NCT05858983

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Frontera Therapeutics · Industry
• Sex: All
• Age: 8 Years – 45 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This study is a multi-center, open-label, phase I/II clinical study to evaluate the safety, tolerability, efficacy, immunogenicity, and in vivo biodistribution characteristics of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.

Conditions

• Biallelic RPE65 Mutation-associated Retinal Dystrophy

Interventions

Type	Name	Description
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

Timeline

• Start date: 2022-11-30
• Primary completion: 2025-11-30
• Completion: 2029-11-30
• First posted: 2023-05-15
• Last updated: 2023-05-15

Locations

1 site across 1 country: China