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New pill aims to shrink enlarged hearts in rare genetic disease

NCT ID NCT05280548

Ongoing Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

Summary

This study is testing whether a daily oral medicine called venglustat can better control heart damage in adults with Fabry disease compared to current standard treatments. About 104 participants with an enlarged heart will take either venglustat or their usual therapy for 18 months while doctors track heart size and kidney function. The goal is to see if the new drug can better manage this serious complication of the disease.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005

    Chicago, Illinois, 60611, United States

  • Emory University School of Medicine - Atlanta- Site Number : 8400009

    Atlanta, Georgia, 30322, United States

  • Investigational Site Number : 0400001

    Graz, 8036, Austria

  • Investigational Site Number : 1240002

    Vancouver, British Columbia, V5Z 1M9, Canada

  • Investigational Site Number : 1240003

    Calgary, Alberta, T2M 0L6, Canada

  • Investigational Site Number : 1240005

    Toronto, Ontario, M5T 3H7, Canada

  • Investigational Site Number : 1240006

    Edmonton, Alberta, T6G 2B7, Canada

  • Investigational Site Number : 1560001

    Chengdu, 610041, China

  • Investigational Site Number : 1560002

    Beijing, 100034, China

  • Investigational Site Number : 1560003

    Shanghai, 200025, China

  • Investigational Site Number : 1560005

    Beijing, 100034, China

  • Investigational Site Number : 1560007

    Guangzhou, 510080, China

  • Investigational Site Number : 1580001

    Taipei, 104, Taiwan

  • Investigational Site Number : 1580003

    Taichung, 407, Taiwan

  • Investigational Site Number : 2030001

    Prague, 128 08, Czechia

  • Investigational Site Number : 2080001

    Copenhagen, 2100, Denmark

  • Investigational Site Number : 2500001

    Garches, 92380, France

  • Investigational Site Number : 2760001

    Würzburg, 97080, Germany

  • Investigational Site Number : 2760003

    Berlin, 10117, Germany

  • Investigational Site Number : 2760004

    Hochheim am Main, 65239, Germany

  • Investigational Site Number : 2760005

    Mainz, 55131, Germany

  • Investigational Site Number : 3000001

    Heraklion, 711 10, Greece

  • Investigational Site Number : 3000002

    Athens, 115 27, Greece

  • Investigational Site Number : 3000003

    Athens, 124 62, Greece

  • Investigational Site Number : 3800001

    Milan, Lombardy, 20122, Italy

  • Investigational Site Number : 3800002

    Naples, Napoli, 80131, Italy

  • Investigational Site Number : 3800003

    Naples, Napoli, 80131, Italy

  • Investigational Site Number : 3800004

    Bologna, 40138, Italy

  • Investigational Site Number : 3920001

    Tokyo, Tokyo, 105-8461, Japan

  • Investigational Site Number : 3920002

    Sendai, Miyagi, 980-8574, Japan

  • Investigational Site Number : 3920003

    Kagoshima, Kagoshima-ken, 890-8520, Japan

  • Investigational Site Number : 3920004

    Fukuoka, Fukuoka, 814-0180, Japan

  • Investigational Site Number : 3920005

    Kawasaki, Kanagawa, 215-0026, Japan

  • Investigational Site Number : 3920006

    Sapporo, Hokkaido, 060-8648, Japan

  • Investigational Site Number : 3920007

    Kagoshima, Kagoshima-ken, 890-0064, Japan

  • Investigational Site Number : 4100001

    Seoul, Seoul-teukbyeolsi, 03722, South Korea

  • Investigational Site Number : 4100002

    Yangsan, Gyeongsangnam-do, 50612, South Korea

  • Investigational Site Number : 5280001

    Amsterdam, 1105 AZ, Netherlands

  • Investigational Site Number : 5780001

    Bergen, 5021, Norway

  • Investigational Site Number : 6160001

    Krakow, 31-202, Poland

  • Investigational Site Number : 6160003

    Lodz, Lódzkie, 02-213, Poland

  • Investigational Site Number : 7240001

    Pontevedra, 36312, Spain

  • Investigational Site Number : 7240002

    Madrid, Madrid, Comunidad de, 28007, Spain

  • Investigational Site Number : 7240003

    Alicante, Alicante, 03010, Spain

  • Investigational Site Number : 7920001

    Ankara, 06560, Turkey (Türkiye)

  • Investigational Site Number : 7920002

    Istanbul, 34098, Turkey (Türkiye)

  • Investigational Site Number : 7920003

    İzmit, 41000, Turkey (Türkiye)

  • Investigational Site Number : 8260001

    London, London, City of, NW3 2QG, United Kingdom

  • Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004

    Fairfax, Virginia, 22030, United States

  • Maryam Banikazemi, MD- Site Number : 8400001

    Hawthorne, New York, 10532, United States

  • Renal Disease Research Institute- Site Number : 8400012

    Dallas, Texas, 75204, United States

  • University of Alabama -The Kirklin Clinic- Site Number : 8400010

    Birmingham, Alabama, 35233, United States

  • University of California Los Angeles Medical Center- Site Number : 8400008

    Los Angeles, California, 90095, United States

  • University of Utah Health Hospital- Site Number : 8400006

    Salt Lake City, Utah, 84132, United States

Conditions

Explore the condition pages connected to this study.

FABRY DISEASE