000'}]}]}, {'title': 'ALT Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.31', 'spread': '0.110', 'groupId': 'OG000'}]}]}, {'title': 'ALT Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.33', 'spread': '0.067', 'groupId': 'OG000'}]}]}, {'title': 'ALT Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.30', 'spread': '0.109', 'groupId': 'OG000'}]}]}, {'title': 'ALT Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.32', 'spread': '0.142', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry, including Liver Function Tests (LFTs): Alkaline Phosphatase (ALP), Gamma Glutamyl Transferase (GGT), Aspartate Amino Transferase (AST), Alanine Aminotransferase (ALT)', 'unitOfMeasure': 'ukat/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Hemoglobin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.32', 'spread': '1.114', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.76', 'spread': '0.847', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.07', 'spread': '0.980', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.99', 'spread': '0.911', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.47', 'spread': '1.369', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.60', 'spread': '1.421', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.47', 'spread': '1.190', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.63', 'spread': '1.149', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 60 (or Final Visit); max 15 months on treatment', 'description': 'Safety and efficacy of hydroxyurea therapy', 'unitOfMeasure': 'g/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Bacterial Infections', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Prior to Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': 'events', 'reportingStatus': 'POSTED', 'populationDescription': "Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nAt screening, subjects' experiences of clinical SCA symptoms within the 12 months prior to the screening visit were recorded.\n\nAt subsequent visits, subjects' experiences of SCA symptoms since the previous visit were recorded, until end of study.\n\nEach subject counted a maximum of once each per SCA symptom for before treatment and after treatment."}, {'type': 'SECONDARY', 'title': 'Viral Infections', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Prior to Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '23', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': 'Events', 'reportingStatus': 'POSTED', 'populationDescription': "At screening, subjects' experiences of clinical SCA symptoms within the 12 months prior to the screening visit were recorded.\n\nAt subsequent visits, subjects' experiences of SCA symptoms since the previous visit were recorded, until end of study.\n\nEach subject counted a maximum of once each per SCA symptom for before treatment and after treatment."}, {'type': 'SECONDARY', 'title': 'Fungal Infections', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Prior to Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': 'events', 'reportingStatus': 'POSTED', 'populationDescription': "At screening, subjects' experiences of clinical SCA symptoms within the 12 months prior to the screening visit were recorded.\n\nAt subsequent visits, subjects' experiences of SCA symptoms since the previous visit were recorded, until end of study.\n\nEach subject counted a maximum of once each per SCA symptom for before treatment and after treatment."}, {'type': 'SECONDARY', 'title': 'Leg Ulcers', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Prior to Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 months -1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Prior to Treatment (6 -17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 months -1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'After Treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry', 'unitOfMeasure': 'events', 'reportingStatus': 'POSTED', 'populationDescription': "At screening, subjects' experiences of clinical SCA symptoms within the 12 months prior to the screening visit were recorded.\n\nAt subsequent visits, subjects' experiences of SCA symptoms since the previous visit were recorded, until end of study.\n\nEach subject counted a maximum of once each per SCA symptom for before treatment and after treatment."}, {'type': 'SECONDARY', 'title': 'Fetal Hemoglobin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.90', 'spread': '8.711', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '21.52', 'spread': '6.889', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.94', 'spread': '7.637', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.57', 'spread': '4.641', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '23.53', 'spread': '11.179', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.32', 'spread': '4.893', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '27.09', 'spread': '11.888', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12.47', 'spread': '6.467', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 60 (or Final Visit); max 15 months on treatment', 'description': 'Biomarker endpoints of Hydroxyurea efficacy', 'unitOfMeasure': 'Hemoglobin F/Total Hemoglobin %', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Mean Corpuscular Volume (MCV)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '83.13', 'spread': '8.643', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '76.42', 'spread': '7.682', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '83.25', 'spread': '9.715', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '86.30', 'spread': '5.410', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '97.64', 'spread': '13.700', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '81.68', 'spread': '14.506', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '102.49', 