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Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Sponsor: Frontera Therapeutics
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.
Official title: 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
Key Details
Gender
All
Age Range
8 Years - 45 Years
Study Type
INTERVENTIONAL
Enrollment
9
Start Date
2022-11-30
Completion Date
2029-11-30
Last Updated
2023-05-15
Healthy Volunteers
No
Interventions
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
Locations (1)
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China