Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03758404 | Gene Therapy for Achromatopsia (CNGA3) | PHASE1 | COMPLETED | 11 | — | — | Aug 12, 2019 | Jun 10, 2021 | Dec 1, 2022 | 2 | United States, United Kingdom |
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: * 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 * Severe unresponsive inflammation * Infective endophthalmitis * Ocular malignancy * Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
| Arm | Type | Description |
|---|---|---|
| Low dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single low dose AAV CNGA3 |
| Intermediate dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single intermediate dose AAV CNGA3 |
| High dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single high dose AAV CNGA3 |
| Name | Type | Description |
|---|---|---|
| adeno-associated virus vector AAV- CNGA3 | BIOLOGICAL | Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene |
Inclusion Criteria: * Are aged years or over * Have achromatopsia confirmed by a retinal specialist investigator Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease with...