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

Early Phase 1

Recruiting

• Conditions: X-Linked Retinitis Pigmentosa

• 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

Study Timeline

• Status Verified: 2022-08
• Study Start: 2023-02-01
• Study First Submitted: 2023-04-29
• Study First Submitted QC: 2023-05-23
• Last Update Submitted: 2023-05-23
• Study First Posted: 2023-05-24
• Last Update Posted: 2023-05-24
• Primary Completion: 2023-10-31
• Study Completion: 2027-11-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 18

Inclusion Criteria

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 ;

Exclusion Criteria

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

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