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

Plain-language summary

This study examines a gene therapy called FT-002 in patients with X-linked retinitis pigmentosa associated with a mutation in the RPGR gene. It enrolls male patients with a clinical XLRP diagnosis who have symptoms such as night blindness and vision loss. It is being conducted in Phase 1/Phase 2 and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

Frontera Therapeutics

Intervention

FT-002

Start date

4/1/2024

Estimated completion

2/1/2026

Countries

China

Related genes

Locations

• Beijing, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.