Clinical Trials Directory

Trials / Completed

CompletedNCT04671433

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

Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Status
Completed
Phase
Phase 3
Study type
Interventional
Enrollment
105 (actual)
Sponsor
Janssen Research & Development, LLC · Industry
Sex
All
Age
3 Years
Healthy volunteers
Not accepted

Summary

A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Conditions

Interventions

TypeNameDescription
BIOLOGICALGenetic: AAV5-hRKp.RPGRBilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
BIOLOGICALGenetic: AAV5-hRKp.RPGRNo intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)

Timeline

Start date
2020-12-04
Primary completion
2024-09-30
Completion
2024-09-30
First posted
2020-12-17
Last updated
2026-03-16

Locations

28 sites across 11 countries: United States, Belgium, Canada, Denmark, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom

Regulatory

Source: ClinicalTrials.gov record NCT04671433. Inclusion in this directory is not an endorsement.