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

Sponsor

Frontera Therapeutics (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05874310

Collaborator

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

Enrollment

Locations

Arms

Anticipated Duration (Months)

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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

Study Type:

Interventional

Anticipated Enrollment :

18 participants

Allocation:

Non-Randomized

Intervention Model:

Sequential Assignment

Intervention Model Description:

X-linked retinitis pigmentosa patientsX-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 :

Feb 1, 2023

Anticipated Primary Completion Date :

Oct 31, 2023

Anticipated Study Completion Date :

Nov 1, 2027

Arms and Interventions

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

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

Primary Outcome Measures

Incidence of AEs [from FT-002 administration through up to 1 years]
Number and proportion of Adverse Events

Secondary Outcome Measures

Change in visual function [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
Change in retinal structure as assessed by Optical Coherence Tomography [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

Criteria

Ages Eligible for Study:

8 Years to 45 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	Peking Union Medical College Hospital	Beijing	Beijing	China	100142
2	Eye & ENT hospital of Fudan university	Shanghai	Shanghai	China	200032

Sponsors and Collaborators

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

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Frontera Therapeutics

ClinicalTrials.gov Identifier:

NCT05874310

Other Study ID Numbers:

FT002RP-1

First Posted:

May 24, 2023

Last Update Posted:

May 24, 2023

Last Verified:

Aug 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Retinitis

Retinitis Pigmentosa

Study Results

No Results Posted as of May 24, 2023