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

May 11, 2023 updated by: Frontera Therapeutics

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

9

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100142
        • Recruiting
        • Peking Union Medical College Hospital
        • Contact:
          • Ruifang Sui, Professor

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
Time Frame: 52 weeks
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
Time Frame: 52 weeks
Changes in visual function from baseline as assessed by FST
52 weeks
Changes in visual function from baseline
Time Frame: 52 weeks
Changes in visual function from baseline as assessed by Mobility courses
52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Frontera Therapeutics

Investigators

  • Principal Investigator: Ruifang Sui, Peking Union Medical College Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 30, 2022

Primary Completion (Anticipated)

November 30, 2025

Study Completion (Anticipated)

November 30, 2029

Study Registration Dates

First Submitted

April 29, 2023

First Submitted That Met QC Criteria

May 11, 2023

First Posted (Actual)

May 15, 2023

Study Record Updates

Last Update Posted (Actual)

May 15, 2023

Last Update Submitted That Met QC Criteria

May 11, 2023

Last Verified

October 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FT001-C101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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