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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05874310
Recruitment Status : Recruiting
First Posted : May 24, 2023
Last Update Posted : May 24, 2023
Sponsor:
Collaborators:
Eye & ENT Hospital of Fudan University
Peking Union Medical College Hospital
Henan Provincial People's Hospital
Information provided by (Responsible Party):
Frontera Therapeutics

Study Description
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Brief Summary:
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Genetic: FT-002 Early Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: X-linked retinitis pigmentosa patients
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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.
Actual Study Start Date : February 1, 2023
Estimated Primary Completion Date : October 31, 2023
Estimated Study Completion Date : November 1, 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of AEs [ Time Frame: from FT-002 administration through up to 1 years ]
    Number and proportion of Adverse Events


Secondary Outcome Measures :
  1. 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

  2. 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


Eligibility Criteria
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Ages Eligible for Study:   8 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05874310


Contacts
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Contact: Xinyan Li +86-021-58206061 xinyan.li@fronteratherapeutics.com
Contact: Minghui Xue +86-021-58206061 minghui.xue@fronteratherapeutics.com

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100142
Contact: Ruifang Sui, Professor         
China, Shanghai
Eye & ENT hospital of Fudan university Recruiting
Shanghai, Shanghai, China, 200032
Contact: Gezhi Xu, Professor         
Contact: Jihong Wu, Professor         
Sponsors and Collaborators
Frontera Therapeutics
Eye & ENT Hospital of Fudan University
Peking Union Medical College Hospital
Henan Provincial People's Hospital
Investigators
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Principal Investigator: Gezhi Xu Eye & ENT Hospital of Fudan University
Principal Investigator: Ruifang Sui Peking Union Medical College Hospital
More Information
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Responsible Party: Frontera Therapeutics
ClinicalTrials.gov Identifier: NCT05874310    
Other Study ID Numbers: FT002RP-1
First Posted: May 24, 2023    Key Record Dates
Last Update Posted: May 24, 2023
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Researchers qualified can request the dataset, including de-identified individual subject data.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn