Ignite Creation Date:
2025-12-24 @ 9:31 PM
Ignite Modification Date:
2026-01-04 @ 3:45 AM
Study NCT ID:
NCT03848832
Status:
TERMINATED
Last Update Posted:
2022-09-02
First Post:
2019-02-19
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2021-11-11', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D015518', 'term': 'Rett Syndrome'}], 'ancestors': [{'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D002185', 'term': 'Cannabidiol'}], 'ancestors': [{'id': 'D002186', 'term': 'Cannabinoids'}, {'id': 'D013729', 'term': 'Terpenes'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@gbio.com', 'phone': '1-833-424-6724', 'title': 'Medical Enquiries', 'organization': 'GW Research Ltd'}, 'certainAgreement': {'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'This study was terminated early due to enrollment challenges and the Coronavirus disease-2019 (COVID-19) pandemic. Numbers of participants in each treatment groups were small, which precluded the planned formal statistical inferences and limited data interpretation.'}}, 'adverseEventsModule': {'timeFrame': 'Up to approximately 207 days', 'description': 'Safety Analysis Set: All participants who received at least 1 dose of investigational medicinal product in the trial were included and analyzed according to the treatment received. One participant who was assigned to the placebo group received GWP42003-P 5 mg/kg. For safety analyses, this participant was assigned to the GWP42003-P 5 mg/kg group.', 'eventGroups': [{'id': 'EG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).', 'otherNumAtRisk': 11, 'deathsNumAtRisk': 11, 'otherNumAffected': 9, 'seriousNumAtRisk': 11, 'deathsNumAffected': 0, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.', 'otherNumAtRisk': 9, 'deathsNumAtRisk': 9, 'otherNumAffected': 7, 'seriousNumAtRisk': 9, 'deathsNumAffected': 0, 'seriousNumAffected': 2}, {'id': 'EG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.', 'otherNumAtRisk': 9, 'deathsNumAtRisk': 9, 'otherNumAffected': 7, 'seriousNumAtRisk': 9, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Coagulopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Eosinophilia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Macrocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Ocular hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Faeces soft', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Tooth discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Sunburn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Blood thyroid stimulating hormone increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Breath sounds abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Mean cell volume increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Hypoglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Selenium deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Joint stiffness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Scoliosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Balance disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Disturbance in attention', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Dizziness postural', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Drooling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Hyperreflexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Somnolence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Agitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Bruxism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Dermatillomania', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Middle insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Nervousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Sleep disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Choking', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Hiccups', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Hyperventilation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Skin discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}], 'seriousEvents': [{'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}, {'term': 'Dyskinesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 9, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDra 21.1'}], 'frequencyThreshold': '1'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '45.9', 'spread': '16.77', 'groupId': 'OG000'}, {'value': '50.0', 'spread': '7.56', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-12.1', 'spread': '13.63', 'groupId': 'OG000'}, {'value': '-6.1', 'spread': '7.22', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': 'RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). The total summed score ranges from 0 to 90, with higher scores representing greater severity.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-to-Treat (ITT) Set: All randomized participants who received at least 1 dose of drug in the trial, and had Baseline efficacy data. Participants with non-missing data were included for analysis.