Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)

Plain-language summary

This study examines a gene therapy called AAV5-RPGR in males with X-linked retinitis pigmentosa. It was designed for male patients aged 5 and over whose diagnosis was confirmed by a retinal specialist. The study was conducted in Phase 1/Phase 2 and has been completed.

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

Completed

Sponsor

MeiraGTx UK II Ltd

Intervention

AAV5-RPGR

Start date

7/31/2017

Estimated completion

11/18/2021

Countries

United States, United Kingdom

Related genes

Locations

• Boston, United States
• Ann Arbor, United States
• Pittsburgh, United States
• Leeds, United Kingdom
• London, United Kingdom

Eligibility criteria

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

Key inclusion Criteria:

* Males aged 5 years or older
* Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)

Key exclusion Criteria:

• Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months