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

Plain-language summary

This study evaluates the gene therapy FT-001 at different doses in people with retinal dystrophy associated with biallelic RPE65 gene mutations (in both copies of the gene). It enrolls people aged 8 to 45 with this diagnosis. It is in Phase 1/Phase 2 and is currently recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

Frontera Therapeutics

Intervention

FT-001 Low Dose, FT-001 Mid Dose, FT-001 High Dose

Start date

11/30/2022

Estimated completion

11/30/2029

Countries

China

Related genes

Locations

• Beijing, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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