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P1565 : MITAPIVAT IMPROVES INEFFECTIVE ERYTHROPOIESIS AND REDUCES IRON OVERLOAD IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY

Researchers

Presenter

  • Eduard J. van Beers

Principal Investigators

  • Hanny Al-Samkari

  • Rachael F. Grace

  • Wilma Barcellini

  • Andreas Glenthj

  • Penelope A. Kosinski

  • Rengyi Xu

  • John B. Porter

  • Kevin H. M. Kuo

Medical Centers

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Haematology Department, University College London Hospitals, London, UK

  • Haematology Department, University College London Hospitals, London, UK

  • Dana-Farber/Boston Children's Cancer...

  • Department of Hematology, University Hospital Rigshospitalet, Copenhagen, Denmark

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

  • Haematology Department,University College London Hospitals,London,Vereinigtes Königreich

  • Dana-Farber/Boston Childrens Hospital Cancer and Blood Disorders Center, Boston, MA

  • Division of Hematol

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

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

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Agi

  • Division of Gynecologic Oncology, Massachusetts General Hospital, Harvard Medical School, Boston, MA

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

  • Department of Medical Oncology, University College London Hospital, London, United Kingdom

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

  • Department of Hematology, University Hospital Rigshospitalet, Copenhagen, Denmark

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

  • Haematology Department, University College London Hospitals, London, UK

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

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

  • Department of Hematology, University Hospital Rigshospitalet, Copenhagen, Denmark

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

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

  • Department of Hematology,

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

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

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

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

  • Division of General Surgery, University of Toronto, Toronto, ON, Canada

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

Locations

  • Netherlands

  • United States

  • Italy

  • Denmark

  • United Kingdom

  • Canada

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

  • Hemochromatosis

Biomarkers

  • TIMP Metallopeptidase Inhibitor 1

Drug/Treatment

  • Erythropoietin

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Double Blind/Blinded

  • Open Label

  • Single Arm

  • Controlled

Phase

  • III

Study Id's

  • NCT03548220

  • NCT03559699

  • NCT03853798

Sponsors

  • N/A

Result

  • N/A

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