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RECRUITING
NCT05858983
PHASE1/PHASE2

Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Sponsor: Frontera Therapeutics

View on ClinicalTrials.gov

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

GENETIC

FT-001 Low Dose

Comparison of different dosages of FT-001

GENETIC

FT-001 Mid Dose

Comparison of different dosages of FT-001

GENETIC

FT-001 High Dose

Comparison of different dosages of FT-001

Locations (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China