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1679 : Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency

Researchers

Presenter

  • Hanny Al-Samkari

Principal Investigators

  • Rachael F. Grace

  • Andreas Glenthoej

  • Oliver Andres

  • Wilma Barcellini

  • Frdric Galactros

  • Kevin H.M. Kuo

  • D. Mark Layton

  • Marta Morado

  • Vip Viprakasit

  • Yan Dong

  • Feng Tai

  • Peter Hawkins

  • Sarah Gheuens

  • Chris Bowden

  • John B. Porter

  • Eduard van Beers

Medical Centers

  • Division of Rheumatology, Massachusetts General Hopsital, Boston, MA,

  • Dana-Farber Cancer Institute/Boston Children's Cancer and Blood Disorder Center, Boston, MA

  • Herlev Univ Hosp, Herlev, Denmark

  • Department of Paediatrics, University of Würzburg, University of Würzburg, Würzburg, Germany

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

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

  • Department of Surgery, University of Toronto, Toronto, ON, Canada

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

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

  • Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University,Bangkok,Thaïlande

  • ARIAD Pharmaceuticals, Inc., Cambridge, MA

  • Haematology Department, University College London Hospitals, London, United Kingdom, United Kingdom

  • Department of Hematology, Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands

Locations

  • United States

  • Denmark

  • Germany

  • Italy

  • France

  • Canada

  • United Kingdom

  • Spain

  • Thailand

  • Netherlands

Companies

  • Agios Pharmaceutical

Study Components

Therapeutic Area

  • Musculoskeletal

Disease

  • Pyruvate kinase (PK) deficiency

  • Osteoporosis

  • Osteopenia

Biomarkers

  • Pyruvate

  • Bone Mineral Density

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Double Blind/Blinded

  • Open Label

  • Single Arm

  • Controlled

Phase

  • III

Study Id's

  • NCT02476916

  • NCT03548220

  • NCT03559699

Sponsors

  • N/A

Result

  • N/A

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