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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Biallelic RPE65 Mutation-associated Retinal Dystrophy

Intervention / Treatment
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* Genetic: FT-001 Low Dose
* Genetic: FT-001 Mid Dose
* Genetic: FT-001 High Dose


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FT-001 Dose 1<br>Intraocular administration of a single low dose of range FT-001 | Genetic: FT-001 Low Dose<br>* Comparison of different dosages of FT-001<br>|
| Experimental: FT-001 Dose 2<br>Intraocular administration of a single Mid dose of range FT-001 | Genetic: FT-001 Mid Dose<br>* Comparison of different dosages of FT-001<br>|
| Experimental: FT-001 Dose 3<br>Intraocular administration of a single High dose of range FT-001 | Genetic: FT-001 High Dose<br>* Comparison of different dosages of FT-001<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs) | Incidence of ocular and non-ocular AEs and SAEs | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in visual function from baseline | Changes in visual function from baseline as assessed by FST | 52 weeks | 
| Changes in visual function from baseline | Changes in visual function from baseline as assessed by Mobility courses | 52 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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FT-001, Biallelic RPE65 Mutation-associated Retinal Dystrophy