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

NCT04794101 · tracked via the Priya Life Science UK tracker

Sponsor

Janssen Research & Development, LLC

Phase

Phase 3

Started

2020-12-04

Last updated

2026-08-28

Condition(s) studied

X-Linked Retinitis Pigmentosa

Investigational drug(s) / intervention(s)

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

Genetic: AAV5-hRKp.RPGR Intermediate Dose: Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Genetic: AAV5-hRKp.RPGR Low Dose: Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Study summary

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

Eligibility

Sex

ALL

Min age

3 Years

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Number of Participants with Ocular and Non-ocular Adverse Events — Day 1 - Month 60
Number of participants with ocular and non-ocular adverse events will be assessed.
Number of Participants With Abnormalities in Laboratory Assessments — Day 1 - Month 60
Number of participants with abnormalities in laboratory assessments will be assessed.
Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment — From Baseline - Month 60
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment — From Baseline- Month 60
Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.

Trial sites (28)

Facility	City	Region
Shiley Eye Institute Jacobs Retina Center	La Jolla	California
Childrens Hospital Los Angeles	Los Angeles	California
Stanford Health Care	Palo Alto	California
VitreoRetinal Associates, PA	Gainesville	Florida
Emory University	Atlanta	Georgia
Massachusetts Eye and Ear Infirmary	Boston	Massachusetts
Univ of Michigan Medical Center	Ann Arbor	Michigan
Duke Eye Center	Durham	North Carolina
University of Pittsburgh Medical Center (UPMC)	Pittsburgh	Pennsylvania
Retina Consultants of Houston	Bellaire	Texas
Ghent University Hospital	Ghent	Belgium
Hospital For Sick Children	Toronto	Ontario
Rigshospitalet Glostrup	Glostrup Municipality	Denmark
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts	Paris	France
Hadassah Medical Center	Jerusalem	Israel
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Ospedale San Paolo	Milan	Italy
Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli	Naples	Italy
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS	Rome	Italy
VUMC Amsterdam	Amsterdam	Netherlands
Radboudumc	Nijmegen	Netherlands
Hosp Univ Fund Jimenez Diaz	Madrid	Spain
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical	Basel	Switzerland
Universite de Lausanne, Hopital ophtalmique Jules-Gonin	Lausanne	Switzerland
NHS Lothian	Edinburgh	United Kingdom
Gartnavel General Hospital	Glasgow	United Kingdom
St James University Hospital	Leeds	United Kingdom
Moorfields Eye Hospital	London	United Kingdom

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Other trials for the same condition

A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP) Active, not recruiting

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04794101 on ClinicalTrials.gov ↗ ← All trials in the UK