'spread': '11.736', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '100.51', 'spread': '6.859', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 60 (or Final Visit); max 15 months on treatment', 'description': 'Biomarker endpoints of Hydroxyurea efficacy', 'unitOfMeasure': 'fL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Cystatin C', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'PK1 Day 1 (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.52', 'spread': '1.288', 'groupId': 'OG000'}]}]}, {'title': 'PK1 Day 1 (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.89', 'spread': '1.170', 'groupId': 'OG000'}]}]}, {'title': 'PK1 Day 1 (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.87', 'spread': '1.001', 'groupId': 'OG000'}]}]}, {'title': 'PK1 Day 1 (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.18', 'spread': '0.549', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.50', 'spread': '1.173', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.36', 'spread': '0.263', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.07', 'spread': '1.074', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.52', 'spread': '1.810', 'groupId': 'OG000'}]}]}, {'title': 'Week 28 (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.67', 'spread': '0.134', 'groupId': 'OG000'}]}]}, {'title': 'Week 28 (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.76', 'spread': 'NA', 'comment': 'No SD possible when only n=1 participant analysed at this timepoint for this age group', 'groupId': 'OG000'}]}]}, {'title': 'Week 28 (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.57', 'spread': 'NA', 'comment': 'No SD possible when only n=1 participant analysed at this timepoint for this age group', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.44', 'spread': '1.340', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.63', 'spread': '0.999', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.19', 'spread': '1.253', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.80', 'spread': '1.192', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PK1 (day 1), week 20-32 (6 months) and Week 60 (Final Visit)', 'description': 'Biomarker Endpoints', 'unitOfMeasure': 'nmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Baseline value is the last non-missing assessment prior to first exposure of oral HU.\n\nAnalysis was from initiation of study drug, again at \\~6months (between 20-32 weeks) and at final study visit.\n\nWeek 28 (6 months - 1.99 years) no measures were analysed at this timepoint.'}, {'type': 'SECONDARY', 'title': 'Incidence of Acute Vaso-Occlusive Pain Crises (VOC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'VOC 12months prior (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.5', 'spread': '0.92', 'groupId': 'OG000'}]}]}, {'title': 'VOC 12months prior (6 months - 1.99 years))', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '0.0', 'groupId': 'OG000'}]}]}, {'title': 'VOC 12months prior (2 - 5.99 years))', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.7', 'spread': '1.08', 'groupId': 'OG000'}]}]}, {'title': 'VOC 12months prior (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6', 'spread': '0.84', 'groupId': 'OG000'}]}]}, {'title': 'VOC 12months after treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.51', 'groupId': 'OG000'}]}]}, {'title': 'VOC 12months after treatment (6 months - 1.99 years))', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.82', 'groupId': 'OG000'}]}]}, {'title': 'VOC 12months after treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '0.0', 'groupId': 'OG000'}]}]}, {'title': 'VOC 12months after treatment (6 - 17.99 years))', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.67', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for vaso-occlusive crisis (Mean \\[SD\\])', 'unitOfMeasure': 'hospitalisation events per month', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Time-averaged hospitalizations after treatment was determined by dividing incidence of hospitalizations prior to treatment by 12 if study end date was more than a year after treatment start date, or by dividing by (End date - Treatment Start Date)/30.4375 otherwise. Hospitalizations did not include visits to ER/accident without hospitalization. Hospitalizations were sub-categorized by a medical monitor. Data were obtained through medical record review and study participant recall.'}, {'type': 'SECONDARY', 'title': 'Number and Frequency of Blood Transfusions', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Overall', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '30', 'groupId': 'OG000'}]}]}, {'title': '2 years - 5.99 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '21', 'groupId': 'OG000'}]}]}, {'title': '6 years - 17.99 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to Week 60', 'description': 'Clinical status endpoints, related to blood transfusions for treatment of SCA, this may be hospitalization, A\\&E, or in-clinic treatment from safety population (n=32)', 'unitOfMeasure': 'events (number of transfusions)', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.\n\nAge group (6 months to 1.99 years) did not report any blood transfusions.'}, {'type': 'SECONDARY', 'title': 'Acute Chest Syndrome (ACS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'ACS Hospitalisations 12 months prior (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.5', 'spread': '0.92', 'groupId': 'OG000'}]}]}, {'title': 'ACS Hospitalisations 12 months prior (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'ACS Hospitalisations 12 months prior (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6', 'spread': '0.89', 'groupId': 'OG000'}]}]}, {'title': 'ACS Hospitalisations 12 months prior (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.7', 'spread': '1.16', 'groupId': 'OG000'}]}]}, {'title': 'ACS Hospitalisations 12 months after treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.37', 'groupId': 'OG000'}]}]}, {'title': 'ACS Hospitalisations 12 months after treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'ACS Hospitalisations 12 months after treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.45', 'groupId': 'OG000'}]}]}, {'title': 'ACS Hospitalisations 12 months after treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1', 'spread': '0.32', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for Acute Chest Syndrome (Mean \\[SD\\])', 'unitOfMeasure': 'hospitalisation events per month', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Time-averaged hospitalizations after treatment was determined by dividing incidence of hospitalizations prior to treatment by 12 if study end date was more than a year after treatment start date, or by dividing by (End date - Treatment Start Date)/30.4375 otherwise. Hospitalizations did not include visits to ER/accident without hospitalization. Hospitalizations were sub-categorized by a medical monitor. Data were obtained through medical record review and study participant recall.'}, {'type': 'SECONDARY', 'title': 'Hospitalizations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Hospitalisations 12 months prior (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.7', 'spread': '1.54', 'groupId': 'OG000'}]}]}, {'title': 'Hospitalisations 12 months prior (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.82', 'groupId': 'OG000'}]}]}, {'title': 'Hospitalisations 12 months prior (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.0', 'spread': '1.59', 'groupId': 'OG000'}]}]}, {'title': 'Hospitalisations 12 months prior (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.9', 'spread': '1.45', 'groupId': 'OG000'}]}]}, {'title': 'Hospitalisations 12 months after treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.65', 'groupId': 'OG000'}]}]}, {'title': 'Hospitalisations 12 months after treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.82', 'groupId': 'OG000'}]}]}, {'title': 'Hospitalisations 12 months after treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.45', 'groupId': 'OG000'}]}]}, {'title': 'Hospitalisations 12 months after treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.5', 'spread': '0.85', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Clinical Status endpoints, all hospitalisations (not ER/Accident without hospitalization) (Mean \\[SD\\])', 'unitOfMeasure': 'hospitalisation events per month', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Time-averaged hospitalizations after treatment was determined by dividing incidence of hospitalizations prior to treatment by 12 if study end date was more than a year after treatment start date, or by dividing by (End date - Treatment Start Date)/30.4375 otherwise. Hospitalizations did not include visits to ER/accident without hospitalization. Hospitalizations were sub-categorized by a medical monitor. Data were obtained through medical record review and study participant recall.'}, {'type': 'SECONDARY', 'title': 'Dose Escalation i.e. Maximum Tolerated Dose (MTD)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'MTD (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '25.63', 'spread': '6.780', 'groupId': 'OG000'}]}]}, {'title': 'MTD (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '23.50', 'spread': '8.944', 'groupId': 'OG000'}]}]}, {'title': 'MTD (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.11', 'spread': '5.465', 'groupId': 'OG000'}]}]}, {'title': 'MTD (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.67', 'spread': '7.528', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Screening Up to Final Visit (Week 60 or Withdrawal), maximum 15 months on IMP', 'description': 'Summary of maximum tolerated dose achieved in mg/kg (Mean \\[SD\\])', 'unitOfMeasure': 'mg/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Clinical status endpoints; Safety population = 32 participants, who received at least one dose of IMP.\n\nWeek 60 is the final study visit, or withdrawal point at which point IMP was halted.\n\nMTD only summarized for subjects who investigator denoted as achieving MTD during the study (n=20). If several doses were marked as MTD for the subject, the highest dose was used.'}, {'type': 'SECONDARY', 'title': 'Other SCA-related Hospitalizations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Other SCA related Hospitalizations 12 months prior (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '0.67', 'groupId': 'OG000'}]}]}, {'title': 'Other SCA related Hospitalizations 12 months prior (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.82', 'groupId': 'OG000'}]}]}, {'title': 'Other SCA related Hospitalizations 12 months prior (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '0.63', 'groupId': 'OG000'}]}]}, {'title': 'Other SCA related Hospitalizations 12 months prior (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '0.70', 'groupId': 'OG000'}]}]}, {'title': 'Other SCA related Hospitalizations 12 months after treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '0.18', 'groupId': 'OG000'}]}]}, {'title': 'Other SCA related Hospitalizations 12 months after treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Other SCA related Hospitalizations 12 months after treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Other SCA related Hospitalizations 12 months after treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.1', 'spread': '0.32', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other Non-SCA related) (mean \\[SD\\])', 'unitOfMeasure': 'hospitalisation events per month', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Time-averaged hospitalizations after treatment was determined by dividing incidence of hospitalizations prior to treatment by 12 if study end date was more than a year after treatment start date, or by dividing by (End date - Treatment Start Date)/30.4375 otherwise. Hospitalizations did not include visits to ER/accident without hospitalization. Hospitalizations were sub-categorized by a medical monitor. Data were obtained through medical record review and study participant recall.'