Ignite Creation Date:
2025-12-25 @ 2:34 AM
Ignite Modification Date:
2026-02-20 @ 6:05 PM
Study NCT ID:
NCT03779334
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-11-26
First Post:
2018-12-17
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Canada', 'China', 'Italy', 'Saudi Arabia']}, 'conditionBrowseModule': {'meshes': [{'id': 'D009134', 'term': 'Muscular Atrophy, Spinal'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000629884', 'term': 'Risdiplam'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'genentech@druginfo.com', 'phone': '800 821-8590', 'title': 'Medical Communications', 'organization': 'Hoffmann-La Roche'}, 'certainAgreement': {'otherDetails': "The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From baseline up to the clinical cut off date (a minimum of 12 months and a maximum of 3.5 years)', 'description': 'The safety population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not.', 'eventGroups': [{'id': 'EG000', 'title': '2 SMN2 Copies, Risdiplam', 'description': 'Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 8, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 3}, {'id': 'EG001', 'title': '3 SMN2 Copies, Risdiplam', 'description': 'Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.', 'otherNumAtRisk': 13, 'deathsNumAtRisk': 13, 'otherNumAffected': 12, 'seriousNumAtRisk': 13, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': '>/=4 SMN2 Copies, Risdiplam', 'description': 'Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 4, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Cryptorchism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Cystoid macular oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Retinal pigmentation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Retinal vascular disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Gastrointestinal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Regurgitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Teething', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 7, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 7, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 5, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Bronchiolitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Croup infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Cytomegalovirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Exanthema subitum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Gastroenteritis norovirus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Gastrointestinal viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Hand-foot-and-mouth disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Impetigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Otitis media acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Respiratory syncytial virus bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Respiratory syncytial virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Respiratory tract infection bacterial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 4, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 5, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Skin infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections 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'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Varicella', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Viral rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Vulvovaginitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Accidental overdose', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Expired product 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'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Intercepted medication error', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Limb injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Overdose', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Underdose', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Cardiac murmur', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Hyperphosphatasaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Hypoglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Iron deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Vitamin D deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Umbilical granuloma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Rhinitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Dermatitis atopic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Dermatitis contact', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Dermatitis diaper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Papule', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Rash maculo-papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Skin discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}], 'seriousEvents': [{'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Femur fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Soft tissue injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Jaundice neonatal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': '2 SMN2 Copies, Risdiplam', 'description': 'Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}], 'classes': [{'categories': [{'measurements': [{'value': '80.0', 'groupId': 'OG000', 'lowerLimit': '34.26', 'upperLimit': '98.98'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000'], 'statisticalMethod': 'Exact Binomial Test', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Performance Criterion = 5%'}], 'paramType': 'NUMBER', 'timeFrame': 'At Month 12', 'description': 'The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. An exact binomial test was performed. If the lower limit of the two-sided 90% CI was above the 5% threshold, the primary objective of the study was considered achieved.