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

NCT06492850 | Recruiting Phase 1 DMC

• 1 other identifier: FT002-C101
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

X-Linked Retinitis Pigmentosa (XLRP)

Keywords

X-Linked Retinitis Pigmentosa (XLRP); FT-002

Outcome Measures

Primary Outcomes (1)

• To evaluate the safety and tolerance of FT-002

  Incidence and severity of AEs

  from FT-002 administration through up to 1 years

Secondary Outcomes (1)

• To evaluate the efficacy of FT-002

  from FT-002 administration through up to 1 years

Study Arms (2)

FT-002 dose 1

EXPERIMENTAL

Low dose FT-002

Genetic: FT-002

High dose FT-002

EXPERIMENTAL

High dose FT-002

Genetic: FT-002

Interventions

FT-002 GENETIC

Intraocular injection of a single dose

FT-002 dose 1; High dose FT-002

Eligibility Criteria

• Age: 8 Years - 45 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.;

You may not qualify if:

• 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.

Sponsors & Collaborators

• Frontera Therapeutics: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100142, China

RECRUITING

Study Officials

• Ruifang Sui

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xinyan Li

CONTACT

+86-021-58206061; xinyan.li@fronteratherapeutics.com

Minghui Xue

CONTACT

+86-021-58206061; minghui.xue@fronteratherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2024
• First Posted: July 9, 2024
• Study Start: April 1, 2024
• Primary Completion: August 1, 2025
• Study Completion: February 1, 2026
• Last Updated: July 9, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)