Gene therapy for X-linked retinitis pigmentosa
Official title: An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.
Run by Frontera Therapeutics · NCT05874310 · Data updated 2026-11-12
This study tests a new gene therapy to see if it can help restore vision or slow down the eye disease in boys and men aged 8 to 45 with X-linked retinitis pigmentosa caused by RPGR gene mutations.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 8 Years to 45 Years |
| Sex | Men only |
| Enrollment | 18 (estimated) |
| Sponsor | Frontera Therapeutics Industry-sponsored |
| Locations | 2 sites (Beijing, Beijing Municipality and 1 other locations) |
| Status | Recruiting |
| Started | February 1, 2023 |
| Main results expected | October 31, 2023 |
| Study ends | November 1, 2027 |
| Registry record updated | May 24, 2023 |
| Trial ID | NCT05874310 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are males aged 8-45 years with confirmed RPGR mutations.
✕Probably not a fit
Patients with other retinal degenerative diseases or those who have previously received gene therapy may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial investigates the use of gene therapy to treat patients with X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. The intervention involves administering FT-002 to eligible male participants aged 8 to 45 years who have confirmed RPGR variants. The study aims to assess the safety and efficacy of this gene therapy approach in restoring vision or slowing disease progression in affected individuals.
Why it matters
Potential benefit: If successful, this gene therapy could significantly improve vision or halt the progression of blindness in patients with XLRP.
How similar studies have gone: Other studies have shown promise in using gene therapy for retinal diseases, suggesting potential success for this approach.
Who can join: the full rules
Show full inclusion / exclusion criteria
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.
Where it's running
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Peking Union Medical College Hospital
Beijing, Beijing Municipality, China Recruiting -
Eye & ENT hospital of Fudan university
Shanghai, Shanghai Municipality, China Recruiting
Study contacts
- Principal investigator: Gezhi Xu — Eye & ENT Hospital of Fudan University
- Study coordinator: Xinyan Li
- Email: xinyan.li@fronteratherapeutics.com
- Phone: +86-021-58206061
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Gene therapy for retinal dystrophy caused by RPE65 mutations
This study is testing a new gene therapy called FT-001 to see if it can improve vision in people with retinal dystrophy caused by RPE65 mutations.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.