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '39.8', 'spread': '12.80', 'groupId': 'OG000'}, {'value': '50.0', 'spread': '7.56', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '12.51', 'groupId': 'OG000'}, {'value': '-6.1', 'spread': '7.22', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': 'RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). The total summed score ranges from 0 to 90, with higher scores represent greater severity.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Set. Participants with non-missing data were included for analysis.'}, {'type': 'SECONDARY', 'title': 'Mean Clinical Global Impressions - Improvement (CGI-I) Score at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'OG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.2', 'spread': '1.20', 'groupId': 'OG000'}, {'value': '2.9', 'spread': '1.07', 'groupId': 'OG001'}, {'value': '3.1', 'spread': '0.38', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': "CGI-I is a 7-point scale that requires the clinician to assess how much a participant's illness has improved or worsened relative to a Baseline state at the beginning of the intervention. This is rated as: 1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; or 7 = very much worse.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Set. Participants with non-missing data were included for analysis.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Mean Clinician Global Impressions - Severity (CGI-S) Score at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'OG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '4.3', 'spread': '1.25', 'groupId': 'OG000'}, {'value': '4.2', 'spread': '0.67', 'groupId': 'OG001'}, {'value': '4.4', 'spread': '0.84', 'groupId': 'OG002'}]}]}, {'title': 'Change from Baseline at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.1', 'spread': '0.33', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '0.38', 'groupId': 'OG001'}, {'value': '0.0', 'spread': '0.00', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': "CGI-S is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment relative to the clinician's experience with participants who had the same diagnosis. This is rated as: 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; or 7 = extremely ill.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Set. Participants with non-missing data were included for analysis.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Mean RSBQ Subscale Scores at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'OG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'classes': [{'title': 'Baseline General Mood Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '6.0', 'spread': '3.65', 'groupId': 'OG000'}, {'value': '8.2', 'spread': '4.58', 'groupId': 'OG001'}, {'value': '8.9', 'spread': '3.14', 'groupId': 'OG002'}]}]}, {'title': 'CFB General Mood Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.6', 'spread': '2.70', 'groupId': 'OG000'}, {'value': '-4.9', 'spread': '2.67', 'groupId': 'OG001'}, {'value': '-2.6', 'spread': '2.57', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Breathing Problems Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '4.7', 'spread': '2.45', 'groupId': 'OG000'}, {'value': '5.0', 'spread': '3.08', 'groupId': 'OG001'}, {'value': '5.0', 'spread': '3.43', 'groupId': 'OG002'}]}]}, {'title': 'CFB Breathing Problems Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '1.86', 'groupId': 'OG000'}, {'value': '-1.0', 'spread': '1.53', 'groupId': 'OG001'}, {'value': '-0.3', 'spread': '1.25', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Hand Behaviors Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '7.8', 'spread': '2.15', 'groupId': 'OG000'}, {'value': '7.6', 'spread': '3.54', 'groupId': 'OG001'}, {'value': '9.1', 'spread': '2.08', 'groupId': 'OG002'}]}]}, {'title': 'CFB Hand Behaviors Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.7', 'spread': '2.24', 'groupId': 'OG000'}, {'value': '-0.9', 'spread': '3.29', 'groupId': 'OG001'}, {'value': '0.1', 'spread': '0.90', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Face Movements Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2.3', 'spread': '2.45', 'groupId': 'OG000'}, {'value': '4.6', 'spread': '2.13', 'groupId': 'OG001'}, {'value': '4.3', 'spread': '2.21', 'groupId': 'OG002'}]}]}, {'title': 'CFB Face Movements Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '1.12', 'groupId': 'OG000'}, {'value': '-1.4', 'spread': '1.72', 'groupId': 'OG001'}, {'value': '-0.1', 'spread': '1.07', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Body Rocking/Expressionless Face Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '4.5', 'spread': '2.59', 'groupId': 'OG000'}, {'value': '5.1', 'spread': '1.96', 'groupId': 'OG001'}, {'value': '5.4', 'spread': '2.59', 'groupId': 'OG002'}]}]}, {'title': 'CFB Body Rocking/Expressionless Face Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.6', 'spread': '2.46', 'groupId': 'OG000'}, {'value': '-0.6', 'spread': '1.13', 'groupId': 'OG001'}, {'value': '-0.1', 'spread': '1.86', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Night-time Behaviors Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.8', 'spread': '1.14', 'groupId': 'OG000'}, {'value': '0.8', 'spread': '0.97', 'groupId': 'OG001'}, {'value': '1.8', 'spread': '1.69', 'groupId': 