New therapy offers hope for controlling rare, Life-Threatening blood disorder
NCT ID NCT03393975
Summary
This study tested a new medicine called BAX 930 for people born with a severe, inherited blood clotting disorder called congenital TTP. The goal was to see if BAX 930, which replaces a missing enzyme, could prevent or treat dangerous clotting events better than the standard treatment. In this completed Phase 3 trial, 52 participants received both the new treatment and the standard treatment in different periods to compare their effectiveness and safety.
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Contacts and locations
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Locations
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AKH - Medizinische Universität Wien
Vienna, 1090, Austria
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Alliance for Childhood Diseases, Cure 4 the Kids Foundation
Las Vegas, Nevada, 89135, United States
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Azienda Ospedaliera Universitaria "Policlinico - Vittorio Emanuele" (Presidio Ferrarotto Alessi)
Catania, 90124, Italy
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Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII)
Bergamo, 24127, Italy
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CHU Saint Etienne - Hôpital Nord
Saint-Priest-en-Jarez Cedex, Pays de la Loire Region, 42270, France
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Complejo Hospitalario Universitario A Coruña
A Coruña, La Coruña, 15006, Spain
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Dipartimento di Medicina Traslazionale e di Precisione - "Sapienza" Universita di Roma
Rome, 00161, Italy
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 168, Italy
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Hopital Claude Huriez - CHU Lille
Lille, Nord, 59037, France
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Hospital General Universitario de Alicante
Alicante, 3010, Spain
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Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
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Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
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Hospital Universitario de Salamanca
Salamanca, 37007, Spain
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Hospital de Cruces
Barakaldo, Vizcaya, 48903, Spain
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Hyogo College of Medicine Hospital
Nishinomiya-shi, Hyōgo, 663-8501, Japan
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Hôpital Necker - Enfants Malades
Paris, Paris, 75015, France
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Hôpital Robert Debré - Paris
Paris, 75019, France
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Hôpital Saint-Antoine
Paris, Paris, 75571, France
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Instytut Hematologii i Transfuzjologii
Warsaw, 02-776, Poland
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Kyushu University Hospital
Fukuoka, Fukuoka, 812-8582, Japan
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Medical Hospital, Tokyo Medical and Dental University
Bunkyō City, Tokyo-To, 113-8519, Japan
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Ohio State Univ College Of Medicine
Columbus, Ohio, 43210, United States
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Royal Manchester Children's Hospital
Manchester, M139WL, United Kingdom
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Samodzielny Publiczny Dzieciecy Szpital Kliniczny
Warsaw, 02-091, Poland
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The Methodist Hospital
Houston, Texas, 77030, United States
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Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
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Universitaetsklinikum Jena
Jena, Thuringia, 07747, Germany
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University College London Hospitals
London, Greater London, NW1 2PG, United Kingdom
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University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
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Winship Cancer Institute
Atlanta, Georgia, 30322, United States
Conditions
Explore the condition pages connected to this study.