One-Shot gene therapy aims to stop debilitating swelling attacks

NCT ID NCT06634420

Summary

This study is testing whether a single infusion of an experimental gene therapy called NTLA-2002 can safely and effectively reduce the number of sudden, severe swelling attacks in people with hereditary angioedema (HAE). About 60 participants will be randomly assigned to receive either the therapy or a placebo infusion. The main goal is to see if the treatment lowers the rate of HAE attacks over 28 weeks, with follow-up for nearly two years.

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Contacts and locations

Locations

  • AARA Research Center

    Dallas, Texas, 75231, United States

  • AP-HM - Hopital de la Timone

    Marseille, 13385, France

  • Allergy & Asthma Clinical Research

    Walnut Creek, California, 94598, United States

  • Amsterdam UMC - Locatie AMC

    Amsterdam, 1105 AZ, Netherlands

  • Asthma & Allergy Associates

    Colorado Springs, Colorado, 80907, United States

  • Bernstein Clinical Research Center, LLC

    Cincinnati, Ohio, 45236, United States

  • CHU Grenoble-Alpes - Hopital Michallon

    Grenoble, 38043, France

  • CHU de Lille - Hopital Claude Huriez

    Lille, 59037, France

  • Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ, United Kingdom

  • Campbelltown Hospital

    Campbelltown, New South Wales, 2560, Australia

  • Charite Universitaetsmedizin Berlin

    Berlin, 12203, Germany

  • IMMUNOe International Research Centers

    Centennial, Colorado, 80112, United States

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Medical Research of Arizona

    Scottsdale, Arizona, 85251, United States

  • Medizinische Hochschule Hannover (MHH)

    Hanover, 30625, Germany

  • NYU Langone Health - Long Island

    Mineola, New York, 11501, United States

  • New Zealand Clinical Research (NZCR)

    Auckland, 1010, New Zealand

  • Ottawa Allergy Research Corporation

    Ottawa, Ontario, K1H 1E4, Canada

  • Penn State Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033, United States

  • Raffi Tachdjian MD, Inc

    Santa Monica, California, 90404, United States

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • The Alfred Hospital

    Melbourne, Victoria, 3004, Australia

  • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

    Frankfurt, 60590, Germany

  • University of Alberta

    Edmonton, Alberta, T6G 2G3, Canada

  • University of California, San Diego (UCSD)

    San Diego, California, 92122, United States

  • University of Cape Town - Lung Institute - Lung Clinical Research Unit

    Cape Town, 7700, South Africa

  • University of South Florida

    Tampa, Florida, 33613, United States

  • Washington University in St. Louis

    St Louis, Missouri, 63141, United States

Conditions

Explore the condition pages connected to this study.