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P1548 : LONG-TERM TREATMENT WITH ORAL MITAPIVAT IS ASSOCIATED WITH NORMALIZATION OF HEMOGLOBIN LEVELS IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY

Researchers

Presenter

  • Wilma Barcellini

Principal Investigators

  • Rachael F. Grace

  • Hanny Al-Samkari

  • Andreas Glenthj

  • Jennifer A. Rothman

  • Marta Morado Arias

  • D. Mark Layton

  • Oliver Andres

  • Peter Hawkins

  • Feng Tai

  • Jaime Morales-Arias

  • Eduard J. van Beers

Medical Centers

  • Duke University Medical Center,Durham, NC,Verenigde Staten

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

  • Duke University Medical Center,Durham, NC,Stati Uniti

  • Duke University Medical Center,Durham, NC,Verenigde Staten

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

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

  • Hem

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

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

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

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

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

  • Duke University, Durham, North Carolina, United States

  • Duk

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

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

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

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Vereinigtes Königreich

  • Agi

  • Duke University Medical Center,Durham, NC,Stati Uniti

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

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Royaume-uni

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

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

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

  • Duke University Medical Center, Durham, NC

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

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

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Royaume-uni

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

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

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

  • Department of Hematology,

  • Hammersmith Hospital,Imperial College Healthcare NHS Trust,London,Vereinigtes Königreich

  • 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

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

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

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

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

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

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • Department of Paediatrics,University of Würzburg,Würzburg,Duit

Locations

  • Italy

  • United States

  • Denmark

  • Uruguay

  • Spain

  • France

  • United Kingdom

  • Germany

  • Netherlands

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

Biomarkers

  • N/A

Drug/Treatment

  • AG348

Outcome

  • N/A

Study Design

  • Randomized

  • Fixed Dose

  • Double Blind/Blinded

  • Controlled

Phase

  • III

Study Id's

  • NCT03548220

  • NCT03853798

Sponsors

  • N/A

Result

  • N/A

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