Ignite Creation Date:
2025-12-24 @ 9:15 PM
Ignite Modification Date:
2026-01-20 @ 7:45 AM
Study NCT ID:
NCT03758404
Status:
COMPLETED
Last Update Posted:
2022-12-01
First Post:
2018-11-27
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Gene Therapy for Achromatopsia (CNGA3)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2022-02-04', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D003117', 'term': 'Color Vision Defects'}], 'ancestors': [{'id': 'D014786', 'term': 'Vision Disorders'}, {'id': 'D012678', 'term': 'Sensation Disorders'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000077765', 'term': 'Cone Dystrophy'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'CNGA3@meiragtx.com', 'phone': '020 3866 4320', 'title': 'Program Manager', 'organization': 'MeiraGTx'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '6 months', 'eventGroups': [{'id': 'EG000', 'title': 'Low Dose AAV - CNGA3', 'description': 'Subretinal administration of a single low dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Intermediate Dose AAV - CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'High Dose AAV - CNGA3', 'description': 'Subretinal administration of a single high dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 5, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Conjunctival haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 5}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Visual acuity reduced', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Conjunctival hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Lenticular opacities', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Ocular discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 4}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eye inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eye pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eye pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Subconjunctival cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Chorioretinal folds', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Foreign body sensation in eyes', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Iridocyclitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Photophobia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Punctate keratitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Retinal disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Uveitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vitreous floaters', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Intraocular pressure increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood glucose increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Intraocular pressure decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Slit-lamp tests abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypophosphataemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hyperlipidaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Type 2 diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Ear pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': "Tenon's cyst", 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Syncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 5, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '5', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Low Dose AAV - CNGA3', 'description': 'Subretinal administration of a single low dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}, {'id': 'OG001', 'title': 'Intermediate Dose AAV - CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}, {'id': 'OG002', 'title': 'High Dose AAV - CNGA3', 'description': 'Subretinal administration of a single high dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 Weeks', 'description': 'The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone:\n\n* Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences\n* Severe unresponsive inflammation\n* Infective endophthalmitis\n* Ocular malignancy\n* Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants.'}, {'type': 'SECONDARY', 'title': 'Improvements in Visual Function as Assessed by Visual Acuity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '5', 'groupId': 'OG002'}, {'value': '11', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Low Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single low dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG001', 'title': 'Intermediate Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG002', 'title': 'High Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG003', 'title': 'Overall', 'description': 'Subretinal administration of a single low, intermediate, or high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}], 'classes': [{'categories': [{'measurements': [{'value': '0.22', 'groupId': 'OG000', 'lowerLimit': '-0.7', 'upperLimit': '1.3'}, {'value': '2.44', 'groupId': 'OG001', 'lowerLimit': '0.0', 'upperLimit': '4.7'}, {'value': '2.40', 'groupId': 'OG002', 'lowerLimit': '-1.0', 'upperLimit': '5.0'}, {'value': '1.82', 'groupId': 'OG003', 'lowerLimit': '-1.0', 'upperLimit': '5.0'}]}]}], 'paramType': 'MEAN', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.', 'unitOfMeasure': 'number of ETDRS letters', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'All of the 11 participants performed the visual acuity assessment at baseline and Week 24.'}, {'type': 'SECONDARY', 'title': 'Improvements in Retinal Function as Assessed by Static Perimetry', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '4', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Low Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single low dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG001', 'title': 'Intermediate Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG002', 'title': 'High Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG003', 'title': 'Overall', 'description': 'Subretinal administration of a single low, intermediate, or high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}], 'classes': [{'categories': [{'measurements': [{'value': '1.17', 'groupId': 'OG000'}, {'value': '-1.83', 'groupId': 'OG001'}, {'value': '0.15', 'groupId': 'OG002', 'lowerLimit': '-0.3', 'upperLimit': '0.9'}, {'value': '-0.01', 'groupId': 'OG003', 'lowerLimit': '-1.8', 'upperLimit': '1.2'}]}]}], 'paramType': 'MEAN', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.', 'unitOfMeasure': 'decibel', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Six of the 11 participants had mean retinal sensitivity data available at both baseline and Week 24 in the treated eye.'}, {'type': 'SECONDARY', 'title': 'Quality of Life Measured by QoL Questionnaires in Children and Adolescents', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Low Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single low dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG001', 'title': 'Intermediate Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG002', 'title': 'High Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG003', 'title': 'Overall', 'description': 'Subretinal administration of a single low, intermediate, or high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}], 'classes': [{'categories': [{'measurements': [{'value': '-15', 'groupId': 'OG000'}, {'value': '18.3', 'groupId': 'OG001', 'lowerLimit': '-1', 'upperLimit': '50'}, {'value': '8.0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '16'}, {'value': '9.3', 'groupId': 'OG003', 'lowerLimit': '-15', 'upperLimit': '50'}]}]}], 'paramType': 'MEAN', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'EQ-VAS data were available for 6 of the 9 children/adolescents enrolled in the study.'