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Primary efficacy population included all infants in the intent-to-treat (ITT) population with two SMN2 copies (excluding the known SMN2 gene modifier mutation c.859G\\>C) and a baseline compound muscle action potential (CMAP) amplitude \\>/= 1.5 mV.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Developing Clinically Manifested SMA', 'timeFrame': 'At Month 12 and 24', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Time to Permanent Ventilation and/or Death', 'timeFrame': 'Up to 7 years', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Are Alive Without Permanent Ventilation', 'timeFrame': 'At Month 12 and 24', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Alive', 'timeFrame': 'At Month 12 and 24', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2)', 'timeFrame': 'At Month 12 and 24', 'description': 'HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline).', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': '2 SMN2 Copies, Risdiplam', 'description': 'Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}], 'classes': [{'categories': [{'measurements': [{'value': '87.5', 'groupId': 'OG000', 'lowerLimit': '52.93', 'upperLimit': '99.36'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At Month 12', 'description': 'Assessed in Item 22 of the BSID-III Gross Motor Scale. The BSID-III is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-to-treat (ITT) population included all enrolled participants, regardless of whether they received risdiplam or not. Only participants with two copies of the SMN2 gene were included in this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Sitting Without Support for 5 Seconds', 'timeFrame': 'At Month 24', 'description': 'Assessed with BSID-III Gross Motor Scale', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Sitting Without Support for 30 Seconds', 'timeFrame': 'At Month 12 and 24', 'description': 'Assessed with BSID-III Gross Motor Scale', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Standing for at Least 3 Seconds', 'timeFrame': 'At Month 24', 'description': 'Assessed with BSID-III Gross Motor Scale', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Walking (Takes at Least 3 Steps)', 'timeFrame': 'At Month 24', 'description': 'Assessed with BSID-III Gross Motor Scale', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Demonstrating the Ability to Achieve a Scaled Score on BSID-III Gross Motor Subtests Within 1.5 Standard Deviations of Chronological Reference Standard', 'timeFrame': 'At Month 24 and 42', 'description': 'Assessed through BSID-III Gross Motor Scale', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': "Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12", 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '5', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '2 SMN2 Copies, Risdiplam', 'description': 'Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'OG001', 'title': '3 SMN2 Copies, Risdiplam', 'description': 'Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'OG002', 'title': '>/=4 SMN2 Copies, Risdiplam', 'description': 'Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '5', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '46.50', 'groupId': 'OG000', 'lowerLimit': '35.0', 'upperLimit': '52.0'}, {'value': '55.00', 'groupId': 'OG001', 'lowerLimit': '44.0', 'upperLimit': '62.0'}, {'value': '50.00', 'groupId': 'OG002', 'lowerLimit': '44.0', 'upperLimit': '52.0'}]}]}, {'title': 'Change from Baseline at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '4', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '9.50', 'groupId': 'OG000', 'lowerLimit': '-6.0', 'upperLimit': '20.0'}, {'value': '8.00', 'groupId': 'OG001', 'lowerLimit': '2.0', 'upperLimit': '20.0'}, {'value': '16.00', 'groupId': 'OG002', 'lowerLimit': '8.0', 'upperLimit': '19.0'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline, Month 12', 'description': 'The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. A positive change from baseline indicates an improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all enrolled participants, regardless of whether they received risdiplam or not.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Achieve a Score of 40 or Higher, 50 or Higher, and 60 or Higher in the CHOP INTEND Motor Function Scale at Month 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '4', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '2 SMN2 Copies, Risdiplam', 'description': 'Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'OG001', 'title': '3 SMN2 Copies, Risdiplam', 'description': 'Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'OG002', 'title': '>/=4 SMN2 Copies, Risdiplam', 'description': 'Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}], 'classes': [{'title': 'Score >=40', 'categories': [{'measurements': [{'value': '75.0', 'groupId': 'OG000', 'lowerLimit': '40.03', 'upperLimit': '95.36'}, {'value': '100', 'groupId': 'OG001', 'lowerLimit': '79.42', 'upperLimit': '100.00'}, {'value': '100', 'groupId': 'OG002', 'lowerLimit': '47.29', 'upperLimit': '100.00'}]}]}, {'title': 'Score >=50', 'categories': [{'measurements': [{'value': '75.0', 'groupId': 'OG000', 'lowerLimit': '40.03', 'upperLimit': '95.36'}, {'value': '100', 'groupId': 'OG001', 'lowerLimit': '79.42', 'upperLimit': '100.00'}, {'value': '100', 'groupId': 'OG002', 'lowerLimit': '47.29', 'upperLimit': '100.00'}]}]}, {'title': 'Score >=60', 'categories': [{'measurements': [{'value': '37.5', 'groupId': 'OG000', 'lowerLimit': '11.11', 'upperLimit': '71.08'}, {'value': '100', 'groupId': 'OG001', 'lowerLimit': '79.42', 'upperLimit': '100.00'}, {'value': '100', 'groupId': 'OG002', 'lowerLimit': '47.29', 'upperLimit': '100.00'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At Month 12', 'description': 'The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. Data are presented with a two-sided 90% Clopper-Pearson (exact) CI for the proportion.