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

NCT ID: NCT06492850

Last Updated: 2024-07-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

32 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-01

Study Completion Date

2026-02-01

Brief Summary

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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).

Detailed Description

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Conditions

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X-Linked Retinitis Pigmentosa (XLRP)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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FT-002 dose 1

Low dose FT-002

Group Type EXPERIMENTAL

FT-002

Intervention Type GENETIC

Intraocular injection of a single dose

High dose FT-002

High dose FT-002

Group Type EXPERIMENTAL

FT-002

Intervention Type GENETIC

Intraocular injection of a single dose

Interventions

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FT-002

Intraocular injection of a single dose

Intervention Type GENETIC

Eligibility Criteria

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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.
Minimum Eligible Age

8 Years

Maximum Eligible Age

45 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Frontera Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ruifang Sui

Role: PRINCIPAL_INVESTIGATOR

Peking Union Medical College Hospital

Locations

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Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Xinyan Li

Role: CONTACT

+86-021-58206061

Minghui Xue

Role: CONTACT

+86-021-58206061

Facility Contacts

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Ruifang Sui, Professor

Role: primary

Other Identifiers

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FT002-C101

Identifier Type: -

Identifier Source: org_study_id

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