// App-Quantinova.ai

P1545 : DURABILITY OF HEMOGLOBIN RESPONSE AND REDUCTION IN TRANSFUSION BURDEN IS MAINTAINED OVER TIME IN PATIENTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT IN A LONG-TERM EXTENSION STUDY

Researchers

Presenter

  • Rachael F. Grace

Principal Investigators

  • Wilma Barcellini

  • Madeleine Verhovsek

  • Jennifer A. Rothman

  • Marta Morado Arias

  • D. Mark Layton

  • Oliver Andres

  • Frdric Galactros

  • Eduard J. van Beers

  • Koichi Onodera

  • Vip Viprakasit

  • Satheesh Chonat

  • John B. Porter

  • Penelope A. Kosinski

  • Peter Hawkins

  • Sarah Gheuens

  • Rengyi Xu

  • Hanny Al-Samkari

Medical Centers

  • Haematology Department, University College London Hospitals, London, UK

  • Hem

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

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

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

  • Agi

  • Department of Medical Oncology, University College London Hospital, London, United Kingdom

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

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

  • Haematology Department, University College London Hospitals, London, UK

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

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

  • McMaster University, Hamilton, ON, Canada

  • 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

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

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

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

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

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

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

  • Duk

  • Duke University Medical Center, Durham, NC

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

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

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

  • Siriraj Hospital,Mahidol University,Bangkok,Thaïlande

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

  • McMaster University, Hamilton, ON, Canada

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

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

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

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

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

  • Haematology Department, University College London Hospitals, London, UK

  • Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

  • Aflac Cancer & Blood Disorders Center of Children's Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, GA

  • Haematology Department,University College London Hospitals,London,Vereinigtes Königreich

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

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

  • Agios Pharmaceuticals Inc.,Cambridge,Estados Unidos

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

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

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

  • Aflac Cancer & Blood Disorders Center of Children's Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, GA

  • Department of Hematology,

  • Siriraj Hospital Mahidol University, Thailand

  • Siriraj Hospital,

  • Siriraj Hospital Mahidol University, Thailand

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

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

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

  • McMaster University,Hamilton, ON,Канада

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

  • McMaster University, Hamilton, ON, Canada

  • Siriraj Hospital Mahidol University, Thailand

  • Aflac Cancer and Blood Disorder Center of Children's Healthcare of Atlanta and Department of P

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

  • Duke University, Durham, North Carolina, United States

  • 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

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

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

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

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

  • Tohoku University Hospital,Sendai,Япония

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore

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

  • AGIOS Pharmaceuticals Inc,Cambridge,United States

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

Locations

  • United States

  • Denmark

  • Italy

  • Canada

  • Uruguay

  • Spain

  • France

  • United Kingdom

  • Germany

  • Netherlands

  • Japan

  • Thailand

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

Disease

  • Pyruvate kinase (PK) deficiency

Biomarkers

  • Red Blood Cells

Drug/Treatment

  • AG348

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Fixed Dose

  • Double Blind/Blinded

  • Open Label

  • Single Arm

  • Controlled

Phase

  • N/A

Study Id's

  • NCT03548220

  • NCT03559699

  • NCT03853798

Sponsors

  • N/A

Result

  • N/A

Copyright © 2026. QuantiNova All rights reserved