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all enrolled participants, regardless of whether they received risdiplam or not.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point', 'timeFrame': 'Up to Month 24', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score', 'timeFrame': 'At Month 60', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height', 'timeFrame': 'At Month 12, 24, 36, 48 and 60', 'description': 'Based on the WHO Child Growth Standards (WHO 2019)', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age', 'timeFrame': 'At Month 12 and 24', 'description': 'Based on the WHO Child Growth Standards (WHO 2019)', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height', 'timeFrame': 'At Month 12, 24, 36, 48 and 60', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Percentiles for Head Circumference- For-age', 'timeFrame': 'At Month 12 and 24', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Chest Circumference', 'timeFrame': 'At Month 12 and 24', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Ratio Between Chest and Head Circumferences', 'timeFrame': 'At Month 12 and 24', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With the Ability to Swallow and to Feed Orally', 'timeFrame': 'At Month 12, 24, 36, 48 and 60', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude', 'timeFrame': 'At Month 12 and 24', 'description': 'Measured by CMAP', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Measurement of Pharmacodynamic Marker Levels in Blood', 'timeFrame': 'Day 1, 56, 196, 364, 728 and at early withdrawal', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Adverse Events', 'timeFrame': 'Up to 7 years', 'description': 'Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Ophthalmological Examination as Appropriate for Age', 'timeFrame': 'Up to 7 years', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Plasma Concentration of Risdiplam and Its Metabolites to Characterize the PK Profile', 'timeFrame': 'Up to 7 years', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': '2 SMN2 Copies, Risdiplam', 'description': 'Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'FG001', 'title': '3 SMN2 Copies, Risdiplam', 'description': 'Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'FG002', 'title': '>/=4 SMN2 Copies, Risdiplam', 'description': 'Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '13'}, {'groupId': 'FG002', 'numSubjects': '5'}]}, {'type': 'Primary Efficacy Population', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '13'}, {'groupId': 'FG002', 'numSubjects': '5'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Ongoing in Study', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}, {'groupId': 'FG001', 'numSubjects': '13'}, {'groupId': 'FG002', 'numSubjects': '5'}]}]}], 'recruitmentDetails': 'Overall, 26 infants with spinal muscular atrophy (SMA) were enrolled in the study across 7 different sites in 7 countries.', 'preAssignmentDetails': 'The study enrolled infants aged from birth to 6 weeks who were genetically diagnosed with SMA but were not yet presenting with symptoms. Study arms were based on the number of copies of the SMN2 gene.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}, {'value': '26', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': '2 SMN2 Copies, Risdiplam', 'description': 'Infants with 2 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'BG001', 'title': '3 SMN2 Copies, Risdiplam', 'description': 'Infants with 3 copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'BG002', 'title': '>/=4 SMN2 Copies, Risdiplam', 'description': 'Infants with 4 or more copies of SMN2 were enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range of close to 2000 ng\\*hr/mL.'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '22.8', 'spread': '5.0', 'groupId': 'BG000'}, {'value': '28.9', 'spread': '7.5', 'groupId': 'BG001'}, {'value': '31.2', 'spread': '6.1', 'groupId': 'BG002'}, {'value': '27.5', 'spread': '7.1', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'days', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '16', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '10', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}, {'value': '22', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '3', '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': '8', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '22', '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': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-03-30', 'size': 1667685, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-02-09T15:59', 'hasProtocol': True}, {'date': '2021-05-12', 'size': 834613, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-02-09T15:59', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open-label, single arm'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 26}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2019-08-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2027-03-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-20', 'studyFirstSubmitDate': '2018-12-17', 'resultsFirstSubmitDate': '2024-02-09', 'studyFirstSubmitQcDate': '2018-12-17', 'lastUpdatePostDateStruct': {'date': '2025-11-26', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2024-02-09', 'studyFirstPostDateStruct': {'date': '2018-12-19', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-03-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-02-20', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support', 'timeFrame': 'At Month 12', 'description': 'The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. An exact binomial test was performed. If the lower limit of the two-sided 90% CI was above the 5% threshold, the primary objective of the study was considered achieved.'