NCT05858983 Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
| NCT ID | NCT05858983 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Frontera Therapeutics |
| Condition | Biallelic RPE65 Mutation-associated Retinal Dystrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 9 participants |
| Start Date | 2022-11-30 |
| Primary Completion | 2025-11-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 9 participants in total. It began in 2022-11-30 with a primary completion date of 2025-11-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
Eligibility 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
Contact & Investigator
Ruifang Sui
PRINCIPAL INVESTIGATOR
Peking Union Medical College Hospital
Frequently Asked Questions
Who can join the NCT05858983 clinical trial?
This trial is open to participants of all sexes, aged 8 Years or older, up to 45 Years, studying Biallelic RPE65 Mutation-associated Retinal Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05858983 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05858983 currently recruiting?
Yes, NCT05858983 is actively recruiting participants. Contact the research team at Xinyan.li@fronteratherapeutics.com for enrollment information.
Where is the NCT05858983 trial being conducted?
This trial is being conducted at Beijing, China.
Who is sponsoring the NCT05858983 clinical trial?
NCT05858983 is sponsored by Frontera Therapeutics. The principal investigator is Ruifang Sui at Peking Union Medical College Hospital. The trial plans to enroll 9 participants.