'OG002'}]}]}, {'title': 'CFB Night-time Behaviors Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.2', 'spread': '0.97', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '1.46', 'groupId': 'OG001'}, {'value': '-0.3', 'spread': '1.11', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Anxiety/Fear Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '4.0', 'spread': '1.89', 'groupId': 'OG000'}, {'value': '4.6', 'spread': '2.46', 'groupId': 'OG001'}, {'value': '5.1', 'spread': '1.45', 'groupId': 'OG002'}]}]}, {'title': 'CFB Anxiety/Fear Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.2', 'spread': '1.92', 'groupId': 'OG000'}, {'value': '-1.3', 'spread': '2.29', 'groupId': 'OG001'}, {'value': '-1.7', 'spread': '0.95', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Walking/Standing Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2.7', 'spread': '1.64', 'groupId': 'OG000'}, {'value': '2.1', 'spread': '1.36', 'groupId': 'OG001'}, {'value': '2.9', 'spread': '1.29', 'groupId': 'OG002'}]}]}, {'title': 'CFB Walking/Standing Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.9', 'spread': '2.09', 'groupId': 'OG000'}, {'value': '0.0', 'spread': '0.58', 'groupId': 'OG001'}, {'value': '0.1', 'spread': '1.07', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': 'RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics in individuals with Rett Syndrome. The 45-item RSBQ is comprised of 8 subscales: 1) general mood (score range 0-16), 2) breathing problems (range 0-10), 3) hand behaviors (range 0-12), 4) face movements (range 0-8), 5) body rocking (BR)/expressionless face (range 0-12), 6) night-time behaviors (range 0-6), 7) anxiety/fear (range 0-8), 8) walking/standing (range 0-4). Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). Higher scores representing greater severity. CFB = Change from Baseline.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Set. Participants with non-missing data were included for analysis.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Mean 9-items Motor Behavioral Assessment (MBA-9) Total Score and Subscale Scores at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'OG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'classes': [{'title': 'Baseline Regression of Motor Skills', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2.2', 'spread': '1.23', 'groupId': 'OG000'}, {'value': '2.6', 'spread': '0.88', 'groupId': 'OG001'}, {'value': '2.6', 'spread': '0.84', 'groupId': 'OG002'}]}]}, {'title': 'CFB Regression of Motor Skills', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '1.58', 'groupId': 'OG000'}, {'value': '-0.3', 'spread': '0.49', 'groupId': 'OG001'}, {'value': '0.0', 'spread': '0.58', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Poor Eye/Social Contact', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1.7', 'spread': '0.82', 'groupId': 'OG000'}, {'value': '1.6', 'spread': '1.01', 'groupId': 'OG001'}, {'value': '1.5', 'spread': '0.97', 'groupId': 'OG002'}]}]}, {'title': 'CFB Poor Eye/Social Contact', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '0.87', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '0.69', 'groupId': 'OG001'}, {'value': '-0.9', 'spread': '1.07', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Lack of Sustained Interest', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1.8', 'spread': '0.79', 'groupId': 'OG000'}, {'value': '2.1', 'spread': '1.17', 'groupId': 'OG001'}, {'value': '1.5', 'spread': '0.71', 'groupId': 'OG002'}]}]}, {'title': 'CFB Lack of Sustained Interest', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '0.71', 'groupId': 'OG000'}, {'value': '-0.3', 'spread': '1.11', 'groupId': 'OG001'}, {'value': '-0.1', 'spread': '0.38', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Does Not Reach for Objects or People', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2.0', 'spread': '1.25', 'groupId': 'OG000'}, {'value': '1.9', 'spread': '1.05', 'groupId': 'OG001'}, {'value': '2.3', 'spread': '1.25', 'groupId': 'OG002'}]}]}, {'title': 'CFB Does Not Reach for Objects or People', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.4', 'spread': '2.24', 'groupId': 'OG000'}, {'value': '-0.4', 'spread': '0.98', 'groupId': 'OG001'}, {'value': '0.1', 'spread': '1.21', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Chewing Difficulties', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1.3', 'spread': '1.34', 'groupId': 'OG000'}, {'value': '1.3', 'spread': '0.50', 'groupId': 'OG001'}, {'value': '1.1', 'spread': '0.74', 'groupId': 'OG002'}]}]}, {'title': 'CFB Chewing Difficulties', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.2', 'spread': '0.67', 'groupId': 'OG000'}, {'value': '0.0', 'spread': '0.00', 'groupId': 'OG001'}, {'value': '0.4', 'spread': '0.53', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Speech Disturbance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3.0', 'spread': '0.47', 'groupId': 'OG000'}, {'value': '2.8', 'spread': '0.83', 'groupId': 'OG001'}, {'value': '2.7', 'spread': '0.48', 'groupId': 'OG002'}]}]}, {'title': 'CFB Speech Disturbance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '0.00', 'groupId': 'OG000'}, {'value': '0.0', 