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

Last updated: May 11, 2023
Sponsor: Frontera Therapeutics
Overall Status: Active - Recruiting

Phase

1/2

Condition

N/A

Treatment

FT-001 Mid Dose

FT-001 High Dose

FT-001 Low Dose

Clinical Study ID

NCT05858983
FT001-C101
  • Ages 8-45
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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

Exclusion Criteria:

  1. Other interfering eye diseases
  2. Presence of any systemic or ocular disease that can cause or likely to cause visionloss
  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

Study Design

Total Participants: 9
Treatment Group(s): 3
Primary Treatment: FT-001 Mid Dose
Phase: 1/2
Study Start date:
November 30, 2022
Estimated Completion Date:
November 30, 2029

Study 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.

Connect with a study center

  • Peking Union Medical College Hospital

    Beijing, Beijing 100142
    China

    Active - Recruiting

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