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2600 : Proof of Concept for the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non–Transfusion-Dependent Thalassemia: Interim Results from an Ongoing, Phase 2, Open-Label, Multicenter Study

Researchers

Presenter

  • Kevin H.M. Kuo

Principal Investigators

  • D. Mark Layton

  • Ashutosh Lal

  • Hanny Al-Samkari

  • Feng Tai

  • Megan Lynch

  • Katrin Uhlig

  • Elliott P. Vichinsky

Medical Centers

  • Universtiy Health Network, Toronto, ON, Canada

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

  • UCSF Benioff Childrens Hospital Oakland, Oakland, CA

  • Division of Hematology, Massachusetts General Hospital, Dana-Farber Cancer Institute, Harvard University, Boston, MA

  • ARIAD Pharmaceuticals, Inc., Cambridge, MA

Locations

  • Canada

  • United Kingdom

  • United States

Companies

  • Agios Pharmaceutical

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Alpha-thalassemia

  • Non‐transfusion‐dependent β‐thalassaemia

Biomarkers

  • Pyruvate

  • Red Blood Cells

Drug/Treatment

  • N/A

Outcome

  • N/A


Study Design

  • Pharmacokinetics

  • Pharmacodynamics

  • Open Label

  • Multicenteric

Phase

  • II

Study Id's

  • NCT03692052

Sponsors

  • N/A

Result

  • N/A