'spread': '0.58', 'groupId': 'OG001'}, {'value': '0.3', 'spread': '0.49', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Hand Clumsiness', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2.8', 'spread': '1.14', 'groupId': 'OG000'}, {'value': '2.8', 'spread': '1.56', 'groupId': 'OG001'}, {'value': '3.1', 'spread': '1.10', 'groupId': 'OG002'}]}]}, {'title': 'CFB Hand Clumsiness', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.6', 'spread': '1.33', 'groupId': 'OG000'}, {'value': '-0.4', 'spread': '1.13', 'groupId': 'OG001'}, {'value': '0.0', 'spread': '0.58', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Dysonia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1.0', 'spread': '1.41', 'groupId': 'OG000'}, {'value': '1.2', 'spread': '1.56', 'groupId': 'OG001'}, {'value': '1.0', 'spread': '1.41', 'groupId': 'OG002'}]}]}, {'title': 'CFB Dysonia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '1.42', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '0.90', 'groupId': 'OG001'}, {'value': '0.1', 'spread': '1.46', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Hypertonia/Rigidity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1.4', 'spread': '1.51', 'groupId': 'OG000'}, {'value': '1.1', 'spread': '1.45', 'groupId': 'OG001'}, {'value': '1.0', 'spread': '1.41', 'groupId': 'OG002'}]}]}, {'title': 'CFB Hypertonia/Rigidity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '0.73', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '0.38', 'groupId': 'OG001'}, {'value': '0.0', 'spread': '1.00', 'groupId': 'OG002'}]}]}, {'title': 'Baseline MBA-9 Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '17.2', 'spread': '5.81', 'groupId': 'OG000'}, {'value': '17.3', 'spread': '6.78', 'groupId': 'OG001'}, {'value': '16.8', 'spread': '5.96', 'groupId': 'OG002'}]}]}, {'title': 'CFB MBA-9 Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '4.87', 'groupId': 'OG000'}, {'value': '-1.9', 'spread': '4.06', 'groupId': 'OG001'}, {'value': '0.0', 'spread': '3.92', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': 'MBA-9 is derived from the full MBA scale (37 Rett syndrome symptoms) by selecting the items deemed amenable to change and which reflected areas of meaningful clinical change. the MBA-9 includes 9 items (1-Regression of motor skills; 2-Poor eye/social contact; 3-Lack of sustained interest; 4-Does not reach for objects or people; 5-Chewing difficulties; 6-Speech disturbance; 7-Hand clumsiness; 8-Dystonia and 9-Hypertonia/rigidity); for each item, the severity of current symptoms is rated by the investigator on a 5-point numerical scale ranging from 0 to 4 with higher scores representing greater severity (0 = normal or never; 1 = mild or rare; 2 = moderate or occasional; 3 = marked or frequent; 4 = very severe or constant). Total MBA-9 score was calculated by summing the scores of 9 different subscale items. The total summed score ranges from 0 to 36, with higher scores representing greater severity. CFB = Change from Baseline.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Set. Participants with non-missing data were included for analysis.'}, {'type': 'SECONDARY', 'title': "Change From Baseline in Mean Children's Sleep Habits Questionnaire (CSHQ) Total Score and Subscale Scores at Week 24", 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'OG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'OG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'classes': [{'title': 'Baseline Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '49.8', 'spread': '9.75', 'groupId': 'OG000'}, {'value': '46.7', 'spread': '4.90', 'groupId': 'OG001'}, {'value': '49.4', 'spread': '5.42', 'groupId': 'OG002'}]}]}, {'title': 'CFB Total score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-1.6', 'spread': '6.17', 'groupId': 'OG000'}, {'value': '-5.0', 'spread': '6.14', 'groupId': 'OG001'}, {'value': '-3.7', 'spread': '4.07', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Bedtime resistance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '9.2', 'spread': '3.43', 'groupId': 'OG000'}, {'value': '7.3', 'spread': '2.12', 'groupId': 'OG001'}, {'value': '7.0', 'spread': '1.25', 'groupId': 'OG002'}]}]}, {'title': 'CFB Bedtime resistance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.0', 'spread': '1.00', 'groupId': 'OG000'}, {'value': '-0.4', 'spread': '1.27', 'groupId': 'OG001'}, {'value': '-0.1', 'spread': '1.68', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Sleep onset delay', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1.5', 'spread': '0.71', 'groupId': 'OG000'}, {'value': '1.4', 'spread': '0.53', 'groupId': 'OG001'}, {'value': '1.8', 'spread': '0.79', 'groupId': 'OG002'}]}]}, {'title': 'CFB Sleep onset delay', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.1', 'spread': '1.27', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '0.69', 'groupId': 'OG001'}, {'value': '-0.6', 'spread': '0.53', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Sleep duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '4.7', 'spread': '2.41', 'groupId': 'OG000'}, {'value': '3.4', 'spread': '0.73', 'groupId': 'OG001'}, {'value': '4.5', 'spread': '1.58', 