Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
NCT ID: NCT04794101
Last Updated: 2025-11-10
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
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE3
97 participants
INTERVENTIONAL
2020-12-04
2029-12-19
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Deferred Treatment From MGT-RPGR-021 of Intermediate Dose
Deferred treatment
Genetic: AAV5-hRKp.RPGR Intermediate Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
Already Treated in MGT-RPGR-021
Already treated
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
Deferred Treatment From MGT-RPGR-021 Low Dose
Deferred treatment
Genetic: AAV5-hRKp.RPGR Low Dose
Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
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
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
Exclusion Criteria
3 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Janssen Research & Development, LLC
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Janssen Research & Development, LLC Clinical Trial
Role: STUDY_DIRECTOR
Janssen Research & Development, LLC
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Shiley Eye Institute Jacobs Retina Center
La Jolla, California, United States
Childrens Hospital Los Angeles
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 Eye and Ear Infirmary
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
Retina Consultants of Houston
Bellaire, Texas, United States
Ghent University Hospital
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
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
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
MGT-RPGR-022
Identifier Type: OTHER
Identifier Source: secondary_id
2020-002255-37
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2024-511411-25-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
MGT-RPGR-022
Identifier Type: -
Identifier Source: org_study_id