New enzyme therapy shows promise in controlling rare blood clotting disorder
NCT ID NCT03393975
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a medicine called BAX 930 in 52 people born with severe TTP, a rare condition that causes dangerous blood clots. The medicine replaces a missing enzyme to prevent or treat sudden flare-ups. Participants received either BAX 930 or standard treatment for 6 months, then switched, followed by everyone getting BAX 930. The goal was to see if BAX 930 reduces flare-ups and is safe.
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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