FT-001 Low Dose: Comparison of different dosages of FT-001
FT-001 Mid Dose: Comparison of different dosages of FT-001
FT-001 High Dose: Comparison of different dosages of FT-001
Study 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
Sex
ALL
Min age
8 Years
Max age
45 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs) — 52 weeks Incidence of ocular and non-ocular AEs and SAEs
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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