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

Frontera Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Biallelic RPE65 Mutation-associated Retinal Dystrophy

Treatments: Genetic: FT-001 Low Dose; Genetic: FT-001 Mid Dose; Genetic: FT-001 High Dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT05858983

FT001-C101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 9 estimated patients

Sex: All

Ages: 8 to 45 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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9 participants in 3 patient groups

FT-001 Dose 1

Experimental group

Description:

Intraocular administration of a single low dose of range FT-001

Treatment:

Genetic: FT-001 Low Dose

FT-001 Dose 2

Experimental group

Description:

Intraocular administration of a single Mid dose of range FT-001

Treatment:

Genetic: FT-001 Mid Dose

FT-001 Dose 3

Experimental group

Description:

Intraocular administration of a single High dose of range FT-001

Treatment:

Genetic: FT-001 High Dose