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.
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Contacts and locations
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Locations
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Advent Health Orlando_Investigational Site Number: 8400008
Orlando, Florida, 32804, United States
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Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001
Naples, 80138, Italy
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Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003
Palermo, 90127, Italy
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Children's Hospital Of Pittsburgh- Site Number : 8400009
Pittsburgh, Pennsylvania, 15213, United States
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Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400013
Cincinnati, Ohio, 45229-3026, United States
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Cleveland Clinic Site Number : 8400016
Cleveland, Ohio, 44195, United States
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Emory Genetics- Site Number : 8400010
Atlanta, Georgia, 30322, United States
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Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002
Monza, 20052, Italy
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Fondazione Policlinico Universitario_Investigational Site Number: 3800004
Roma, 00168, Italy
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Fukuoka University Hospital_Investigational Site Number: 3920004
Fukuoka-shi, Fukuoka, 814-0180, Japan
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Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002
La Rioja, F5300, Argentina
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Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001
Monterrey, Nuevo León, 64460, Mexico
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Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
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IRCCS Policlinico di Sant'Orsola_Investigational Site Number : 3800005
Bologna, 40138, Italy
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ISphinCS GmbH_Investigational Site Number: 2760004
Hochheim am Main, 65239, Germany
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Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 0760002
São Paulo, 04020-041, Brazil
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Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003
Quilmes, B1878GEG, Argentina
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Institutul Clinic Fundeni_Investigational Site Number: 6420001
Bucharest, 22328, Romania
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Investigational Site Number : 0360001
Parkville, Victoria, 3050, Australia
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Investigational Site Number : 1560006
Zhengzhou, 450052, China
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Investigational Site Number : 2500001
Garches, 92380, France
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Investigational Site Number : 2760005
Mainz, 55131, Germany
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Investigational Site Number : 3000003
Athens, 115 28, Greece
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Investigational Site Number : 3920005
Kawasaki, Kanagawa, 215-0026, Japan
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Investigational Site Number : 6160002
Poznan, Greater Poland Voivodeship, 60-780, Poland
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Investigational Site Number : 6160004
Rzeszów, 35-301, Poland
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Investigational Site Number : 7560001
Zurich, 8091, Switzerland
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Investigational Site Number : 7920001
Ankara, 06560, Turkey (Türkiye)
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Investigational Site Number : 7920002
İzmit, 41000, Turkey (Türkiye)
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Investigational Site Number : 7920004
Malatya, 44280, Turkey (Türkiye)
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Investigational Site Number : 8260003
Salford, Manchester, M6 8HD, United Kingdom
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Investigational Site Number: 0400001
Vienna, 1090, Austria
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Investigational Site Number: 2080001
Copenhagen, 2100, Denmark
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Investigational Site Number: 2460001
Turku, FI-20520, Finland
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Investigational Site Number: 2760001
Würzburg, 97080, Germany
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Investigational Site Number: 2760003
München, 80336, Germany
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Investigational Site Number: 5780001
Bergen, 5021, Norway
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Investigational Site Number: 6160003
Wroclaw, 50-556, Poland
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Investigational Site Number: 8260001
Cambridge, Cambridgeshire, CB2 2QQ, United Kingdom
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Investigational Site Number: 8260002
London, London, City of, NW3 2QG, United Kingdom
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Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004
Fairfax, Virginia, 22030, United States
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M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003
Calgary, Alberta, T2E7Z4, Canada
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Medicine Dalhousie University_Investigational Site Number : 1240001
Halifax, Nova Scotia, B3H 2Y9, Canada
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Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011
Birmingham, Alabama, 35233, United States
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No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003
Shanghai, 200010, China
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No.8, Xishiku Street, Xicheng District_Site Number: 1560001
Beijing, 100034, China
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No.85 South Jiefang road, Yingze District_Site Number: 1560004
Taiyuan, 030001, China
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Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_Investigational Site Number: 4840002
Mexico City, 06700, Mexico
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Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012
Dallas, Texas, 75235, United States
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The Jikei University Hospital_Investigational Site Number: 3920003
Minato-ku, Tokyo, 105-8471, Japan
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Tohoku University Hospital_Investigational Site Number: 3920001
Sendai-shi, Miyagi, 980-8574, Japan
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UCLA Medical Center_Investigational Site Number: 8400006
Los Angeles, California, 90095, United States
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University Health Network_Investigational Site Number : 1240005
Toronto, Ontario, M5T 3L9, Canada
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University Hospital of Heraklion_Investigational Site Number: 3000001
Heraklion, 711 10, Greece
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University Hospital of Ioannina_Investigational Site Number: 3000002
Ioannina, 455 00, Greece
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University Of Utah Health Sciences Center- Site Number : 8400005
Salt Lake City, Utah, 84132, United States
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University of California Irvine Medical Center- Site Number : 8400019
Orange, California, 92868, United States
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Westchester Medical Center Healthcare Corporation- Site Number : 8400001
Hawthorne, New York, 10532, United States
Conditions
Explore the condition pages connected to this study.