Ignite Creation Date:
2025-12-24 @ 3:28 PM
Ignite Modification Date:
2026-02-20 @ 9:04 PM
Study NCT ID:
NCT01363492
Status:
COMPLETED
Last Update Posted:
2021-06-09
First Post:
2011-03-31
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety Study of Replagal® Therapy in Children With Fabry Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular 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': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000627036', 'term': 'agalsidase alfa'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTransparency@shire.com', 'phone': '+1 866 842 5335', 'title': 'Study Director', 'organization': 'Shire'}, 'certainAgreement': {'otherDetails': "Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline to week 55', 'eventGroups': [{'id': 'EG000', 'title': 'Replagal® (0.2 mg/kg)', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW', 'otherNumAtRisk': 14, 'otherNumAffected': 14, 'seriousNumAtRisk': 14, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Iron deficiency anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Sinus bradycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Ear pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Hordeolum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Retinal vascular disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Mouth ulceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 12, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Tooth malformation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 12, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Chest 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'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 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'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Avulsion fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Facial bones fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Scratch', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Tooth fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Body temperature increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Heart rate increased', 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'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Muscular weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Restlessness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Asthma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 8, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 11, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pharyngeal erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Throat irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Throat tightness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Angioedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Petechiae', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pityriasis rosea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 4, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Serious Adverse Event (SAE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Replagal (0.2 mg/kg)', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to week 55', 'unitOfMeasure': 'events', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Number of Treatment Emergent Adverse Event (TEAE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Replagal (0.2 mg/kg)', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW'}], 'classes': [{'categories': [{'measurements': [{'value': '166', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to week 55', 'unitOfMeasure': 'events', 'reportingStatus': 'POSTED'}, {'type': 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alfa) administered over 40 minutes EOW'}], 'classes': [{'categories': [{'measurements': [{'value': '-5.71', 'spread': '8.799', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 55', 'unitOfMeasure': '(nmol/mL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Urine Gb3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Replagal (0.2 mg/kg)', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW'}], 'classes': [{'categories': [{'measurements': [{'value': '-1403.25', 'spread': '3636.711', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 55', 'unitOfMeasure': '(nmol/g creatinine)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Heart Rate Variability Parameter SDNN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Replagal 0.2 mg/kg', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW'}], 'classes': [{'categories': [{'measurements': [{'value': '10.46', 'spread': '24.223', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 55', 'unitOfMeasure': 'msec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Heart Rate Variability Parameter rMSSD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Replagal 0.2 mg/kg', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW'}], 'classes': [{'categories': [{'measurements': [{'value': '1.46', 'spread': '53.502', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 55', 'unitOfMeasure': 'msec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Heart Rate Variability Parameter pNN50', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Replagal 0.2 mg/kg', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW'}], 'classes': [{'categories': [{'measurements': [{'value': '-4.13', 'spread': '20.166', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to week 55', 'unitOfMeasure': 'msec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Replagal® (0.2 mg/kg)', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes every other week (EOW)'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Replagal (0.2 mg/kg)', 'description': '0.2 mg/kg Replagal (agalsidase alfa) administered over 40 minutes EOW'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '12.16', 'spread': '2.992', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'title': '<=18 years', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Age at informed consent', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'UNITED STATES', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Heart rate variability parameter SDNN', 'classes': [{'categories': [{'measurements': [{'value': '103.46', 'spread': '32.928', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'msec', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Heart rate variability parameter rMSSD', 'classes': [{'categories': [{'measurements': [{'value': '75.92', 'spread': '45.747', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'msec', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Heart rate variability parameter pNN50', 'classes': [{'categories': [{'measurements': [{'value': '32.79', 'spread': '19.997', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'msec', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Left