}, {'type': 'SECONDARY', 'title': 'Parent/Caregiver Palatability and Acceptability Questionnaire', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Taste (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '84.2', 'spread': '26.21', 'groupId': 'OG000'}]}]}, {'title': 'Taste (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '71.3', 'spread': '23.30', 'groupId': 'OG000'}]}]}, {'title': 'Taste (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '91.1', 'spread': '23.86', 'groupId': 'OG000'}]}]}, {'title': 'Taste (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '80.8', 'spread': '30.14', 'groupId': 'OG000'}]}]}, {'title': 'Smell (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '83.8', 'spread': '21.73', 'groupId': 'OG000'}]}]}, {'title': 'Smell (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '74.8', 'spread': '25.51', 'groupId': 'OG000'}]}]}, {'title': 'Smell (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '87.5', 'spread': '17.26', 'groupId': 'OG000'}]}]}, {'title': 'Smell (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '83.1', 'spread': '26.28', 'groupId': 'OG000'}]}]}, {'title': 'Aftertaste (>6 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '67.6', 'spread': '23.21', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Taken once at any point after 8 weeks on study medication (or at early Withdrawal)', 'description': 'Clinical status endpoints. Visual Analogue scale used to determine, taste, smell, aftertaste, acceptability and ease of dosing (1-100mm scale) with 1 being worst possible and 100 being best possible.\n\nAssessed for \\<6 years of age by parents/guardians. Assessed for \\>6 years of age, combination of parent/guardian and participant responses.', 'unitOfMeasure': 'mm', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Results are presented for all 32 safety participants.'}, {'type': 'SECONDARY', 'title': 'Vitamin D', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '59.35', 'spread': '32.613', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '83.07', 'spread': '32.329', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '65.43', 'spread': '29.076', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '35.39', 'spread': '24.403', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '62.26', 'spread': '30.220', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '86.44', 'spread': '25.475', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '57.54', 'spread': '34.093', 'groupId': 'OG000'}]}]}, {'title': 'Final Visit (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '52.97', 'spread': '14.749', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Screening Up to Final Visit (Week 60 or WD)', 'description': 'Biochemistry', 'unitOfMeasure': 'nmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population = 32 participants, who received at least one dose of oral hydroxyurea.\n\nBaseline value is the last non-missing assessment prior to first exposure of oral hydroxyurea.\n\nWeek 60 is the final study visit or withdrawal at which point oral hydroxyurea was halted.'}, {'type': 'SECONDARY', 'title': 'Other Non-SCA-related Hospitalizations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Other Non-SCA related Hospitalizations 12 months prior (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.47', 'groupId': 'OG000'}]}]}, {'title': 'Other Non-SCA related Hospitalizations 12 months prior (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Other Non-SCA related Hospitalizations 12 months prior (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.58', 'groupId': 'OG000'}]}]}, {'title': 'Other Non-SCA related Hospitalizations 12 months prior (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.42', 'groupId': 'OG000'}]}]}, {'title': 'Other Non-SCA related Hospitalizations 12 months after treatment (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Other Non-SCA related Hospitalizations 12 months after treatment (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Other Non-SCA related Hospitalizations 12 months after treatment (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Other Non-SCA related Hospitalizations 12 months after treatment (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '12 months prior to treatment and 12 months post-treatment; maximum 15 months on IMP', 'description': 'Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other SCA-related)', 'unitOfMeasure': 'events', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Hospitalizations 'Prior to Treatment' having a start date within 1 year prior to initial treatment. Hospitalizations 'After Treatment' defined as all hospitalizations (not ER/Accident without hospitalization) within 1 year after treatment start date."