NCT06492850RecruitingPhase 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.
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Sponsor
- Frontera Therapeutics
- Enrollment
- 32
- Locations
- 1
- Primary Endpoint
- To evaluate the safety and tolerance of FT-002
Study 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).
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 8 Years to 45 Years (Child, Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
High dose FT-002
Experimental
High dose FT-002
Intervention: FT-002 (Genetic)
FT-002 dose 1
Experimental
Low dose FT-002
Intervention: FT-002 (Genetic)
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)
Investigators
Study Sites (1)
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