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

Last updated: March 28, 2025
Sponsor: Janssen Research & Development, LLC
Overall Status: Completed

Phase

3

Condition

Retinitis Pigmentosa

Eye Disorders/infections

Posterior Uveitis

Treatment

Genetic: AAV5-hRKp.RPGR

Genetic: AAV5-RPGR

Clinical Study ID

NCT04671433
CR109258
MGT-RPGR-021
2020-002873-88
  • Ages > 3
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male or female

  • 3 years of age or older

  • Has XLRP confirmed by a retinal specialist and has a predicted disease-causingsequence variant in RPGR confirmed by an accredited laboratory

Exclusion

Exclusion Criteria:

  • Has had ocular surgery within 3 months prior to screening or is anticipated torequire ocular surgery within 6 months after the study intervention administration

  • Any investigational ocular treatment or any other ocular treatment that couldconfound the interpretation of the efficacy results or affect participant compliancewith the visit schedule

  • Has undergone prior retinal surgery involving the macula, macular laserphotocoagulation, external-beam radiation therapy, transpupillary thermotherapy,glaucoma filtration surgery or corneal surgery

Study Design

Total Participants: 97
Treatment Group(s): 2
Primary Treatment: Genetic: AAV5-hRKp.RPGR
Phase: 3
Study Start date:
December 04, 2020
Estimated Completion Date:
September 30, 2024

Connect with a study center

  • UZ Gent

    Gent, 9000
    Belgium

    Site Not Available

  • Ghent University Hospital

    Ghent, 9000
    Belgium

    Site Not Available

  • Hospital For Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Site Not Available

  • Rigshospitalet Glostrup

    Glostrup, 2600
    Denmark

    Site Not Available

  • Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts

    Paris, 75012
    France

    Site Not Available

  • Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts

    Paris, 75012
    France

    Site Not Available

  • Hadassah Medical Center

    Jerusalem, 91120
    Israel

    Site Not Available

  • Azienda Ospedaliero Universitaria Careggi

    Firenze, 50134
    Italy

    Site Not Available

  • Azienda Ospedaliero, Universitaria Careggi

    Florence,
    Italy

    Site Not Available

  • Ospedale San Paolo di Milano

    Milan,
    Italy

    Site Not Available

  • Ospedale San Paolo

    Milano, 20142
    Italy

    Site Not Available

  • AOU Università degli, Studi della Campania

    Napoli,
    Italy

    Site Not Available

  • Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli

    Napoli, 80131
    Italy

    Site Not Available

  • IRCCS Fondazione G. B. Bietti , Ospedale Britannico

    Roma,
    Italy

    Site Not Available

  • IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS

    Roma, 00198
    Italy

    Site Not Available

  • Amsterdam UMC

    Amsterdam, 1105AZ
    Netherlands

    Site Not Available

  • VUMC Amsterdam

    Amsterdam, 1105AZ
    Netherlands

    Site Not Available

  • Radboud University Medical Center

    Nijmegen, 6525EX
    Netherlands

    Site Not Available

  • Radboudumc

    Nijmegen, 6525EX
    Netherlands

    Site Not Available

  • Hosp Univ Fund Jimenez Diaz

    Madrid, 28040
    Spain

    Site Not Available

  • Hosp. Univ. Fund. Jimenez Diaz

    Madrid, 28040
    Spain

    Site Not Available

  • University Hospital Fundación Jiménez Díaz

    Madrid, 28040
    Spain

    Site Not Available

  • University Hospital Basel

    Basel, 4031
    Switzerland

    Site Not Available

  • University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical

    Basel, 4031
    Switzerland

    Site Not Available

  • Hôpital ophtalmique Jules-Gonin

    Lausanne, 1004
    Switzerland

    Site Not Available

  • Universite de Lausanne, Hopital ophtalmique Jules-Gonin

    Lausanne, 1004
    Switzerland

    Site Not Available

  • NHS Lothian

    Edinburgh, EH3 9HA
    United Kingdom

    Site Not Available

  • Gartnavel General Hospital

    Glasgow, G12 0YN
    United Kingdom

    Site Not Available

  • Glasgow Centre for Ophthalmic Research, Gartnaval General Hospital

    Glasgow, G12 0YN
    United Kingdom

    Site Not Available

  • Leeds Centre for Ophthalmology, St James' University Hospital

    Leeds, LS9 7TF
    United Kingdom

    Site Not Available

  • St James University Hospital

    Leeds, LS9 7TF
    United Kingdom

    Site Not Available

  • Moorfield Eye Hospital (MEH)

    London, EC1V 2PD
    United Kingdom

    Site Not Available

  • Moorfields Eye Hospital

    London, EC1V 2PD
    United Kingdom

    Site Not Available

  • Shiley Eye Institute / Jacobs Retina Center

    La Jolla, California 92093-0946
    United States

    Site Not Available

  • Shiley Eye Institute Jacobs Retina Center

    La Jolla, California 92093 0946
    United States

    Site Not Available

  • Children's Hospital Los Angeles

    Los Angeles, California 90027
    United States

    Site Not Available

  • Childrens Hospital

    Los Angeles, California 90027
    United States

    Site Not Available

  • Stanford Health Care

    Palo Alto, California 94303
    United States

    Site Not Available

  • Stanford University

    Palo Alto, California 94303
    United States

    Site Not Available

  • Shiley Eye Institute, University of California San Diego

    San Diego, California 92093-0946
    United States

    Site Not Available

  • Vitreo Retinal Associates

    Gainesville, Florida 32607
    United States

    Site Not Available

  • VitreoRetinal Associates, PA

    Gainesville, Florida 32607
    United States

    Site Not Available

  • Emory University

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • The Emory Eye Centre

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Massachusetts Eye and Ear, Centre for Clinical Research Operations

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Massachusetts General Hospital - Center for Celiac Research and Treatment

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Univ of Michigan Medical Center

    Ann Arbor, Michigan 48105
    United States

    Site Not Available

  • University of Michigan, W.K. Kellogg Eye Centre

    Ann Arbor, Michigan 48105
    United States

    Site Not Available

  • Duke Eye Center

    Durham, North Carolina 27705
    United States

    Site Not Available

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania 15213
    United States

    Site Not Available

  • University of Pittsburgh Medical Centre

    Pittsburgh, Pennsylvania 15213
    United States

    Site Not Available

  • Retina Consultants of Houston

    Bellaire, Texas 77401
    United States

    Site Not Available

  • Retinal Consultants of Houston

    Bellaire, Texas 77401
    United States

    Site Not Available

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