}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Transcranial Doppler Velocity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'classes': [{'title': 'Screening (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '141.5', 'spread': '34.71', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '90.5', 'spread': '9.33', 'groupId': 'OG000'}]}]}, {'title': 'Screening (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '150.0', 'spread': '34.78', 'groupId': 'OG000'}]}]}, {'title': 'Screening (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '148.3', 'spread': '22.42', 'groupId': 'OG000'}]}]}, {'title': 'Week 40-56 (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '133.7', 'spread': '26.52', 'groupId': 'OG000'}]}]}, {'title': 'Week 40-56 (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '95.7', 'spread': '15.18', 'groupId': 'OG000'}]}]}, {'title': 'Week 40-56 (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '138.6', 'spread': '24.86', 'groupId': 'OG000'}]}]}, {'title': 'Week 40-56 (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '140.7', 'spread': '20.79', 'groupId': 'OG000'}]}]}, {'title': 'Baseline Shift (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-11.5', 'spread': '20.74', 'groupId': 'OG000'}]}]}, {'title': 'Baseline Shift (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.0', 'spread': 'NA', 'comment': 'Insufficient sample size.', 'groupId': 'OG000'}]}]}, {'title': 'Baseline Shift (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-13.7', 'spread': '22.02', 'groupId': 'OG000'}]}]}, {'title': 'Baseline Shift (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-9.0', 'spread': '20.68', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 40-56 (Follow up scan).', 'description': 'Exploratory Endpoint; clinical output of hydroxyurea treatment', 'unitOfMeasure': 'cm/sec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Time Averaged Mean Maximum Velocity (TAMV) in cm/sec at Baseline and Week 40-56 Visit (Safety Population).\n\nmax 15 months on treatment at Week 60.\n\nOnly participants with non-missing values at screening and end of study are included in the Baseline shift summary (change from baseline).'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'comment': 'Overall (Screened)', 'achievements': [{'comment': '36 Participants were screened but 33 enrolled', 'groupId': 'FG000', 'numSubjects': '33'}]}, {'type': 'Pre-treatment Discontinuation', 'achievements': [{'comment': '1 participant discontinued prior to treatment initiation. The participant was excluded from the safety population but was included in some baseline data.', 'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Safety Population', 'achievements': [{'comment': '32 participants (split into age categories of 6months-1.99 years, 2-5.99 years and 6-17.99 years) were included in the safety population and initiated treatment with hydroxyurea.', 'groupId': 'FG000', 'numSubjects': '32'}]}, {'type': 'Age Group (6 Months - 1.99 Years)', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'Discontinuations From Age Group (6 Months - 1.99 Years)', 'achievements': [{'comment': 'Reasons for discontinuation:\n\nn=1 physician decision n=2 other (participants decision not to continue)', 'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Completed From Age Group (6 Months - 1.99 Years)', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Age Group (2 - 5.99 Years)', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}]}, {'type': 'Discontinuations From Age Group (2 - 5.99 Years)', 'achievements': [{'comment': 'Reason for discontinuation:\n\nn=2 other (migrated; participants decision to withdraw unwilling to travel)', 'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Completions From Age Group (2 - 5.99 Years)', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'Age Group (6 - 17.99 Years)', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'Discontinuations From Age Group (6 - 17.99 Years)', 'achievements': [{'comment': 'Reason for discontinuation:\n\nn=1 protocol deviation n=2 other (participant unwilling to continue, non compliant)', 'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Completions From Age Group (6 - 17.99 Years)', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'COMPLETED', 'comment': 'Overall from all age groups', 'achievements': [{'groupId': 'FG000', 'numSubjects': '24'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '7'}]}]}], 'recruitmentDetails': 'Participants were recruited from one Jamaican sickle cell clinic (n=12) and five UK hospitals (n=20). The first participant was enrolled on 03 Jan 2019 and the last participant was enrolled on 16 Dec 2020.', 'preAssignmentDetails': '33 participants enrolled met the inclusion criteria; 32 initiated treatment with open label Hydroxyurea and one participant discontinued pre-treatment.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Oral Hydroxyurea', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.\n\nHydroxyurea: Oral Hydroxyurea 100 mg/ml solution'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': 'Age by Category', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Age Group (6 months - 1.99 years)', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Age Group (2 - 5.99 years)', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}, {'title': 'Age Group (6 - 17.99 years)', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'title': 'Sex: Female, Male (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '17', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}, {'title': 'Sex: Female, Male (Age group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Sex: Female, Male (Age group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}]}, {'title': 'Sex: Female, Male (Age group 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Overall demographic population of 33 participants.'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'title': 'Ethnicity Overall', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '33', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Ethnicity (Age Group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Ethnicity (Age Group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Ethnicity (Age Group 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Overall demographic population of 33 participants.'