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 Therapeutics1 site in 1 country9 target enrollmentNovember 30, 2022

ConditionsBiallelic RPE65 Mutation-associated Retinal Dystrophy

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Biallelic RPE65 Mutation-associated Retinal Dystrophy
Sponsor: Frontera Therapeutics
Enrollment: 9
Locations: 1
Primary Endpoint: Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Status: Recruiting
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

November 30, 2022

End Date

November 30, 2029

Last Updated

2 years ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Frontera Therapeutics

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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 Criteria

•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