RecruitingPhase 1Phase 2NCT05858983

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

Sponsor

Frontera Therapeutics

Enrollment

9 participants

Start Date

Nov 30, 2022

Study Type

INTERVENTIONAL

Conditions

Biallelic RPE65 Mutation-associated Retinal Dystrophy

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

Min Age: 8 YearsMax Age: 45 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying FT-001 High Dose, FT-001 Low Dose, and others for people with biallelic rpe65 mutation-associated retinal dystrophy. The study is currently recruiting participants at 1 location. People eligible for this study include aged 8 Years to 45 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICFT-001 Low Dose

Comparison of different dosages of FT-001

GENETICFT-001 Mid Dose

Comparison of different dosages of FT-001

GENETICFT-001 High Dose

Comparison of different dosages of FT-001

Locations(1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

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NCT05858983