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Race (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '31', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): [sic] Caribbean', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): Black British', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Race (Age Group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): [sic] Caribbean', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): Black British', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Race (Age Group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '15', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): [sic] Caribbean', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): Black British', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Race (Age Group 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): [sic] Caribbean', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Other (Specify): Black British', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Measure Analysis Population Description: Overall demographic population of 33 participants. This has been further subdivided to give the demographic per age group.\n\nAll participants identified as Black or African American except for two UK participants who self-described as Other: "Caribbean" \\[sic\\] and "Black British".'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United Kingdom', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '20', 'groupId': 'BG000'}]}]}, {'title': 'Jamaica', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'SCA Type', 'classes': [{'title': 'SCA Type (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'BG000'}]}], 'categories': [{'title': 'HbSS', 'measurements': [{'value': '32', 'groupId': 'BG000'}]}, {'title': 'HbSbeta0', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'SCA Type (Age Group 6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'title': 'HbSS', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'HbSbeta0', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'SCA Type (Age Group 2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}]}], 'categories': [{'title': 'HbSS', 'measurements': [{'value': '15', 'groupId': 'BG000'}]}, {'title': 'HbSbeta0', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'SCA Type (Age Group 6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}]}], 'categories': [{'title': 'HbSS', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'HbSbeta0', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Measure Analysis Population Description: SCD Genotype of overall demographic population of 33 participants.', 'unitOfMeasure': 'Participants', 'populationDescription': 'SCD Genotype of overall demographic population of 33 participants. SCD is caused by a mutation in the β-globin gene HBB. Hemoglobin SS (HbSS) represents a large proportion of SCD in the Americas, UK, and certain regions of Africa while higher proportions of hemoglobin SC are observed in Burkina Faso and hemoglobin Sβ-thalassemia (HbS/β0 or HbS/β+) in Greece and India. Patients with HbSS and HbS/βo-thalassaemia typically have a more severe course of disease than other genotypes.'}, {'title': 'Height', 'classes': [{'title': 'Height (cm) (Overall)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '109.26', 'spread': '26.038', 'groupId': 'BG000'}]}]}, {'title': 'Height (cm) Age Group (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '79.40', 'spread': '5.237', 'groupId': 'BG000'}]}]}, {'title': 'Height (cm) Age Group (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '98.08', 'spread': '6.947', 'groupId': 'BG000'}]}]}, {'title': 'Height (cm) Age Group (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '142.09', 'spread': '15.872', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'Height in (cm) for safety population at baseline, defined as the last non-missing measurement prior to first exposure to oral hydroxurea.', 'unitOfMeasure': 'cm', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Overall safety population of 32 participants, data available at screening for 31 participants.\n\nHeight or length was unobtainable for 1 participant who was under the age of 2 years (Age group 6 months - 1.99 years).'}, {'title': 'Weight', 'classes': [{'title': 'Weight (kg) Overall', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '19.63', 'spread': '10.955', 'groupId': 'BG000'}]}]}, {'title': 'Weight (kg) Age Group (6 months - 1.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '11.18', 'spread': '1.017', 'groupId': 'BG000'}]}]}, {'title': 'Weight (kg) Age Group (2 - 5.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '14.74', 'spread': '2.092', 'groupId': 'BG000'}]}]}, {'title': 'Weight (kg) Age Group (6 - 17.99 years)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '32.52', 'spread': '11.441', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'Weight in (kg) for safety population at baseline, defined as the last non-missing measurement prior to first exposure to oral hydroxyurea.', 'unitOfMeasure': 'kg', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Overall safety population of 32 participants.'}], 'populationDescription': 'Participant demographics were summarized for all 33 participants. One participant discontinued prior to receiving a first dose of IMP. The remaining safety population consisted of 32 participants who all received at least one dose of the IMP and one valid primary efficacy PK measurement. This safety population was used for all analyses of baseline characteristics, secondary and exploratory outcomes, as well as safety outcomes.