Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
- Conditions
- X-Linked Retinitis Pigmentosa
- Interventions
- Genetic: FT-002
- Registration Number
- NCT05874310
- Lead Sponsor
- Frontera Therapeutics
- Brief Summary
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 18
1.Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;
1.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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Low dose FT-002 FT-002 Intraocular injection of a single low dose of FT-002 High dose FT-002 FT-002 Intraocular injection of a single High dose of FT-002 Intermediate dose FT-002 FT-002 Intraocular injection of a single Intermediate dose of FT-002
- Primary Outcome Measures
Name Time Method Incidence of AEs from FT-002 administration through up to 1 years Number and proportion of Adverse Events
- Secondary Outcome Measures
Name Time Method Change in retinal structure as assessed by Optical Coherence Tomography from FT-002 administration through up to 1 years Change in Outer Nuclear Layer Thickness from baseline as assessed by Optical Coherence Tomography
Change in visual function from FT-002 administration through up to 1 years Change in retinal function as assessed by mean retinal sensitivity within the 30-degree visual field
Trial Locations
- Locations (2)
Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China
Eye & ENT hospital of Fudan university
🇨🇳Shanghai, Shanghai, China