Gene therapy trial seeks to halt vision loss from inherited eye disease

NCT ID NCT04794101

Summary

This is a long-term follow-up study for people who received an experimental gene therapy for X-linked retinitis pigmentosa (XLRP), a genetic disease that causes progressive vision loss. The main goals are to check the long-term safety of the treatment and to see if it continues to help preserve vision over time. The study is monitoring 97 participants, including children as young as 3 years old, who have a specific genetic cause for their condition.

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Contacts and locations

Locations

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

    Naples, 80131, Italy

  • Azienda Ospedaliero Universitaria Careggi

    Florence, 50134, Italy

  • Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts

    Paris, 75012, France

  • Childrens Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Duke Eye Center

    Durham, North Carolina, 27705, United States

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Gartnavel General Hospital

    Glasgow, G12 0YN, United Kingdom

  • Ghent University Hospital

    Ghent, 9000, Belgium

  • Hadassah Medical Center

    Jerusalem, 91120, Israel

  • Hosp Univ Fund Jimenez Diaz

    Madrid, 28040, Spain

  • Hospital For Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    London, EC1V 2PD, United Kingdom

  • NHS Lothian

    Edinburgh, EH3 9HA, United Kingdom

  • Radboudumc

    Nijmegen, 6525EX, Netherlands

  • Rigshospitalet Glostrup

    Glostrup Municipality, 2600, Denmark

  • Shiley Eye Institute Jacobs Retina Center

    La Jolla, California, 92093 0946, United States

  • St James University Hospital

    Leeds, LS9 7TF, United Kingdom

  • Stanford Health Care

    Palo Alto, California, 94303, United States

  • Univ of Michigan Medical Center

    Ann Arbor, Michigan, 48105, United States

  • Universite de Lausanne, Hopital ophtalmique Jules-Gonin

    Lausanne, 1004, Switzerland

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

    Basel, 4031, Switzerland

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania, 15213, United States

  • VUMC Amsterdam

    Amsterdam, 1105AZ, Netherlands

  • VitreoRetinal Associates, PA

    Gainesville, Florida, 32607, United States

Conditions

Explore the condition pages connected to this study.