'groupId': 'OG002'}]}]}, {'title': 'CFB Sleep duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0.4', 'spread': '1.13', 'groupId': 'OG000'}, {'value': '-0.3', 'spread': '0.95', 'groupId': 'OG001'}, {'value': '-0.4', 'spread': '1.72', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Sleep anxiety', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '5.6', 'spread': '1.65', 'groupId': 'OG000'}, {'value': '4.6', 'spread': '0.73', 'groupId': 'OG001'}, {'value': '4.9', 'spread': '1.10', 'groupId': 'OG002'}]}]}, {'title': 'CFB Sleep anxiety', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.1', 'spread': '0.78', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '0.38', 'groupId': 'OG001'}, {'value': '0.0', 'spread': '1.15', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Night wakings', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '5.3', 'spread': '2.36', 'groupId': 'OG000'}, {'value': '4.4', 'spread': '1.33', 'groupId': 'OG001'}, {'value': '4.6', 'spread': '1.43', 'groupId': 'OG002'}]}]}, {'title': 'CFB Night wakings', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.4', 'spread': '0.88', 'groupId': 'OG000'}, {'value': '-0.6', 'spread': '1.40', 'groupId': 'OG001'}, {'value': '0.1', 'spread': '0.69', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Parasomnias', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '11.0', 'spread': '1.49', 'groupId': 'OG000'}, {'value': '10.4', 'spread': '1.42', 'groupId': 'OG001'}, {'value': '11.7', 'spread': '2.63', 'groupId': 'OG002'}]}]}, {'title': 'CFB Parasomnias', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.6', 'spread': '1.51', 'groupId': 'OG000'}, {'value': '-1.1', 'spread': '1.68', 'groupId': 'OG001'}, {'value': '-1.4', 'spread': '2.30', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Sleep disordered breathing', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '4.2', 'spread': '1.69', 'groupId': 'OG000'}, {'value': '4.0', 'spread': '1.22', 'groupId': 'OG001'}, {'value': '3.7', 'spread': '1.06', 'groupId': 'OG002'}]}]}, {'title': 'CFB Sleep disordered breathing', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.4', 'spread': '1.74', 'groupId': 'OG000'}, {'value': '-0.9', 'spread': '1.07', 'groupId': 'OG001'}, {'value': '-0.1', 'spread': '0.38', 'groupId': 'OG002'}]}]}, {'title': 'Baseline Daytime sleepiness', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '10', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '11.6', 'spread': '3.20', 'groupId': 'OG000'}, {'value': '13.2', 'spread': '2.39', 'groupId': 'OG001'}, {'value': '13.7', 'spread': '2.50', 'groupId': 'OG002'}]}]}, {'title': 'CFB Daytime sleepiness', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '-0.7', 'spread': '3.64', 'groupId': 'OG000'}, {'value': '-1.4', 'spread': '2.07', 'groupId': 'OG001'}, {'value': '-1.3', 'spread': '2.56', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': 'CSHQ is a caregiver-completed sleep screening instrument designed for school-aged children; which includes 33 items within 8 subscales: 1) bedtime resistance (score range 6-18), 2) sleep onset delay (range 1-3), 3) sleep duration (range 3-9), 4) sleep anxiety (range 4-12), 5) night wakings (range 3-9), 6) parasomnias (range 7-21), 7) sleep-disordered breathing (range 3-9), 8) daytime sleepiness (range 8-24). Item scores range from 1 to 3, where 3="usually" (≥5 times/week), 2="sometimes" (2-4 times/week), and 1="rarely" (≤1 time/week); for items 31 and 32; 3=fall asleep, 2=very sleepy, 1=not sleepy. In general, a score of 3 indicates greater severity, however, 6 items (1-Goes to bed at same time; 2-Falls asleep in 20 minutes; 3-Falls asleep in own bed; 10-Sleeps the right amount; 11-Sleeps same amount each day; 26-Wakes by himself) are reverse scored. Total summed score ranges from 33 to 99, with higher scores representing more disturbed sleep behavior. CFB = Change from Baseline.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Set. Participants with non-missing data were included for analysis.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'FG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'FG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '9'}, {'groupId': 'FG002', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}, {'groupId': 'FG001', 'numSubjects': '6'}, {'groupId': 'FG002', 'numSubjects': '4'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '6'}]}], 'dropWithdraws': [{'type': 'Withdrawal of Consent Due To Covid-19', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Withdrawal (Covid-19)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '3'}]}, {'type': 'Sponsor Decision - Covid-19 Precaution', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'A total of 41 participants were screened for eligibility; 29 were randomized to the study treatments, and 12 were screen failures.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}, {'value': '29', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': '5 mg/kg/Day GWP42003-P', 'description': 'Participants received 5 milligrams (mg)/kilogram (kg)/day GWP42003-P, administered as 100 mg/milliliter (mL) oral solution twice daily (BID).'}, {'id': 'BG001', 'title': '15 mg/kg/Day GWP42003-P', 'description': 'Participants received 15 mg/kg/day GWP42003-P, administered as 100 mg/mL oral solution BID.'}, {'id': 'BG002', 'title': 'Placebo', 'description': 'Participants received placebo oral solution matched to 5 or 15 mg/kg/day GWP42003-P, BID.'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}, {'value': '29', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '9.7', 'spread': '6.02', 'groupId': 'BG000'}, {'value': '10.7', 'spread': '3.64', 'groupId': 'BG001'}, {'value': '8.6', 'spread': '5.53', 'groupId': 'BG002'}, {'value': '9.7', 'spread': '5.11', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}, {'value': '29', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}, {'value': '28', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'One participant assigned to the 5 mg/kg/Day placebo group was misdosed with 5 mg/kg/Day GWP42003-P. The participant was included in the 5 mg/kg/Day placebo group in the Intent-to-Treat Set but was included in the 5 mg/kg/day GWP42003-P group in the Safety Analysis Set (for which Baseline data are reported).'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-08-07', 'size': 2568106, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-09-28T12:15', 'hasProtocol': True}, {'date': '2021-03-31', 'size': 733267, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2021-09-28T12:15', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}}, 'statusModule': {'whyStopped': 'The study was terminated due to enrollment challenges and the COVID-19 pandemic.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-07-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-08', 'completionDateStruct': {'date': '2021-01-21', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-08-31', 'studyFirstSubmitDate': '2019-02-19', 'resultsFirstSubmitDate': '2021-10-14', 'studyFirstSubmitQcDate': '2019-02-19', 'lastUpdatePostDateStruct': {'date': '2022-09-02', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-12-01', 'studyFirstPostDateStruct': {'date': '2019-02-21', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2021-12-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-01-21', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in the Mean Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at Week 24 for the 15 mg/kg/Day GWP42003-P Dose Level Compared With Placebo', 'timeFrame': 'Baseline; Week 24', 'description': 'RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). The total summed score ranges from 0 to 90, with higher scores representing greater severity.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in the Mean RSBQ Total Score at Week 24 for the 5 mg/kg/Day GWP42003-P Dose Level Compared With Placebo', 'timeFrame': 'Baseline; Week 24', 'description': 'RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics (45 items) in individuals with Rett Syndrome. Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). The total summed score ranges from 0 to 90, with higher scores represent greater severity.'}, {'measure': 'Mean Clinical Global Impressions - Improvement (CGI-I) Score at Week 24', 'timeFrame': 'Baseline; Week 24', 'description': "CGI-I is a 7-point scale that requires the clinician to assess how much a participant's illness has improved or worsened relative to a Baseline state at the beginning of the intervention. This is rated as: 1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; or 7 = very much worse."}, {'measure': 'Change From Baseline in Mean Clinician Global Impressions - Severity (CGI-S) Score at Week 24', 'timeFrame': 'Baseline; Week 24', 'description': "CGI-S is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment relative to the clinician's experience with participants who had the same diagnosis. This is rated as: 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; or 7 = extremely ill."}, {'measure': 'Change From Baseline in Mean RSBQ Subscale Scores at Week 24', 'timeFrame': 'Baseline; Week 24', 'description': 'RSBQ is a caregiver-completed questionnaire that measures the frequency of current disease characteristics in individuals with Rett Syndrome. The 45-item RSBQ is comprised of 8 subscales: 1) general mood (score range 0-16), 2) breathing problems (range 0-10), 3) hand behaviors (range 0-12), 4) face movements (range 0-8), 5) body rocking (BR)/expressionless face (range 0-12), 6) night-time behaviors (range 0-6), 7) anxiety/fear (range 0-8), 8) walking/standing (range 0-4). Each item is rated on a 3-point scale (0-2); 0 indicating an item that is "not true as far as you know," 1 indicating an item is "somewhat or sometimes true," and 2 indicating an item that is "very true or often true." Item 31 ("Uses eye gaze to convey feelings, needs and wishes") is reverse scored (0 indicating "very true or often true", 1 indicating "somewhat or sometimes true," and 2 indicating "not true as far as you know"). Higher scores representing greater severity. CFB = Change from Baseline.'