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P1522 : LONG-TERM EFFICACY AND SAFETY OF THE ORAL PYRUVATE KINASE ACTIVATOR MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

Researchers

Presenter

  • Kevin H. M. Kuo

Principal Investigators

  • D. Mark Layton

  • Ashutosh Lal

  • Hanny Al-Samkari

  • Penelope A. Kosinski

  • Bo Tong

  • Megan Lynch

  • Katrin Uhlig

  • Elliott P. Vichinsky

Medical Centers

  • Division of Hematology,UCSF Benioff Children's Hospital Oakland,Oakland, CA,United States

  • Division of Hematology,UCSF Benioff Chil

  • Division of Hematology,UCSF Benioff Children's Hospital Oakland,Oakland, CA,Vereinigte Staaten

  • Massachusetts General Hospital Cancer Center/Harvard Medical School,Boston, MA,Vereinigte Staaten

  • Division of Hematology,UCSF Benioff Children's Hospital Oakland,Oakland, CA,Vereinigte Staaten

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

  • Division of Hematol

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

  • Agi

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

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

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

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

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • Massachusetts General Hospital Cancer Center/Harvard Medical School,Boston, MA,Vereinigte Staaten

  • Division of Hematology,UCSF Benioff Children's Hospital Oakland,Oakland, CA,Vereinigte Staaten

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • Massachusetts General Hospital Cancer Center/Harvard Medical School,Boston, MA,Vereinigte Staaten

  • Massachusetts General Hospital/Harvard Medical School, Boston, Massachusetts, United States

Locations

  • Canada

  • United Kingdom

  • United States

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Ophthalmology (OP)

Disease

  • Beta-thalassemia

Biomarkers

  • Red Blood Cells

Drug/Treatment

  • Adenosine-5'-triphosphate

Outcome

  • N/A


Study Design

  • N/A

Phase

  • II

Study Id's

  • NCT03692052

Sponsors

  • N/A

Result

  • N/A