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

Frontera Therapeutics

Status and phase: Enrolling; Early Phase 1

Conditions: X-Linked Retinitis Pigmentosa

Treatments: Genetic: FT-002

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT05874310

FT002RP-1

Details and patient eligibility

About

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

Enrollment: 18 estimated patients

Sex: Male

Ages: 8 to 45 years old

Volunteers: No 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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18 participants in 3 patient groups

Low dose FT-002

Experimental group

Description:

Intraocular injection of a single low dose of FT-002

Treatment:

Genetic: FT-002

Intermediate dose FT-002

Experimental group

Description:

Intraocular injection of a single Intermediate dose of FT-002

Treatment:

Genetic: FT-002

High dose FT-002

Experimental group

Description:

Intraocular injection of a single High dose of FT-002

Treatment:

Genetic: FT-002