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

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

Status
Recruiting
Phases
Phase 1Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT05858983
Enrollment
9
Registered
2023-05-15
Start date
2022-11-30
Completion date
2029-11-30
Last updated
2023-05-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Biallelic RPE65 Mutation-associated Retinal Dystrophy

Keywords

FT-001, Biallelic RPE65 Mutation-associated Retinal Dystrophy

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.

Interventions

GENETICFT-001 Low Dose

Comparison of different dosages of FT-001

GENETICFT-001 Mid Dose

Comparison of different dosages of FT-001

GENETICFT-001 High Dose

Comparison of different dosages of FT-001

Sponsors

Frontera Therapeutics
Lead SponsorINDUSTRY

Study design

Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
8 Years to 45 Years
Healthy volunteers
No

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

Design outcomes

Primary

MeasureTime frameDescription
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)52 weeksIncidence of ocular and non-ocular AEs and SAEs

Secondary

MeasureTime frameDescription
Changes in visual function from baseline52 weeksChanges in visual function from baseline as assessed by FST

Countries

China

Contacts

Primary ContactXinyan Li
Xinyan.li@fronteratherapeutics.com+86-021-58206061
Backup ContactMinghui Xue
minghui.xue@fronteratherapeutics.com+86-021-58206061

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026