Gene therapy for retinal dystrophy caused by RPE65 mutations
Official title: A Multi-center, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of FT-001 Administered Via Subretinal Injection in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Run by Frontera Therapeutics · NCT05858983 · Data updated 2026-11-12
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.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 8 Years to 45 Years |
| Sex | All |
| Enrollment | 9 (estimated) |
| Sponsor | Frontera Therapeutics Industry-sponsored |
| Locations | 1 site (Beijing, Beijing Municipality) |
| Status | Recruiting |
| Started | November 30, 2022 |
| Main results expected | November 30, 2025 |
| Study ends | November 30, 2029 |
| Registry record updated | May 15, 2023 |
| Trial ID | NCT05858983 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals aged 8-45 years with a clinical diagnosis of biallelic RPE65 mutation-associated retinal dystrophy.
✕Probably not a fit
Patients with other eye diseases or systemic conditions that could affect vision are unlikely to 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 evaluates the safety, tolerability, and efficacy of a gene therapy called FT-001 administered subretinally in patients with biallelic RPE65 mutation-associated retinal dystrophy. It is a multi-center, open-label study that will assess various outcomes, including visual acuity and potential side effects, over a monitoring period of five years. The trial includes different dosage levels of FT-001 to determine the optimal treatment approach.
Why it matters
Potential benefit: If successful, this therapy could significantly improve vision and quality of life for patients with this specific genetic condition.
How similar studies have gone: Previous studies on gene therapy for retinal dystrophies have shown promising results, indicating potential for success in this approach.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Subjects who are able to understand and sign the ICF 2. Female or male aged 8-45 years old when signing the ICF 3. Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy Exclusion Criteria: 1. Other interfering eye diseases 2. Presence of any systemic or ocular disease that can cause or likely to cause vision loss 3. There is evidence of obviously uncontrolled concomitant diseases 4. Known to have active or suspected autoimmune diseases 5. With active systemic infection under treatment 6. Pregnant or lactating women 7. Other conditions unsuitable for the study as determined by the investigator
Where it's running
-
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
- Email: Xinyan.li@fronteratherapeutics.com
- Phone: +86-021-58206061
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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.