Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
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
Frontera Therapeutics
9 participants
Nov 30, 2022
INTERVENTIONAL
Conditions
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
Inclusion Criteria3
- 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 Criteria7
- 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
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Interventions
Comparison of different dosages of FT-001
Comparison of different dosages of FT-001
Comparison of different dosages of FT-001
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT05858983