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

Phase 2RecruitingNCT05858983

Frontera Therapeutics9 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Biallelic RPE65 Mutation-associated Retinal Dystrophy

Interventions

FT-001 Low DoseGENETIC

Comparison of different dosages of FT-001

FT-001 Mid DoseGENETIC

Comparison of different dosages of FT-001

FT-001 High DoseGENETIC

Comparison of different dosages of FT-001

Eligibility

Sex: ALLMin age: 8 YearsMax age: 45 Years

Medical Language ↔ Plain English

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

Locations (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

RECRUITING

Outcomes

Primary Outcomes

Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)

Incidence of ocular and non-ocular AEs and SAEs

Time frame: 52 weeks

Secondary Outcomes

Changes in visual function from baseline

Changes in visual function from baseline as assessed by FST

Time frame: 52 weeks

Changes in visual function from baseline

Changes in visual function from baseline as assessed by Mobility courses

Time frame: 52 weeks

Central Contacts

Xinyan Li

CONTACT

+86-021-58206061 Xinyan.li@fronteratherapeutics.com

Minghui Xue

CONTACT

+86-021-58206061 minghui.xue@fronteratherapeutics.com

Data from ClinicalTrials.gov

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