}], 'secondaryOutcomes': [{'measure': 'Percentage of Participants Developing Clinically Manifested SMA', 'timeFrame': 'At Month 12 and 24'}, {'measure': 'Time to Permanent Ventilation and/or Death', 'timeFrame': 'Up to 7 years'}, {'measure': 'Percentage of Participants Who Are Alive Without Permanent Ventilation', 'timeFrame': 'At Month 12 and 24'}, {'measure': 'Percentage of Participants Alive', 'timeFrame': 'At Month 12 and 24'}, {'measure': 'Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2)', 'timeFrame': 'At Month 12 and 24', 'description': 'HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking'}, {'measure': 'Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline).', 'timeFrame': 'At Month 12', 'description': 'Assessed in Item 22 of the BSID-III Gross Motor Scale. The BSID-III is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method.'}, {'measure': 'Percentage of Participants Sitting Without Support for 5 Seconds', 'timeFrame': 'At Month 24', 'description': 'Assessed with BSID-III Gross Motor Scale'}, {'measure': 'Percentage of Participants Sitting Without Support for 30 Seconds', 'timeFrame': 'At Month 12 and 24', 'description': 'Assessed with BSID-III Gross Motor Scale'}, {'measure': 'Percentage of Participants Standing for at Least 3 Seconds', 'timeFrame': 'At Month 24', 'description': 'Assessed with BSID-III Gross Motor Scale'}, {'measure': 'Percentage of Participants Walking (Takes at Least 3 Steps)', 'timeFrame': 'At Month 24', 'description': 'Assessed with BSID-III Gross Motor Scale'}, {'measure': 'Percentage of Participants Demonstrating the Ability to Achieve a Scaled Score on BSID-III Gross Motor Subtests Within 1.5 Standard Deviations of Chronological Reference Standard', 'timeFrame': 'At Month 24 and 42', 'description': 'Assessed through BSID-III Gross Motor Scale'}, {'measure': "Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12", 'timeFrame': 'Baseline, Month 12', 'description': 'The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. A positive change from baseline indicates an improvement.'}, {'measure': 'Percentage of Participants Who Achieve a Score of 40 or Higher, 50 or Higher, and 60 or Higher in the CHOP INTEND Motor Function Scale at Month 12', 'timeFrame': 'At Month 12', 'description': 'The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. Data are presented with a two-sided 90% Clopper-Pearson (exact) CI for the proportion.'}, {'measure': 'Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point', 'timeFrame': 'Up to Month 24'}, {'measure': 'Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score', 'timeFrame': 'At Month 60'}, {'measure': 'Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height', 'timeFrame': 'At Month 12, 24, 36, 48 and 60', 'description': 'Based on the WHO Child Growth Standards (WHO 2019)'}, {'measure': 'Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age', 'timeFrame': 'At Month 12 and 24', 'description': 'Based on the WHO Child Growth Standards (WHO 2019)'}, {'measure': 'Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height', 'timeFrame': 'At Month 12, 24, 36, 48 and 60'}, {'measure': 'Change From Baseline Percentiles for Head Circumference- For-age', 'timeFrame': 'At Month 12 and 24'}, {'measure': 'Change From Baseline in Chest Circumference', 'timeFrame': 'At Month 12 and 24'}, {'measure': 'Ratio Between Chest and Head Circumferences', 'timeFrame': 'At Month 12 and 24'}, {'measure': 'Percentage of Participants With the Ability to Swallow and to Feed Orally', 'timeFrame': 'At Month 12, 24, 36, 48 and 60'}, {'measure': 'Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitude', 'timeFrame': 'At Month 12 and 24', 'description': 'Measured by CMAP'}, {'measure': 'Measurement of Pharmacodynamic Marker Levels in Blood', 'timeFrame': 'Day 1, 56, 196, 364, 728 and at early withdrawal'}, {'measure': 'Percentage of Participants With Adverse Events', 'timeFrame': 'Up to 7 years', 'description': 'Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5'}, {'measure': 'Ophthalmological Examination as Appropriate for Age', 'timeFrame': 'Up to 7 years'}, {'measure': 'Plasma Concentration of Risdiplam and Its Metabolites to Characterize the PK Profile', 'timeFrame': 'Up to 7 years'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Muscular Atrophy, Spinal']}, 'referencesModule': {'references': [{'pmid': '40802943', 'type': 'DERIVED', 'citation': 'Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Beldzinska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Risdiplam in Presymptomatic Spinal Muscular Atrophy. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120.'}]}, 'descriptionModule': {'briefSummary': 'A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).', 'detailedDescription': 'The study is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with symptoms. There will be a screening, treatment, open-label extension (OLE) and a follow-up. All participants will receive risdiplam orally once daily for 2 years followed by an OLE phase of at least 3 years and a follow-up (if applicable), for a total treatment duration of at least 5 years for each participant enrolled.