Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation pha
| Condition(s) | X-Linked Retinitis Pigmentosa (XLRP) |
|---|---|
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Study type | Interventional |
| Summary | The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase). |
| Who can participate | Inclusion Criteria: * Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form; * Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF; * Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.; Exclusion Criteria: * Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product. |
| Ages | 8 Years to 45 Years |
| Sex | Male |
| Lead sponsor | Frontera Therapeutics |
| Locations | Beijing, Beijing Municipality, China |
| Start date | 2024-04-01 |
| NCT ID | NCT06492850 |
| Official listing | https://clinicaltrials.gov/study/NCT06492850 |