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.

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

  • AKH - Medizinische Universität Wien

    Vienna, 1090, Austria

  • Alliance for Childhood Diseases, Cure 4 the Kids Foundation

    Las Vegas, Nevada, 89135, United States

  • Azienda Ospedaliera Universitaria "Policlinico - Vittorio Emanuele" (Presidio Ferrarotto Alessi)

    Catania, 90124, Italy

  • Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII)

    Bergamo, 24127, Italy

  • CHU Saint Etienne - Hôpital Nord

    Saint-Priest-en-Jarez Cedex, Pays de la Loire Region, 42270, France

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Complejo Hospitalario Universitario A Coruña

    A Coruña, La Coruña, 15006, Spain

  • Dipartimento di Medicina Traslazionale e di Precisione - "Sapienza" Universita di Roma

    Rome, 00161, Italy

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168, Italy

  • Hopital Claude Huriez - CHU Lille

    Lille, Nord, 59037, France

  • Hospital General Universitario de Alicante

    Alicante, 3010, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario Virgen del Rocio

    Seville, 41013, Spain

  • Hospital Universitario de Salamanca

    Salamanca, 37007, Spain

  • Hospital de Cruces

    Barakaldo, Vizcaya, 48903, Spain

  • Hyogo College of Medicine Hospital

    Nishinomiya-shi, Hyōgo, 663-8501, Japan

  • Hôpital Necker - Enfants Malades

    Paris, Paris, 75015, France

  • Hôpital Robert Debré - Paris

    Paris, 75019, France

  • Hôpital Saint-Antoine

    Paris, Paris, 75571, France

  • Instytut Hematologii i Transfuzjologii

    Warsaw, 02-776, Poland

  • Kyushu University Hospital

    Fukuoka, Fukuoka, 812-8582, Japan

  • Medical Hospital, Tokyo Medical and Dental University

    Bunkyō City, Tokyo-To, 113-8519, Japan

  • Ohio State Univ College Of Medicine

    Columbus, Ohio, 43210, United States

  • Royal Manchester Children's Hospital

    Manchester, M139WL, United Kingdom

  • Samodzielny Publiczny Dzieciecy Szpital Kliniczny

    Warsaw, 02-091, Poland

  • The Methodist Hospital

    Houston, Texas, 77030, United States

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • Universitaetsklinikum Jena

    Jena, Thuringia, 07747, Germany

  • University College London Hospitals

    London, Greater London, NW1 2PG, United Kingdom

  • University of Oklahoma

    Oklahoma City, Oklahoma, 73104, United States

  • Winship Cancer Institute

    Atlanta, Georgia, 30322, United States

Conditions

Explore the condition pages connected to this study.