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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03252847 | Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) | PHASE1 | COMPLETED | 49 | — | — | Jul 31, 2017 | Nov 18, 2021 | Jan 8, 2025 | 5 | United States, United Kingdom |
The primary outcome is defined as any of the below occurring during the 9 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Product (ATIMP), not surgery alone: * 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 |
|---|---|---|
| Phase 1 (Part 1, Dose Escalation) | EXPERIMENTAL | Participants receive one of three doses of AAV5-RPGR |
| Phase 2 (Part 2; Expansion) | EXPERIMENTAL | Participants receive one of two doses of AAV5-RPGR |
| Name | Type | Description |
|---|---|---|
| AAV5-RPGR | GENETIC | Single, subretinal administration of AAV5-RPGR |
Key inclusion Criteria: * Males aged 5 years or older * Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI) Key exclusion Criteria: • Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months