Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

July 1, 2024 updated by: Frontera Therapeutics

A Phase I/II Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of FT-002 Subretinal Injection in Subjects With RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa.

The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

32

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:

  • Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form;
  • Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF;
  • Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.;

Exclusion Criteria:

  • Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product.

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-002 dose 1
Low dose FT-002
Genetic: FT-002
Intraocular injection of a single dose
Experimental: High dose FT-002
Genetic: FT-002
Intraocular injection of a single dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate the safety and tolerance of FT-002
Time Frame: from FT-002 administration through up to 1 years
Incidence and severity of AEs
from FT-002 administration through up to 1 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate the efficacy of FT-002
Time Frame: from FT-002 administration through up to 1 years
Changes in visual sensitivity /FST/BCVAfrom baseline
from FT-002 administration through up to 1 years

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)

April 1, 2024

Primary Completion (Estimated)

August 1, 2025

Study Completion (Estimated)

February 1, 2026

Study Registration Dates

First Submitted

July 1, 2024

First Submitted That Met QC Criteria

July 1, 2024

First Posted (Actual)

July 9, 2024

Study Record Updates

Last Update Posted (Actual)

July 9, 2024

Last Update Submitted That Met QC Criteria

July 1, 2024

Last Verified

July 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FT002-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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