Gene therapy for a specific type of inherited vision loss

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.

This study is testing a one-time gene therapy injection to see if it can help improve vision in boys and young men with a specific inherited eye condition caused by a gene mutation.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 32 (estimated)
• Ages: 8 Years to 45 Years
• Sex: Male
• Sponsor: Frontera Therapeutics (Industry-sponsored)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06492850 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety, tolerability, and efficacy of a one-time subretinal injection of FT-002 in male subjects aged 8 to 45 years with X-linked retinitis pigmentosa caused by RPGR gene mutations. It consists of a Phase I dose escalation phase followed by a Phase II dose expansion phase. The goal is to determine how well this gene therapy can improve vision and manage symptoms associated with this condition.

Who should consider this trial

Good fit: Ideal candidates are males aged 8 to 45 years with a clinically diagnosed case of X-linked retinitis pigmentosa due to RPGR gene mutations.

Not a fit: Patients with other retinal degenerative diseases or those who have previously received gene therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve vision and quality of life for patients with X-linked retinitis pigmentosa.

How similar studies have performed: Other studies have shown promise with gene therapy approaches for retinal diseases, indicating potential for success in this novel application.

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.

Where this trial is running

Beijing, Beijing Municipality

• Peking Union Medical College Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Principal investigator: Ruifang Sui — Peking Union Medical College Hospital
• Study coordinator: Xinyan Li

Conditions X-Linked Retinitis Pigmentosa; FT-002