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S271 : ACTIVATE-T: A PHASE 3, OPEN-LABEL, MULTICENTER STUDY OF MITAPIVAT IN ADULTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED

Researchers

Presenter

  • Andreas Glenthj

Principal Investigators

  • Eduard J. van Beers

  • Hanny Al-Samkari

  • Vip Viprakasit

  • Kevin H. M. Kuo

  • Frdric Galactros

  • Satheesh Chonat

  • John Porter

  • Sarah Gheuens

  • Emily Xu

  • Peter Hawkins

  • Erin Zagadailov

  • Abdulafeez Oluyadi

  • Wilma Barcellini

Medical Centers

  • Division of Cardiology, The Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States,

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • Department of Haematology, University College London Cancer Institute,London,United Kingdom

  • Department of Hematology, Rigshospitalet, Copenhagen, Denmark

  • Unite des Maladies Genetiques du Globule Rouge, CHU Henri Mondor, Creteil, France

  • Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

  • Aflac Cancer & Blood Disorders Center of Children's Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, GA

  • IRCCS Ca Granda, Ospedale Maggiore Policlinico, Milano, Italy

  • Van Creveldkliniek - Department ofHaematology, University Medical Center Utrecht,Utrecht, Netherlands

  • Division of Dermatology, University of Toronto, Toronto,Canada

Locations

  • United States

  • United Kingdom

  • Denmark

  • France

  • Thailand

  • Italy

  • Netherlands

  • Canada

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

Biomarkers

  • Red Blood Cells

Drug/Treatment

  • AG348

Outcome

  • N/A

Study Design

  • Fixed Dose

  • Open Label

  • Multicenteric

  • Dose Escalation

Phase

  • III

Study Id's

  • NCT03559699

Sponsors

  • N/A

Result

  • N/A

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