Overview
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.
Description
This study is a multi-center, open-label, phase I/II clinical study to evaluate the safety, tolerability, efficacy, immunogenicity, and in vivo biodistribution characteristics of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.
Eligibility
Inclusion Criteria:
- Subjects who are able to understand and sign the ICF
- Female or male aged 8-45 years old when signing the ICF
- Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
Exclusion Criteria:
- Other interfering eye diseases
- Presence of any systemic or ocular disease that can cause or likely to cause vision loss
- There is evidence of obviously uncontrolled concomitant diseases
- Known to have active or suspected autoimmune diseases
- With active systemic infection under treatment
- Pregnant or lactating women
- Other conditions unsuitable for the study as determined by the investigator