Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
NCT ID: NCT04671433
Last Updated: 2025-09-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
97 participants
INTERVENTIONAL
2020-12-04
2024-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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Experimental - Immediate Treatment
Intermediate dose.
Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Deferred Treatment
Deferred Treatment
Genetic: AAV5-hRKp.RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)
Experimental Immediate Treatment
Low dose.
Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Interventions
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Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Genetic: AAV5-hRKp.RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
* Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
3 Years
ALL
No
Sponsors
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Janssen Research & Development, LLC
INDUSTRY
Responsible Party
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Principal Investigators
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Janssen Research & Development, LLC Clinical Trial
Role: STUDY_DIRECTOR
Janssen Research & Development, LLC
Locations
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Shiley Eye Institute Jacobs Retina Center
La Jolla, California, United States
Childrens Hospital
Los Angeles, California, United States
Stanford Health Care
Palo Alto, California, United States
VitreoRetinal Associates, PA
Gainesville, Florida, United States
Emory University
Atlanta, Georgia, United States
Massachusetts General Hospital - Center for Celiac Research and Treatment
Boston, Massachusetts, United States
Univ of Michigan Medical Center
Ann Arbor, Michigan, United States
Duke Eye Center
Durham, North Carolina, United States
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
UZ Gent
Ghent, , Belgium
Hospital For Sick Children
Toronto, Ontario, Canada
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
Napoli, , Italy
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
Roma, , 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
Countries
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Other Identifiers
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MGT-RPGR-021
Identifier Type: OTHER
Identifier Source: secondary_id
2020-002873-88
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CR109258
Identifier Type: -
Identifier Source: org_study_id
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