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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.
This is not medical advice. Consult your specialist.
View the official record on ClinicalTrials.gov →