Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

Phase 1

Recruiting

• Conditions: X-Linked Retinitis Pigmentosa (XLRP)
• Interventions: Genetic: FT-002

• Registration Number: NCT06492850
• Lead Sponsor: Frontera Therapeutics

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2024-04-01
• Status Verified: 2024-07
• Study First Submitted: 2024-07-01
• Study First Submitted QC: 2024-07-01
• Last Update Submitted: 2024-07-01
• Study First Posted: 2024-07-09
• Last Update Posted: 2024-07-09
• Primary Completion: 2025-08-01
• Study Completion: 2026-02-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 32

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
FT-002 dose 1	FT-002	Low dose FT-002
High dose FT-002	FT-002	High dose FT-002

Primary Outcome Measures

Name	Time	Method
To evaluate the safety and tolerance of FT-002	from FT-002 administration through up to 1 years	Incidence and severity of AEs

Secondary Outcome Measures

Name	Time	Method
To evaluate the efficacy of FT-002	from FT-002 administration through up to 1 years	Changes in visual sensitivity /FST/BCVAfrom baseline

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China