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S270 : ACTIVATE: A PHASE 3, RANDOMIZED, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF MITAPIVAT IN ADULTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE NOT REGULARLY TRANSFUSED

Researchers

Presenter

  • Hanny Al-Samkari

Principal Investigators

  • Frdric Galactros

  • Andreas Glenthj

  • Jennifer A. Rothman

  • Oliver Andres

  • Rachael F. Grace

  • Marta Morado Arias

  • D. Mark Layton

  • Koichi Onodera

  • Madeleine Verhovsek

  • Wilma Barcellini

  • Emily Xu

  • Peter Hawkins

  • Erin Zagadailov

  • Sarah Gheuens

  • Eduard J. van Beers

Medical Centers

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

  • Van Creveldkliniek Department of Internal Medicine,University Medical Center Utrecht,Utrecht,Pays-bas

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

  • Hematology Department, Hospital Universitario La Princesa, Madrid, Spain

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • Department of Paediatrics,University of Wurzburg,Wurzburg,Germany

  • Department of Hematology, Rigshospitalet, Copenhagen, Denmark

  • 4Dana-Farber/Boston Children's Cancer and Blood Disorders Center,Boston,United States

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

  • McMaster University, Hamilton, ON, Canada

  • Duke University, Durham, North Carolina, United States

  • St Marys Hospital, Imperial CollegeHealthcare NHS Trust, London, UK

Locations

  • United States

  • Netherlands

  • France

  • Spain

  • Germany

  • Japan

  • Denmark

  • Italy

  • Canada

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

Biomarkers

  • Lactate dehydrogenase

Drug/Treatment

  • AG348

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Fixed Dose

  • Double Blind/Blinded

  • Multicenteric

  • Controlled

  • Dose Escalation

Phase

  • III

Study Id's

  • NCT03548220

Sponsors

  • N/A

Result

  • N/A

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