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-06-03', 'size': 1289835, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-12-14T06:33', 'hasProtocol': True}, {'date': '2022-01-21', 'size': 983429, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-12-14T06:34', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open label, observational, pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months-17.99 years over a 12-15-month period'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 33}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-01-03', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-08', 'completionDateStruct': {'date': '2021-12-29', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-08-12', 'studyFirstSubmitDate': '2018-12-03', 'resultsFirstSubmitDate': '2022-12-21', 'studyFirstSubmitQcDate': '2018-12-03', 'lastUpdatePostDateStruct': {'date': '2024-10-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-08-12', 'studyFirstPostDateStruct': {'date': '2018-12-04', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-10-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-05-19', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Transcranial Doppler Velocity', 'timeFrame': 'Baseline to Week 40-56 (Follow up scan).', 'description': 'Exploratory Endpoint; clinical output of hydroxyurea treatment'}], 'primaryOutcomes': [{'measure': 'Clearance (CL/F)', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).'}, {'measure': 'Volume of Distribution (V/F)', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).'}, {'measure': 'Time to Maximum Concentration (Tmax)', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean Tmax (h) pharmacokinetic parameter derived using the final population PK model.'}, {'measure': 'Maximum Plasma Concentration Cmax (ug/mL)', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean Cmax (ug/mL) pharmacokinetic parameter derived using the final population PK model.'}, {'measure': 'Area Under Plasma Concentration Time Curve (AUC 0-Inf)', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean AUC 0-Infinity (hr\\*ug/mL) pharmacokinetic parameters derived using the final population PK model.'}, {'measure': 'Terminal Half-life (Hours)', 'timeFrame': '0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)', 'description': 'Mean Terminal Half-life (hours) pharmacokinetic parameter derived using the final population PK model.'}], 'secondaryOutcomes': [{'measure': 'Adverse Events', 'timeFrame': 'Screening Up to Week 64', 'description': "Incidence of Adverse events during the course of the trial from screening to final follow up phone call at Week 64.\n\nRelatedness refers to 'at least possibly related to the IMP', with severity/toxicity assessed using the CTCAE Toxicity Grade and seriousness based on the standard definition for SAE in accordance with GCP. With the exception of SCA related events, requiring \\>7day hospitalisation, or extension of hospitalisation before being reported as an SAE due to their frequency within the disease population. All other non-SCA related SAEs were reportable."}, {'measure': 'Absolute Neutrophil Count (ANC)', 'timeFrame': 'Baseline and Week 60 (or final visit); max 15 months on treatment', 'description': 'Safety review for haematological toxicity (mild myelosuppression target: 1-3x10\\^9/L)'}, {'measure': 'White Blood Cell Count (Leukocytes)', 'timeFrame': 'Baseline to Week 60 or Final Visit; max 15 months on treatment', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Platelets', 'timeFrame': 'Baseline to Week 60 (or Final Visit), max 15 months on treatment', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Mean Corpuscular Hemoglobin (MCH)', 'timeFrame': 'Baseline to Week 60 (or Final Visit), max 15 months on treatment', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Hematocrit', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Bilirubin', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Elevation in Liver Function Tests (LFTs)', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry, including Liver Function Tests (LFTs): Alkaline Phosphatase (ALP), Gamma Glutamyl Transferase (GGT), Aspartate Amino Transferase (AST), Alanine Aminotransferase (ALT)'}, {'measure': 'Hemoglobin', 'timeFrame': 'Baseline to Week 60 (or Final Visit); max 15 months on treatment', 'description': 'Safety and efficacy of hydroxyurea therapy'}, {'measure': 'Bacterial Infections', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Viral Infections', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Fungal Infections', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Leg Ulcers', 'timeFrame': 'Up to Week 60', 'description': 'Impact of Hydroxyurea on Safety, Hematology and Biochemistry'}, {'measure': 'Fetal Hemoglobin', 'timeFrame': 'Baseline to Week 60 (or Final Visit); max 15 months on treatment', 'description': 'Biomarker endpoints of Hydroxyurea efficacy'}, {'measure': 'Mean Corpuscular Volume (MCV)', 'timeFrame': 'Baseline to Week 60 (or Final Visit); max 15 months on treatment', 'description': 'Biomarker endpoints of Hydroxyurea efficacy'}, {'measure': 'Cystatin C', 'timeFrame': 'PK1 (day 1), week 20-32 (6 months) and Week 60 (Final Visit)', 'description': 'Biomarker Endpoints'}, {'measure': 'Incidence of Acute Vaso-Occlusive Pain Crises (VOC)', 'timeFrame': '12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for vaso-occlusive crisis (Mean \\[SD\\])'}, {'measure': 'Number and Frequency of Blood Transfusions', 'timeFrame': 'Up to Week 60', 'description': 'Clinical status endpoints, related to blood transfusions for treatment of SCA, this may be hospitalization, A\\&E, or in-clinic treatment from safety population (n=32)'}, {'measure': 'Acute Chest Syndrome (ACS)', 'timeFrame': '12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for Acute Chest Syndrome (Mean \\[SD\\])'}, {'measure': 'Hospitalizations', 'timeFrame': '12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Clinical Status endpoints, all hospitalisations (not ER/Accident without hospitalization) (Mean \\[SD\\])'}, {'measure': 'Dose Escalation i.e. Maximum Tolerated Dose (MTD)', 'timeFrame': 'Screening Up to Final Visit (Week 60 or Withdrawal), maximum 15 months on IMP', 'description': 'Summary of maximum tolerated dose achieved in mg/kg (Mean \\[SD\\])'}, {'measure': 'Other SCA-related Hospitalizations', 'timeFrame': '12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP', 'description': 'Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other Non-SCA related) (mean \\[SD\\])'}, {'measure': 'Parent/Caregiver Palatability and Acceptability Questionnaire', 'timeFrame': 'Taken once at any point after 8 weeks on study medication (or at early Withdrawal)', 'description': 'Clinical status endpoints. Visual Analogue scale used to determine, taste, smell, aftertaste, acceptability and ease of dosing (1-100mm scale) with 1 being worst possible and 100 being best possible.