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

Primary Purpose

X-Linked Retinitis Pigmentosa

Status

Recruiting

Phase

Early Phase 1

Locations

China

Study Type

Interventional

Intervention

FT-002

About this trial

This is an interventional treatment trial for X-Linked Retinitis Pigmentosa

Eligibility Criteria

8 Years - 45 Years (Child, Adult)MaleDoes not accept 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.

Sites / Locations

Peking Union Medical College HospitalRecruiting
Eye & ENT hospital of Fudan universityRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Low dose FT-002

Intermediate dose FT-002

High dose FT-002

Arm Description

Intraocular injection of a single low dose of FT-002

Intraocular injection of a single Intermediate dose of FT-002

Intraocular injection of a single High dose of FT-002

Outcomes

Primary Outcome Measures

Incidence of AEs

Number and proportion of Adverse Events

Secondary Outcome Measures

Change in visual function

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

Change in Outer Nuclear Layer Thickness from baseline as assessed by Optical Coherence Tomography

Full Information

NCT ID

NCT05874310

First Posted

April 29, 2023

Last Updated

May 23, 2023

Sponsor

Frontera Therapeutics

Collaborators

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

1. Study Identification

Unique Protocol Identification Number

NCT05874310

Brief Title

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

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.

Study Type

Interventional

2. Study Status

Record Verification Date

August 2022

Overall Recruitment Status

Recruiting

Study Start Date

February 1, 2023 (Actual)

Primary Completion Date

October 31, 2023 (Anticipated)

Study Completion Date

November 1, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Frontera Therapeutics

Collaborators

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

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

X-Linked Retinitis Pigmentosa

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Sequential Assignment

Model Description

X-linked retinitis pigmentosa patients

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

18 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Low dose FT-002

Arm Type

Experimental

Arm Description

Intraocular injection of a single low dose of FT-002

Arm Title

Intermediate dose FT-002

Arm Type

Experimental

Arm Description

Intraocular injection of a single Intermediate dose of FT-002

Arm Title

High dose FT-002

Arm Type

Experimental

Arm Description

Intraocular injection of a single High dose of FT-002

Intervention Type

Genetic

Intervention Name(s)

FT-002

Intervention Description

Comparison of different dosages of FT-002

Primary Outcome Measure Information:

Title

Incidence of AEs

Description

Number and proportion of Adverse Events

Time Frame

from FT-002 administration through up to 1 years

Secondary Outcome Measure Information:

Title

Change in visual function

Description

Change in retinal function as assessed by mean retinal sensitivity within the 30-degree visual field

Time Frame

from FT-002 administration through up to 1 years

Title

Change in retinal structure as assessed by Optical Coherence Tomography

Description

Change in Outer Nuclear Layer Thickness from baseline as assessed by Optical Coherence Tomography

Time Frame

from FT-002 administration through up to 1 years

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

8 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility Criteria

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Xinyan Li

Phone

+86-021-58206061

xinyan.li@fronteratherapeutics.com

First Name & Middle Initial & Last Name or Official Title & Degree

Minghui Xue

Phone

+86-021-58206061

minghui.xue@fronteratherapeutics.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Gezhi Xu

Organizational Affiliation

Eye & ENT Hospital of Fudan University

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Ruifang Sui

Organizational Affiliation

Peking Union Medical College Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Peking Union Medical College Hospital

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100142

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Ruifang Sui, Professor

Facility Name

Eye & ENT hospital of Fudan university

City

Shanghai

State/Province

Shanghai

ZIP/Postal Code

200032

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Gezhi Xu, Professor

First Name & Middle Initial & Last Name & Degree

Jihong Wu, Professor

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing Plan Description

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

Learn more about this trial

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

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