Ventricular Mass Index (LVMI)', 'classes': [{'categories': [{'measurements': [{'value': '35.37', 'spread': '10.129', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': '(g/m^2.7)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Midwall Fractional Shortening (MFS)', 'classes': [{'categories': [{'measurements': [{'value': '18.63', 'spread': '2.891', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': '(%)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Plasma Gb3', 'classes': [{'categories': [{'measurements': [{'value': '14.79', 'spread': '12.228', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': '(nmol/mL)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Urine Gb3', 'classes': [{'categories': [{'measurements': [{'value': '1775.08', 'spread': '3691.087', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': '(nmol/g creatinine)', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-05-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-05', 'completionDateStruct': {'date': '2013-04-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-05-24', 'studyFirstSubmitDate': '2011-03-31', 'resultsFirstSubmitDate': '2014-03-25', 'studyFirstSubmitQcDate': '2011-05-27', 'lastUpdatePostDateStruct': {'date': '2021-06-09', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2014-04-17', 'studyFirstPostDateStruct': {'date': '2011-06-01', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-05-20', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-04-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Serious Adverse Event (SAE)', 'timeFrame': 'Baseline to week 55'}, {'measure': 'Number of Treatment Emergent Adverse Event (TEAE)', 'timeFrame': 'Baseline to week 55'}, {'measure': 'Development of IgG Anti-Agalsidase Alfa Antibody', 'timeFrame': 'Baseline to Week 55', 'description': 'Reflects development of Anti-Agalsidase antibodies post baseline'}, {'measure': 'Change From Baseline in Heart Rate Variability Parameter SDNN', 'timeFrame': 'Baseline to week 55'}, {'measure': 'Change From Baseline in Heart Rate Variability Parameter rMSSD', 'timeFrame': 'Baseline to week 55'}, {'measure': 'Change From Baseline in Heart Rate Variability Parameter pNN50', 'timeFrame': 'Baseline to week 55'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in LVMI', 'timeFrame': 'Baseline to week 55'}, {'measure': 'Change From Baseline in MFS', 'timeFrame': 'Baseline to week 55'}, {'measure': 'Change From Baseline in Plasma Gb3', 'timeFrame': 'Baseline to week 55'}, {'measure': 'Change From Baseline in Urine Gb3', 'timeFrame': 'Baseline to week 55'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['agalsidase alfa', 'Replagal', 'Enzyme Replacement Therapy'], 'conditions': ['Fabry Disease']}, 'referencesModule': {'references': [{'pmid': '27307708', 'type': 'DERIVED', 'citation': 'Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A. An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naive to enzyme replacement therapy. Drug Des Devel Ther. 2016 May 25;10:1771-81. doi: 10.2147/DDDT.S102761. eCollection 2016.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).', 'detailedDescription': 'In 2008, a change in the agalsidase alfa drug substance manufacturing process was made. There are no changes to the drug product formulation, manufacturing site, manufacturing process, or container closure.\n\nAn agalsidase alfa bioreactor manufacturing process (agalAF1) utilizing animal component-free media replaced the previous roller bottle (RB) process.\n\nThis study will evaluate the safety of Replagal AF, manufactured using the new bioreactor process at a dose of 0.2 mg/kg infused IV over 40 minutes, every other week (EOW) in children with Fabry disease who are 7 years to less than 18 years of age and who are naive to ERT.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '7 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\nPatients must meet all of the following criteria to be enrolled in this study.\n\n1. All patients must be diagnosed with Fabry disease by the following criteria:\n\n * Male Patients: The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of alfa-galactosidase A activity measured in serum, leukocytes, or fibroblasts or has a confirmed mutation of the alfa-galactosidase-A gene.\n * Female Patients: The patient is a heterozygous female with Fabry disease as confirmed by a mutation of the alfa-galactosidase A gene.\n\n Note: If the diagnosis of Fabry disease is previously documented in the patient's medical record, screening tests do not need to be repeated.\n2. The patient is 7 to \\<18 years of age\n3. The patient is ERT-naïve\n4. Adequate general health (as determined by the Investigators) to undergo the specified phlebotomy regimen and protocol-related procedures and no safety or medical contraindications for participation\n5. The minor child must assent to participate in the protocol and the parent(s) or legally authorized representative(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed with the child and the child's parent(s) or legally authorized representative(s)\n\nExclusion Criteria:\n\nPatients who meet any of the following criteria will be excluded from the study.\n\n1. Patient and/or the patient's parent(s) or legally authorized representative(s) are unable to understand the nature, scope, and possible consequences of the study\n2. Patient is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator or the medical monitor.\n3. Otherwise unsuitable for the study, in the opinion of the Investigator."}, 'identificationModule': {'nctId': 'NCT01363492', 'briefTitle': 'Safety Study of Replagal® Therapy in Children With Fabry Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy', 'orgStudyIdInfo': {'id': 'HGT-REP-084'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Replagal 0.2 mg/kg every other week (EOW)', 'interventionNames': ['Biological: Replagal (agalsidase alfa)']}], 'interventions': [{'name': 'Replagal (agalsidase alfa)', 'type': 'BIOLOGICAL', 'otherNames': ['agalsidase alfa'], 'description': '0.2 mg/kg administered over 40 minutes every other week (EOW)', 'armGroupLabels': ['Replagal 0.2 mg/kg every other week (EOW)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory Division of Medical Genetics', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '75246', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Baylor University Medical Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Hospital', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '22152', 'city': 'Fairfax', 'state': 'Virginia', 'country': 'United States', 'facility': 'O & O Alpan LLC', 'geoPoint': {'lat': 38.84622, 'lon': -77.30637}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shire', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}