\n\nAssessed for \\<6 years of age by parents/guardians. Assessed for \\>6 years of age, combination of parent/guardian and participant responses.'}, {'measure': 'Vitamin D', 'timeFrame': 'From Screening Up to Final Visit (Week 60 or WD)', 'description': 'Biochemistry'}, {'measure': 'Other Non-SCA-related Hospitalizations', 'timeFrame': '12 months prior to treatment and 12 months post-treatment; maximum 15 months on IMP', 'description': 'Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other SCA-related)'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hydroxyurea', 'Hydroxycarbamide', 'Xromi'], 'conditions': ['Sickle Cell Disease', 'Sickle-Cell; Hemoglobin Disease, Thalassemia', 'Sickle Cell-beta-thalassemia', 'Sickle Cell Hemoglobin C']}, 'descriptionModule': {'briefSummary': 'An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e. to the day before 18th birthday), with a 12 to 15 month treatment period for each participant. The study treatment duration will be for 6 months at the maximum tolerated dose \\[MTD\\], which is usually reached by 6 months after initiation of treatment. For patients in whom time to MTD is longer than 6 months or not achieved at all, the maximum duration of study treatment will be 15 months.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Male or female aged from 6 months to 17.99 years of age (i.e. to the day before 18th birthday).\n2. Diagnosis of sickle cell anemia (HbSS and HbSβº).\n3. Parent(s)/legal guardian able and willing to provide written informed consent for the child to take part in the study.\n4. Where applicable, the child should assent to undergo blood sampling for pharmacokinetic and biochemistry purposes and to allow physiological measurements to be made.\n\nExclusion Criteria:\n\n1. Any clinically significant medical condition or abnormality, which, in the opinion of the Investigator, might have compromised the safety of the patient or which might have interfered with the study.\n2. Hydroxyurea use within 6 months before enrolment.\n3. Renal insufficiency (known creatinine more than twice the upper limit of normal (ULN) for age and \\>1.0 mg/dL \\[88.4 μmol/L\\]).\n4. Clinical evidence of hepatic compromise with alanine aminotransferase (ALT) \\>3 times the ULN (a temporary swing in ALT did not result in exclusion).\n5. Other significant organ system dysfunction based on the site Investigators discretion.\n6. Severe active infections: fungal, viral or bacterial (as confirmed by culture), examples included tuberculosis, malaria, active hepatitis, osteomyelitis or any other illness that would have precluded the use of HU in normal clinical practice.\n7. Active chronic leg ulcers.\n8. Known allergy to oral HU solution or any of the excipients.\n9. Positive pregnancy test for females of child-bearing potential (in post-menarcheal females) before initiation of treatment, unless participant was sexually abstinent. Note: True abstinence was considered as being in line with the preferred and usual lifestyle of the participant. Periodic abstinence (such as calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.\n10. Inadequate contraception measures in sexually active females (post-menarcheal females) and males of child-bearing age (see Section 9.5.1.10.4).\n11. Breastfeeding at study initiation.\n12. Participation in another clinical trial of an IMP.\n13. Known infection with HIV.'}, 'identificationModule': {'nctId': 'NCT03763656', 'acronym': 'HUPK', 'briefTitle': 'Pharmacokinetics of Oral Hydroxyurea Solution', 'organization': {'class': 'INDUSTRY', 'fullName': 'Nova Laboratories Limited'}, 'officialTitle': 'A Prospective Open Label, Pharmacokinetic Study of an Oral Hydroxyurea Solution in Children With Sickle Cell Anemia.', 'orgStudyIdInfo': {'id': 'INV543'}, 'secondaryIdInfos': [{'id': '2017-004568-37', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open Label', 'description': 'Novel oral solution formulation of hydroxyurea', 'interventionNames': ['Drug: Oral Hydroxyurea (100 mg/mL) Solution']}], 'interventions': [{'name': 'Oral Hydroxyurea (100 mg/mL) Solution', 'type': 'DRUG', 'description': 'Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.', 'armGroupLabels': ['Open Label']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Kingston', 'country': 'Jamaica', 'facility': 'Dr Angela E Rankine- Mullings', 'geoPoint': {'lat': 17.99702, 'lon': -76.79358}}, {'city': 'Birmingham', 'country': 'United Kingdom', 'facility': "Birmingham Women's and Children's NHS Foundation Trust", 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'Liverpool', 'country': 'United Kingdom', 'facility': "Alder Hey Children's NHS Foundation Trust", 'geoPoint': {'lat': 53.41058, 'lon': -2.97794}}, {'city': 'London', 'country': 'United Kingdom', 'facility': "Evelina London Children's Hospital", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'country': 'United Kingdom', 'facility': "King's College Hospital NHS Foundation Trust", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'country': 'United Kingdom', 'facility': "The Royal London Children's Hospital, Barts Health NHS Trust", 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Angela E Rankine- Mullings, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of the West Indies, Mona, Kingston, Jamaica'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Nova Laboratories Limited', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}