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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03001310 | Gene Therapy for Achromatopsia (CNGB3) | PHASE1 | COMPLETED | 23 | — | — | Jan 16, 2017 | Oct 25, 2019 | Mar 8, 2023 | 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 |
|---|---|---|
| Biological-Low dose AAV - CNGB3 | EXPERIMENTAL | Subretinal administration of a single low dose of AAV - CNGB3 |
| Biological-Medium dose AAV - CNGB3 | EXPERIMENTAL | Subretinal administration of a single intermediate dose of AAV - CNGB3 |
| Biological-High dose AAV - CNGB3 | EXPERIMENTAL | Subretinal administration of a single high dose of AAV - CNGB3 |
| Name | Type | Description |
|---|---|---|
| AAV - CNGB3 | BIOLOGICAL | Comparison of different dosages of AAV-CNGB3 |
Inclusion Criteria: * Are aged 3 years or older * Have achromatopsia confirmed by a retinal specialist (CI or PI) Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease wit...