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '6 Weeks', 'minimumAge': '1 Day', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Males and females aged from birth (1 day) to 6 weeks (42 days) of age at the time of first dose (Day 1); a minimum age of 7 days at first dose is required for the first infant to be enrolled\n* Gestational age of 37-42 weeks for singleton births; gestational age of 34-42 weeks for twins\n* Body weight \\>= 3rd percentile for age, using appropriate country-specific guidelines\n* Genetic diagnosis of 5q-autosomal recessive SMA, including confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the SMN1 gene\n* Absence of clinical signs or symptoms at screening (Day -42 to Day -2) or at baseline (Day -1) that are, in the opinion of the investigator, strongly suggestive of SMA\n* Receiving adequate nutrition and hydration at the time of screening, in the opinion of the investigator\n* Adequately recovered from any acute illness at baseline and considered well enough to participate in the study, in the opinion of the investigator\n* Able and expected to be able to safely travel to the study site for the entire duration of the study and in accordance to the frequency of required study visits, in the opinion of the investigator\n* Able to complete all study procedures, measurements, and visits, and the parent (or caregiver), in the opinion of the investigator, has adequately supportive psychosocial circumstances\n* Parent (or caregiver) is willing to consider nasogastric, naso-jejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator\n* Parent (or caregiver) is willing to consider the use of non-invasive ventilation during the study, if recommended by the investigator\n\nExclusion Criteria:\n\n* Concomitant or previous participation in any investigational drug or device study at any time\n* Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide, SMN2-splicing modifier, or gene therapy either in a clinical study or as part of medical care\n* Presence of significant concurrent syndromes or diseases\n* In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures\n* Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation\n* Awake hypoxemia (SaO2 \\< 95%) with or without ventilator support\n* Multiple or fixed contractures and/or hip subluxation or dislocation at birth\n* Systolic blood pressure or diastolic blood pressure or heart rate considered to be clinically significant by the investigator\n* Presence of clinically relevant ECG abnormalities before study drug administration; corrected QT interval using Bazett's method \\> 460 ms; personal or family history (first degree relatives) of congenital long QT syndrome indicating a safety risk for patients as determined by the investigator. First-degree atrioventricular block or isolated right bundle branch block are allowed\n* The infant (and the mother, if breastfeeding the infant) taking any inhibitor of CYP3A4 taken within 2 weeks, any inducer of CYP3A4 taken within 4 weeks, any OCT 2 and MATE substrates within 2 weeks and known FMO1 or FMO3 inhibitors or substrates\n* Clinically significant abnormalities in laboratory test results\n* Ascertained or presumptive hypersensitivity to risdiplam or to the constituents of its formulation\n* Treatment with oral salbutamol or another beta-2 adrenergic agonist taken orally for SMA is not allowed. Use of inhaled beta-2 adrenergic agonists is allowed\n* Infants exposed to drugs with known retinal toxicity given to mothers during pregnancy (and lactation) should not be enrolled. Anticipated need for drugs known to cause retinal toxicity during the study.\n* Diagnosis of ophthalmic diseases"}, 'identificationModule': {'nctId': 'NCT03779334', 'acronym': 'Rainbowfish', 'briefTitle': 'A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Hoffmann-La Roche'}, 'officialTitle': 'An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy', 'orgStudyIdInfo': {'id': 'BN40703'}, 'secondaryIdInfos': [{'id': '2018-002087-12', 'type': 'EUDRACT_NUMBER'}, {'id': '2023-506009-20-00', 'type': 'CTIS'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open-label Risdiplam', 'description': 'Participants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range.', 'interventionNames': ['Drug: Risdiplam']}], 'interventions': [{'name': 'Risdiplam', 'type': 'DRUG', 'otherNames': ['Evrysdi'], 'description': 'Risdiplam will be administered orally.', 'armGroupLabels': ['Open-label Risdiplam']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32837', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': "Nemours Children's Hospital", 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'zip': '2031', 'city': 'Randwick', 'state': 'New South Wales', 'country': 'Australia', 'facility': "Sydney Children's Hospital", 'geoPoint': {'lat': -33.91439, 'lon': 151.24895}}, {'zip': '4000', 'city': 'Liège', 'country': 'Belgium', 'facility': 'Chr de La Citadelle', 'geoPoint': {'lat': 50.63373, 'lon': 5.56749}}, {'zip': '05403-000', 'city': 'São Paulo', 'state': 'São Paulo', 'country': 'Brazil', 'facility': 'Hospital das Clinicas - FMUSP_X', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'zip': '80-952', 'city': 'Gda?sk', 'country': 'Poland', 'facility': 'Szpital Gdanskiego Uniwersytetu Medycznego'}, {'zip': '125412', 'city': 'Moscow', 'state': 'Moscow Oblast', 'country': 'Russia', 'facility': 'Russian Children Neuromuscular Center of Veltischev'}, {'zip': '807', 'city': 'Kaohsiung City', 'country': 'Taiwan', 'facility': 'Kaohsiung Medical University Chung-Ho Hospital', 'geoPoint': {'lat': 22.61626, 'lon': 120.31333}}], 'overallOfficials': [{'name': 'Clinical Trials', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Hoffmann-La Roche'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': "For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\\_sharing"}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hoffmann-La Roche', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}