Gene therapy for retinal dystrophy caused by RPE65 mutations
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
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 |
| Enrollment | 9 (estimated) |
| Ages | 8 Years to 45 Years |
| Sex | All |
| Sponsor | Frontera Therapeutics Industry-sponsored |
| Locations | 1 site (Beijing, Beijing Municipality) |
| Trial ID | NCT05858983 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 8-45 years with a clinical diagnosis of biallelic RPE65 mutation-associated retinal dystrophy.
Not a fit: Patients with other eye diseases or systemic conditions that could affect vision are unlikely to benefit from this study.
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 performed: Previous studies on gene therapy for retinal dystrophies have shown promising results, indicating potential for success in this approach.
Eligibility criteria
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 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
- 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.