New enzyme therapy shows promise in controlling rare blood clotting disorder

NCT ID NCT03393975

Completed Disease control Sponsor: Baxalta now part of Shire Source: ClinicalTrials.gov ↗

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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Conditions

The condition(s) this trial relates to.

congenital thrombotic thrombocytopenic purpura thrombotic thrombocytopenic purpura

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP)

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

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