}, {'measure': 'Change From Baseline in Mean 9-items Motor Behavioral Assessment (MBA-9) Total Score and Subscale Scores at Week 24', 'timeFrame': 'Baseline; Week 24', 'description': 'MBA-9 is derived from the full MBA scale (37 Rett syndrome symptoms) by selecting the items deemed amenable to change and which reflected areas of meaningful clinical change. the MBA-9 includes 9 items (1-Regression of motor skills; 2-Poor eye/social contact; 3-Lack of sustained interest; 4-Does not reach for objects or people; 5-Chewing difficulties; 6-Speech disturbance; 7-Hand clumsiness; 8-Dystonia and 9-Hypertonia/rigidity); for each item, the severity of current symptoms is rated by the investigator on a 5-point numerical scale ranging from 0 to 4 with higher scores representing greater severity (0 = normal or never; 1 = mild or rare; 2 = moderate or occasional; 3 = marked or frequent; 4 = very severe or constant). Total MBA-9 score was calculated by summing the scores of 9 different subscale items. The total summed score ranges from 0 to 36, with higher scores representing greater severity. CFB = Change from Baseline.'}, {'measure': "Change From Baseline in Mean Children's Sleep Habits Questionnaire (CSHQ) Total Score and Subscale Scores at Week 24", 'timeFrame': 'Baseline; Week 24', 'description': 'CSHQ is a caregiver-completed sleep screening instrument designed for school-aged children; which includes 33 items within 8 subscales: 1) bedtime resistance (score range 6-18), 2) sleep onset delay (range 1-3), 3) sleep duration (range 3-9), 4) sleep anxiety (range 4-12), 5) night wakings (range 3-9), 6) parasomnias (range 7-21), 7) sleep-disordered breathing (range 3-9), 8) daytime sleepiness (range 8-24). Item scores range from 1 to 3, where 3="usually" (≥5 times/week), 2="sometimes" (2-4 times/week), and 1="rarely" (≤1 time/week); for items 31 and 32; 3=fall asleep, 2=very sleepy, 1=not sleepy. In general, a score of 3 indicates greater severity, however, 6 items (1-Goes to bed at same time; 2-Falls asleep in 20 minutes; 3-Falls asleep in own bed; 10-Sleeps the right amount; 11-Sleeps same amount each day; 26-Wakes by himself) are reverse scored. Total summed score ranges from 33 to 99, with higher scores representing more disturbed sleep behavior. CFB = Change from Baseline.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Cannabidiol', 'CBD', 'Epidiolex', 'GWP42003-P'], 'conditions': ['Rett Syndrome', 'RTT']}, 'descriptionModule': {'briefSummary': 'To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in participants with Rett syndrome.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Participant (if possessing adequate understanding, in the investigator's opinion) and/or their parent(s)/legal representative is willing and able to give informed consent/assent for participation in the trial.\n* Participant and their caregiver are willing and able (in the investigator's opinion) to comply with all trial requirements (including the completion of all caregiver assessments by the same caregiver throughout the trial).\n* Participant must weigh at least 10 kilograms.\n* Clinical diagnosis of Rett syndrome (typical or atypical), defined according to RettSearch Consortium criteria\n* Confirmed pathogenic genetic mutation of the MECP2 gene\n* Participant must be post-regression (≥ 6 months since last loss of hand use or verbal language or gross motor regression).\n* Participant must have a disease severity of between 10 and 36, defined according to the Clinical Severity Scale (CSS).\n* All medications or interventions (including antiepileptic drugs \\[AEDs\\] and non-pharmacological interventions - dietary supplements, probiotics, physical therapy, speech therapy, etc.) for Rett syndrome-related symptoms must have been stable for 4 weeks prior to screening and the participant/caregiver must be willing to maintain a stable regimen throughout the trial.\n* Ability to swallow the investigational medicinal product (IMP) provided as a liquid solution, or the ability for IMP to be delivered via gastrostomy (G) or nasogastric (NG) feeding tube (only G-or NG-tubes made from polyurethane or silicon are allowed)\n* Participant and/or parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.\n* Participant and/or parent(s)/legal representative is willing to allow the participant's primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial, if the primary care practitioner/consultant is different than the investigator.\n\nKey Exclusion Criteria:\n\n* Participant meets exclusion criteria for Rett syndrome diagnosis (traumatic brain injury, neurometabolic disease, or severe infection that causes neurological problems; grossly abnormal psychomotor development in the first 6 months of life).\n* Participant has clinically significant abnormal laboratory values, in the investigator's opinion.\n* Participant is taking more than 2 concurrent AEDs.\n* Any history of suicidal behavior or any suicidal ideation in the last month or at screening\n* Clinically relevant abnormalities in the electrocardiogram (ECG) measured at screening or randomization\n* Concurrent cardiovascular conditions which will, in the investigator's opinion, interfere with the ability to assess their ECGs or put the participant at risk because of participation in the trial\n* First or second degree relative with a history of significant ECG abnormalities, in the opinion of the investigator (e.g. premature cardiac arrest, sudden death)\n* Any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP (active or placebo), such as sesame oil\n* Participant has moderately impaired hepatic function at screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 3 × upper limit of normal (ULN) or total bilirubin \\> 2 × ULN.