}, {'type': 'SECONDARY', 'title': 'Quality of Life Measured by QoL Questionnaires in Adults', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}, {'value': '2', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Low Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single low dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG001', 'title': 'Intermediate Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG002', 'title': 'High Dose Adeno-associated Virus (AAV) CNGA3', 'description': 'Subretinal administration of a single high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}, {'id': 'OG003', 'title': 'Overall', 'description': 'Subretinal administration of a single low, intermediate, or high dose AAV CNGA3\n\nadeno-associated virus vector AAV- CNGA3: Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene'}], 'classes': [{'categories': [{'measurements': [{'value': '1.5', 'groupId': 'OG002', 'lowerLimit': '-7', 'upperLimit': '10'}, {'value': '1.5', 'groupId': 'OG003', 'lowerLimit': '-7', 'upperLimit': '10'}]}]}], 'paramType': 'MEAN', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'EQ-VAS data were available for both adults enrolled in the study.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Low Dose AAV - CNGA3', 'description': 'Subretinal administration of a single low dose adeno-associated virus AAV-CNGA3\n\nAAV-CNGA3: AAV gene therapy for defects in CNGA3 gene'}, {'id': 'FG001', 'title': 'Intermediate Dose AAV - CNGA3', 'description': 'Subretinal administration of a single intermediate dose adeno-associated virus AAV-CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}, {'id': 'FG002', 'title': 'High Dose AAV - CNGA3', 'description': 'Subretinal administration of a single high dose adeno-associated virus AAV-CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '5'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '5'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Participants were recruited from two medical centers between 12 August 2019 (date first participant signed informed) and 19 November 2020 (date last participant signed informed consent). A total of 11 participants were enrolled in the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}, {'value': '11', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'Low Dose AAV - CNGA3', 'description': 'Subretinal administration of a single low dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}, {'id': 'BG001', 'title': 'Intermediate Dose AAV - CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}, {'id': 'BG002', 'title': 'High Dose AAV - CNGA3', 'description': 'Subretinal administration of a single high dose AAV - CNGA3\n\nAAV- CNGA3: AAV gene therapy for defects in CNGA3 gene'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'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': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '5', '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': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '6', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-04-16', 'size': 2248291, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-12-23T03:56', 'hasProtocol': True}, {'date': '2021-09-02', 'size': 414356, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2021-12-23T04:07', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 11}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-08-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-11', 'completionDateStruct': {'date': '2021-06-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-11-03', 'studyFirstSubmitDate': '2018-11-27', 'resultsFirstSubmitDate': '2022-01-06', 'studyFirstSubmitQcDate': '2018-11-28', 'lastUpdatePostDateStruct': {'date': '2022-12-01', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2022-02-10', 'studyFirstPostDateStruct': {'date': '2018-11-29', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2022-02-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-06-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone.', 'timeFrame': '6 Weeks', 'description': 'The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone:\n\n* Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences\n* Severe unresponsive inflammation\n* Infective endophthalmitis\n* Ocular malignancy\n* Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)'}], 'secondaryOutcomes': [{'measure': 'Improvements in Visual Function as Assessed by Visual Acuity', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.'}, {'measure': 'Improvements in Retinal Function as Assessed by Static Perimetry', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.'}, {'measure': 'Quality of Life Measured by QoL Questionnaires in Children and Adolescents', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.'}, {'measure': 'Quality of Life Measured by QoL Questionnaires in Adults', 'timeFrame': '6 Months', 'description': 'Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Achromatopsia']}, 'descriptionModule': {'briefSummary': 'A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia', 'detailedDescription': 'CNGA3 retinal gene therapy for patients with achromatopsia'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Are aged years or over\n* Have achromatopsia confirmed by a retinal specialist investigator\n\nExclusion Criteria:\n\n* Are females who are pregnant or breastfeeding\n* Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months\n* Have any other condition that the investigator considers makes them inappropriate for entry into the trial'}, 'identificationModule': {'nctId': 'NCT03758404', 'acronym': 'CNGA3', 'briefTitle': 'Gene Therapy for Achromatopsia (CNGA3)', 'organization': {'class': 'INDUSTRY', 'fullName': 'MeiraGTx UK II Ltd'}, 'officialTitle': 'An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3', 'orgStudyIdInfo': {'id': 'MGT012'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Low dose adeno-associated virus (AAV) CNGA3', 'description': 'Subretinal administration of a single low dose AAV CNGA3', 'interventionNames': ['Biological: adeno-associated virus vector AAV- CNGA3']}, {'type': 'EXPERIMENTAL', 'label': 'Intermediate dose adeno-associated virus (AAV) CNGA3', 'description': 'Subretinal administration of a single intermediate dose AAV CNGA3', 'interventionNames': ['Biological: adeno-associated virus vector AAV- CNGA3']}, {'type': 'EXPERIMENTAL', 'label': 'High dose adeno-associated virus (AAV) CNGA3', 'description': 'Subretinal administration of a single high dose AAV CNGA3', 'interventionNames': ['Biological: adeno-associated virus vector AAV- CNGA3']}], 'interventions': [{'name': 'adeno-associated virus vector AAV- CNGA3', 'type': 'BIOLOGICAL', 'description': 'Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene', 'armGroupLabels': ['High dose adeno-associated virus (AAV) CNGA3', 'Intermediate dose adeno-associated virus (AAV) CNGA3', 'Low dose adeno-associated virus (AAV) CNGA3']}]}, 'contactsLocationsModule': {'locations': [{'zip': '48105', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'Kellogg Eye Center', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': 'EC1V 2PD', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Moorfields Eye Hospital NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'James Bainbridge', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Chief Investigator'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'MeiraGTx UK II Ltd', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Janssen Research & Development, LLC', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}