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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02781480 | Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) | PHASE1 | COMPLETED | 15 | — | — | Apr 1, 2016 | Dec 1, 2018 | Jul 12, 2021 | 2 | United States, United Kingdom |
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more. * Severe unresponsive inflammation. * Infective endophthalmitis. * Ocular malignancy. * Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
| Arm | Type | Description |
|---|---|---|
| Low dose AAV-RPE65 | EXPERIMENTAL | Subretinal administration of a single low dose of range AAV-RPE65 |
| Intermediate dose AAV-RPE65 | EXPERIMENTAL | Subretinal administration of a single intermediate dose of range AAV-RPE65 |
| High dose AAV-RPE65 | EXPERIMENTAL | Subretinal administration of a single high dose of range AAV-RPE65 |
| Name | Type | Description |
|---|---|---|
| AAV RPE65 | BIOLOGICAL | Comparison of different dosages of AAV RPE65 |
Key Inclusion Criteria: * Aged 3 years or older * Early-onset severe retinal dystrophy consistent with RPE65 deficiency Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Have participated in another research study involving an investigational therapy for ocular disease within ...