Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Plain-language summary

This is a follow-up study examining a gene therapy called AAV5-hRKp.RPGR in patients with X-linked retinitis pigmentosa caused by variants in the RPGR gene. It is open to patients aged 3 and older who have a confirmed disease-causing variant in RPGR. It is in Phase 3 and is currently active but not recruiting new participants.

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

Trial details

Phase

Phase 3

Status

Active, not recruiting

Sponsor

Janssen Research & Development, LLC

Intervention

Genetic: AAV5-hRKp.RPGR Intermediate Dose, Genetic: AAV5-hRKp.RPGR Low Dose

Start date

12/4/2020

Estimated completion

12/7/2029

Countries

United States, Belgium, Canada, Denmark, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom

Related genes

Locations

• La Jolla, United States
• Los Angeles, United States
• Palo Alto, United States
• Gainesville, United States
• Atlanta, United States
• Boston, United States
• Ann Arbor, United States
• Durham, United States
• Pittsburgh, United States
• Bellaire, United States
• Ghent, Belgium
• Toronto, Canada
• Glostrup Municipality, Denmark
• Paris, France
• Jerusalem, Israel
• Florence, Italy
• Milan, Italy
• Naples, Italy
• Rome, Italy
• Amsterdam, Netherlands
• Nijmegen, Netherlands
• Madrid, Spain
• Basel, Switzerland
• Lausanne, Switzerland
• Edinburgh, United Kingdom
• Glasgow, United Kingdom
• 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.

Inclusion Criteria:

* Male or female, 3 years of age or older, has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory.

Exclusion Criteria:

* None