New drug trial targets debilitating pain of rare genetic disease

NCT ID NCT05206773

Summary

This study is testing whether an investigational drug called venglustat can reduce nerve pain and abdominal pain in people with Fabry disease. It will involve about 122 participants aged 16 and older who are not currently on treatment. For 12 months, some will receive the drug and others a placebo, followed by an option for everyone to receive the drug for up to 4 more years.

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

  • Advent Health Orlando_Investigational Site Number: 8400008

    Orlando, Florida, 32804, United States

  • Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001

    Naples, 80138, Italy

  • Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003

    Palermo, 90127, Italy

  • Children's Hospital Of Pittsburgh- Site Number : 8400009

    Pittsburgh, Pennsylvania, 15213, United States

  • Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400013

    Cincinnati, Ohio, 45229-3026, United States

  • Cleveland Clinic Site Number : 8400016

    Cleveland, Ohio, 44195, United States

  • Emory Genetics- Site Number : 8400010

    Atlanta, Georgia, 30322, United States

  • Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002

    Monza, 20052, Italy

  • Fondazione Policlinico Universitario_Investigational Site Number: 3800004

    Roma, 00168, Italy

  • Fukuoka University Hospital_Investigational Site Number: 3920004

    Fukuoka-shi, Fukuoka, 814-0180, Japan

  • Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002

    La Rioja, F5300, Argentina

  • Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001

    Monterrey, Nuevo León, 64460, Mexico

  • Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • IRCCS Policlinico di Sant'Orsola_Investigational Site Number : 3800005

    Bologna, 40138, Italy

  • ISphinCS GmbH_Investigational Site Number: 2760004

    Hochheim am Main, 65239, Germany

  • Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 0760002

    São Paulo, 04020-041, Brazil

  • Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003

    Quilmes, B1878GEG, Argentina

  • Institutul Clinic Fundeni_Investigational Site Number: 6420001

    Bucharest, 22328, Romania

  • Investigational Site Number : 0360001

    Parkville, Victoria, 3050, Australia

  • Investigational Site Number : 1560006

    Zhengzhou, 450052, China

  • Investigational Site Number : 2500001

    Garches, 92380, France

  • Investigational Site Number : 2760005

    Mainz, 55131, Germany

  • Investigational Site Number : 3000003

    Athens, 115 28, Greece

  • Investigational Site Number : 3920005

    Kawasaki, Kanagawa, 215-0026, Japan

  • Investigational Site Number : 6160002

    Poznan, Greater Poland Voivodeship, 60-780, Poland

  • Investigational Site Number : 6160004

    Rzeszów, 35-301, Poland

  • Investigational Site Number : 7560001

    Zurich, 8091, Switzerland

  • Investigational Site Number : 7920001

    Ankara, 06560, Turkey (Türkiye)

  • Investigational Site Number : 7920002

    İzmit, 41000, Turkey (Türkiye)

  • Investigational Site Number : 7920004

    Malatya, 44280, Turkey (Türkiye)

  • Investigational Site Number : 8260003

    Salford, Manchester, M6 8HD, United Kingdom

  • Investigational Site Number: 0400001

    Vienna, 1090, Austria

  • Investigational Site Number: 2080001

    Copenhagen, 2100, Denmark

  • Investigational Site Number: 2460001

    Turku, FI-20520, Finland

  • Investigational Site Number: 2760001

    Würzburg, 97080, Germany

  • Investigational Site Number: 2760003

    München, 80336, Germany

  • Investigational Site Number: 5780001

    Bergen, 5021, Norway

  • Investigational Site Number: 6160003

    Wroclaw, 50-556, Poland

  • Investigational Site Number: 8260001

    Cambridge, Cambridgeshire, CB2 2QQ, United Kingdom

  • Investigational Site Number: 8260002

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

  • Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004

    Fairfax, Virginia, 22030, United States

  • M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003

    Calgary, Alberta, T2E7Z4, Canada

  • Medicine Dalhousie University_Investigational Site Number : 1240001

    Halifax, Nova Scotia, B3H 2Y9, Canada

  • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011

    Birmingham, Alabama, 35233, United States

  • No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003

    Shanghai, 200010, China

  • No.8, Xishiku Street, Xicheng District_Site Number: 1560001

    Beijing, 100034, China

  • No.85 South Jiefang road, Yingze District_Site Number: 1560004

    Taiyuan, 030001, China

  • Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_Investigational Site Number: 4840002

    Mexico City, 06700, Mexico

  • Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012

    Dallas, Texas, 75235, United States

  • The Jikei University Hospital_Investigational Site Number: 3920003

    Minato-ku, Tokyo, 105-8471, Japan

  • Tohoku University Hospital_Investigational Site Number: 3920001

    Sendai-shi, Miyagi, 980-8574, Japan

  • UCLA Medical Center_Investigational Site Number: 8400006

    Los Angeles, California, 90095, United States

  • University Health Network_Investigational Site Number : 1240005

    Toronto, Ontario, M5T 3L9, Canada

  • University Hospital of Heraklion_Investigational Site Number: 3000001

    Heraklion, 711 10, Greece

  • University Hospital of Ioannina_Investigational Site Number: 3000002

    Ioannina, 455 00, Greece

  • University Of Utah Health Sciences Center- Site Number : 8400005

    Salt Lake City, Utah, 84132, United States

  • University of California Irvine Medical Center- Site Number : 8400019

    Orange, California, 92868, United States

  • Westchester Medical Center Healthcare Corporation- Site Number : 8400001

    Hawthorne, New York, 10532, United States

Conditions

Explore the condition pages connected to this study.