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

Frontera Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: X-Linked Retinitis Pigmentosa (XLRP)

Treatments: Genetic: FT-002

Study type: Interventional

Funder types: Industry

Identifiers

NCT06492850

FT002-C101

Details and patient eligibility

About

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).

Enrollment: 32 estimated patients

Sex: Male

Ages: 8 to 45 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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32 participants in 2 patient groups

FT-002 dose 1

Experimental group

Description:

Low dose FT-002

Treatment:

Genetic: FT-002

High dose FT-002

Experimental group

Description:

High dose FT-002

Treatment:

Genetic: FT-002