Extended hope: Long-Term drug study for rare blood disorder
NCT ID NCT03853798
Summary
This study aimed to understand the long-term safety and effectiveness of the drug mitapivat for adults with pyruvate kinase deficiency, a rare genetic blood disorder. It was an extension study for 90 participants who had already benefited from mitapivat in earlier trials. The main goal was to monitor side effects and see if the drug continued to help control the disease over a longer period.
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Contacts and locations
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Locations
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AORN Cardarelli
Napoli, 00165, Italy
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Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CHU Hopitaux de Bordeaux - Hôpital Saint-André
Bordeaux, 33000, France
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CHU Hôpital Henri Mondor
Créteil, 94010, France
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Centre Hospitalier Universitaire Vaudois (CHUV)
Vaud (Lausanne), 1011, Switzerland
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Charite - UB - CVK - Medizinische Klinik
Berlin, 10117, Germany
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Emory-Children's Center
Atlanta, Georgia, 30322, United States
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Faculty of Medicine Siriraj Hospital
Bangkok, 10700, Thailand
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Hacettepe University Faculty of Medicine
Ankara, 06100, Turkey (Türkiye)
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Herlev University Hospital
Herlev, 2730, Denmark
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Hospital La Timone
Marseille, 13385, France
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Hospital Universitario La Paz
Madrid, 28046, Spain
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Hospital. U. Vall d'Hebron
Barcelona, 08035, Spain
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Houston Methodist Research Institute
Houston, Texas, 77030, United States
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Htal Clínico Universitario Virgen de la Arrixaca.
Murcia, 30120, Spain
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Imperial College Healthcare NHS Trust
London, W2 INY, United Kingdom
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Indiana Hemophilia & Thrombosis Center Inc.
Indianapolis, Indiana, 46260, United States
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Institut Universitaire du Cancer de Toulouse - Oncopole
Toulouse, 31100, France
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Kansai Medical University, Dep. of Pediatrics, Hirakata Hospital
Osaka, 573-1010, Japan
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Kyoto Katsura Hospital
Kyoto, 615-8256, Japan
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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McMaster University
Hamilton, Ontario, L8N3Z5, Canada
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Mie University Hospital
Tsu, Mie-ken, 514-8507, Japan
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Osp Maggiore Policlinico Milano
Milan, 20122, Italy
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Ospedale Galliera
Genova, 16128, Italy
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Seattle Cancer Care Alliance
Seattle, Washington, 98195, United States
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St James's Hospital
Dublin, Ireland
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Toho University Omori Medical Center
Tokyo, 8541, Japan
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Tohoku University Hospital
Sendai, Miyagi, 980-8574, Japan
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UCSF Benioff Children's Hospital, Oakland
Oakland, California, 95609, United States
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UNICAMP - Hemocentro
São Paulo, 13083-878, Brazil
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University College London
London, WC1E 6BT, United Kingdom
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University of Utah
Salt Lake City, Utah, 84113, United States
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Università della Campania "Luigi Vanvitelli"
Napoli, 80318, Italy
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Universitätsklinik Würzburg
Würzburg, 97080, Germany
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Van Creveldkliniek
Utrecht, 3508 GA, Netherlands
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Wayne State University School of Medicine
Detroit, Michigan, 48201, United States
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Yeungnam University Hospital
Daegu, 705-703, South Korea
Conditions
Explore the condition pages connected to this study.