Clinical trial

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.

NCT05874310

Name

FT002RP-1

Description

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

Trial arms

Trial start

2023-02-01

Estimated PCD

2023-10-31

Trial end

2027-11-01

Status

Recruiting

Phase

Early phase I

Treatment

FT-002

Comparison of different dosages of FT-002

Arms:

High dose FT-002, Intermediate dose FT-002, Low dose FT-002

Size

Primary endpoint

Incidence of AEs

from FT-002 administration through up to 1 years

Eligibility criteria

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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['EARLY_PHASE1'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'X-linked retinitis pigmentosa patients', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 18, 'type': 'ESTIMATED'}}

Updated at

2023-05-24

1 organization

1 product

1 indication

Organization

Frontera Therapeutics

Product

FT-002

Indication

Retinitis Pigmentosa