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

Phase 1

Recruiting

• Conditions: Biallelic RPE65 Mutation-associated Retinal Dystrophy
• Interventions: Genetic: FT-001 High Dose; Genetic: FT-001 Mid Dose; Genetic: FT-001 Low Dose

• Registration Number: NCT05858983
• Lead Sponsor: Frontera Therapeutics

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.

Study Timeline

• Status Verified: 2022-10
• Study Start: 2022-11-30
• Study First Submitted: 2023-04-29
• Study First Submitted QC: 2023-05-11
• Last Update Submitted: 2023-05-11
• Study First Posted: 2023-05-15
• Last Update Posted: 2023-05-15
• Primary Completion: 2025-11-30
• Study Completion: 2029-11-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

1. Subjects who are able to understand and sign the ICF
2. Female or male aged 8-45 years old when signing the ICF
3. Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy

Exclusion Criteria

1. Other interfering eye diseases
2. Presence of any systemic or ocular disease that can cause or likely to cause vision loss
3. There is evidence of obviously uncontrolled concomitant diseases
4. Known to have active or suspected autoimmune diseases
5. With active systemic infection under treatment
6. Pregnant or lactating women
7. Other conditions unsuitable for the study as determined by the investigator

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
FT-001 Dose 3	FT-001 High Dose	Intraocular administration of a single High dose of range FT-001
FT-001 Dose 2	FT-001 Mid Dose	Intraocular administration of a single Mid dose of range FT-001
FT-001 Dose 1	FT-001 Low Dose	Intraocular administration of a single low dose of range FT-001

Primary Outcome Measures

Name	Time	Method
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)	52 weeks	Incidence of ocular and non-ocular AEs and SAEs

Secondary Outcome Measures

Name	Time	Method
Changes in visual function from baseline	52 weeks	Changes in visual function from baseline as assessed by Mobility courses

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China