Gene therapy for X-linked retinitis pigmentosa
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.
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 |
| Enrollment | 18 (estimated) |
| Ages | 8 Years to 45 Years |
| Sex | Male |
| Sponsor | Frontera Therapeutics Industry-sponsored |
| Locations | 2 sites (Beijing, Beijing Municipality and 1 other locations) |
| Trial ID | NCT05874310 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are males aged 8-45 years with confirmed RPGR 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 gene therapy could significantly improve vision or halt the progression of blindness in patients with XLRP.
How similar studies have performed: Other studies have shown promise in using gene therapy for retinal diseases, suggesting potential success for this approach.
Eligibility criteria
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 this trial is running
Beijing, Beijing Municipality and 1 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.