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EP692 : EARLY-ONSET OSTEOPENIA AND OSTEOPOROSIS IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY

Researchers

Presenter

  • Hanny Al-Samkari

Principal Investigators

  • Rachael F. Grace

  • Andreas Glenthj

  • Oliver Andres

  • Wilma Barcellini

  • Frederic Galactros

  • Kevin H. M. Kuo

  • D. Mark Layton

  • Marta Morado Arias

  • Vip Viprakasit

  • Yan Dong

  • Feng Tai

  • Peter Hawkins

  • Sarah Gheuens

  • Chris Bowden

  • John Porter

  • Eduard J. van Beers

Medical Centers

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

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

  • University College London Hospitals NHS Trust, London, United Kingdom

  • Rheumatology, Massachusetts General Hospital, Boston, United States

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

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

  • Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

  • Department of Hematology, Rigshospitalet, Copenhagen, Denmark

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

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

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

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

Locations

  • Netherlands

  • Spain

  • United Kingdom

  • United States

  • France

  • Thailand

  • Denmark

  • Germany

  • Italy

  • Canada

Companies

  • N/A

Study Components

Therapeutic Area

  • Musculoskeletal

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

  • Osteoporosis

  • Osteopenia

Biomarkers

  • Bone Mineral Density

Drug/Treatment

  • AG348

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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