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

May 23, 2023 updated by: Frontera Therapeutics

An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.

A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

18

Phase

  • Early 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
    • Shanghai
      • Shanghai, Shanghai, China, 200032
        • Recruiting
        • Eye & Ent Hospital of Fudan University
        • Contact:
          • Gezhi Xu, Professor
        • Contact:
          • Jihong Wu, 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 that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;

Exclusion Criteria:

1.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: Low dose FT-002
Intraocular injection of a single low dose of FT-002
Genetic: FT-002
Comparison of different dosages of FT-002
Experimental: Intermediate dose FT-002
Intraocular injection of a single Intermediate dose of FT-002
Genetic: FT-002
Comparison of different dosages of FT-002
Experimental: High dose FT-002
Intraocular injection of a single High dose of FT-002
Genetic: FT-002
Comparison of different dosages of FT-002

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of AEs
Time Frame: from FT-002 administration through up to 1 years
Number and proportion of Adverse Events
from FT-002 administration through up to 1 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in visual function
Time Frame: from FT-002 administration through up to 1 years
Change in retinal function as assessed by mean retinal sensitivity within the 30-degree visual field
from FT-002 administration through up to 1 years
Change in retinal structure as assessed by Optical Coherence Tomography
Time Frame: from FT-002 administration through up to 1 years
Change in Outer Nuclear Layer Thickness from baseline as assessed by Optical Coherence Tomography
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

Collaborators

Peking Union Medical College Hospital
Eye & ENT Hospital of Fudan University
Henan Provincial People's Hospital

Investigators

  • Principal Investigator: Ruifang Sui, Peking Union Medical College Hospital
  • Principal Investigator: Gezhi Xu, Eye & Ent Hospital of Fudan University

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)

February 1, 2023

Primary Completion (Anticipated)

October 31, 2023

Study Completion (Anticipated)

November 1, 2027

Study Registration Dates

First Submitted

April 29, 2023

First Submitted That Met QC Criteria

May 23, 2023

First Posted (Actual)

May 24, 2023

Study Record Updates

Last Update Posted (Actual)

May 24, 2023

Last Update Submitted That Met QC Criteria

May 23, 2023

Last Verified

August 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FT002RP-1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Researchers qualified can request the dataset, including de-identified individual subject data.

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.

Clinical Trials on X-Linked Retinitis Pigmentosa

Clinical Trials on FT-002

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Zimbabwe

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe