New hope for babies with severe bleeding disorder
NCT ID NCT04431726
Summary
This study is testing a preventive drug called emicizumab in babies under 1 year old who have severe hemophilia A, a serious genetic bleeding disorder. The main goal is to see if regular injections can safely reduce the number of bleeding episodes these infants experience. After an initial year, participants can continue receiving the drug for up to 7 years of follow-up to monitor long-term safety and joint health.
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Contacts and locations
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Locations
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AORN Santobono Pausilipon
Naples, Campania, 80123, Italy
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AOU Careggi
Florence, Tuscany, 50134, Italy
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AOU di Parma
Parma, Emilia-Romagna, 43126, Italy
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Adana Acibadem Hospital
Adana, 01130, Turkey (Türkiye)
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Charlotte Maxeke Johannesburg Hospital
Johannesburg, 2193, South Africa
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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Cliniques Universitaires St-Luc
Brussels, 1200, Belgium
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Ege University, School of Medicine
Izmir, 35100, Turkey (Türkiye)
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Groupe Hospitalier Necker Enfants Malades
Paris, 75015, France
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Hacettepe University Medical Faculty
Ankara, 06100, Turkey (Türkiye)
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Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950, Spain
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Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
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Hospital Universitario la Paz
Madrid, 28046, Spain
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Hospital das Clínicas Faculdades Médicas de Ribeirão Preto
Ribeirão Preto, São Paulo, 14051-140, Brazil
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Hämophilie-Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546, Germany
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IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122, Italy
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Medizinische Universität Wien
Vienna, 1090, Austria
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Ondokuz Mayis Univ. Med. Fac.
Samsun, 55139, Turkey (Türkiye)
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Perth Children's Hospital
Nedlands, Western Australia, 6009, Australia
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Queen Elizabeth University Hospital
Glasgow, G51 4TF, United Kingdom
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Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Sheba Medical Center - National Hemophilia Center
Tel Litwinsky, 5262100, Israel
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The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Tulane University Health Sciences Center
New Orleans, Louisiana, 70112, United States
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UZ Leuven Gasthuisberg
Leuven, 3000, Belgium
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University of Colorado Denver, Children's Hospital
Aurora, Colorado, 80045, United States
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University of Michigan
Ann Arbor, Michigan, 48109-0934, United States
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Universitätsklinikum Bonn
Bonn, 53127, Germany
Conditions
Explore the condition pages connected to this study.