NCT06492850
Recruiting
Phase 1
A Phase I/II Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of FT-002 Subretinal Injection in Subjects With RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa.
ConditionsX-Linked Retinitis Pigmentosa (XLRP)
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- X-Linked Retinitis Pigmentosa (XLRP)
- Sponsor
- Frontera Therapeutics
- Enrollment
- 32
- Locations
- 1
- Primary Endpoint
- To evaluate the safety and tolerance of FT-002
- Status
- Recruiting
- Last Updated
- last year
Overview
Brief 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).
Investigators
Eligibility Criteria
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.
Outcomes
Primary Outcomes
To evaluate the safety and tolerance of FT-002
Time Frame: from FT-002 administration through up to 1 years
Incidence and severity of AEs
Secondary Outcomes
- To evaluate the efficacy of FT-002(from FT-002 administration through up to 1 years)
Study Sites (1)
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