\n* Participant is of childbearing potential, unless willing to ensure that they or their partner use a highly effective method of birth control (e.g., combined \\[estrogen and progestogen containing\\] hormonal contraception associated with inhibition of ovulation \\[oral, intravaginal, or transdermal\\], progestogen-only hormonal contraception associated with inhibition of ovulation \\[oral, injectable, or implantable\\], intrauterine devices/hormone-releasing systems, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the trial and for 3 months thereafter.\n* Pregnant (positive pregnancy test) or lactating\n* Received an IMP within the 3 months prior to screening\n* Participant has been taking felbamate for less than 1 year prior to screening.\n* Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®), or cannabidiol oral solutions (including CBD-OS \\[GWP42003-P\\]) within the 3 months prior to screening and is unwilling to abstain for the duration of the trial\n* Participant has a positive delta-9-tetrahydrocannabinol (THC) test at screening.\n* Any other systemic dysfunction (e.g., gastrointestinal, renal, respiratory) or significant disease or disorder which, in the opinion of the investigator, may either put the participant at risk because of participation in the trial, may influence the result of the trial, or the participant's ability to participate in the trial\n* Any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if she took part in the trial\n* Participant has been previously randomized into this trial.\n* Participant has traveled outside the country of residence planned during the trial."}, 'identificationModule': {'nctId': 'NCT03848832', 'acronym': 'ARCH', 'briefTitle': 'Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Jazz Pharmaceuticals'}, 'officialTitle': 'A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome', 'orgStudyIdInfo': {'id': 'GWND18064'}, 'secondaryIdInfos': [{'id': '2018-003370-27', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '5 milligrams per kilogram per day (mg/kg/day) GWP42003-P', 'description': '100 milligrams per milliliter (mg/mL) GWP42003-P oral solution. Taken twice daily (morning and evening).', 'interventionNames': ['Drug: GWP42003-P']}, {'type': 'EXPERIMENTAL', 'label': '15 mg/kg/day GWP42003-P', 'description': '100 mg/mL GWP42003-P oral solution. Taken twice daily (morning and evening).', 'interventionNames': ['Drug: GWP42003-P']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo oral solution (0 mg/mL GWP42003-P) volume matched to 5 mg/kg/day or 15 mg/kg/day GWP42003-P. Taken twice daily (morning and evening).', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'GWP42003-P', 'type': 'DRUG', 'otherNames': ['Cannabidiol', 'CBD', 'Epidiolex', 'CBD-OS'], 'description': 'GWP42003-P presented as an oral solution containing cannabidiol', 'armGroupLabels': ['15 mg/kg/day GWP42003-P', '5 milligrams per kilogram per day (mg/kg/day) GWP42003-P']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Matching placebo oral solution', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35294-0021', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '92123', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55101', 'city': 'Saint Paul', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 44.94441, 'lon': -93.09327}}, {'zip': '63110-1093', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '10467', 'city': 'The Bronx', 'state': 'New York', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.84985, 'lon': -73.86641}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '29646', 'city': 'Greenwood', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 34.1954, 'lon': -82.16179}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '16147', 'city': 'Genoa', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 44.40478, 'lon': 8.94439}}, {'zip': '98124', 'city': 'Messina', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 38.19394, 'lon': 15.55256}}, {'zip': '20142', 'city': 'Milan', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'zip': '00165', 'city': 'Rome', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'zip': '08022', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '08950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '28009', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '28040', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '46026', 'city': 'Valencia', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.47391, 'lon': -0.37966}}, {'zip': 'L12 2AP', 'city': 'Liverpool', 'country': 'United Kingdom', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 53.41058, 'lon': -2.97794}}, {'zip': 'SE1 7EU', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'SE5 8BB', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Jazz Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}