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2575 : Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension

Researchers

Presenter

  • Antonio Risitano

Principal Investigators

  • Jun-Ho Jang

  • Lee Gyeong-Won

  • Wanchai Wanachiwanawin

  • Hubert Schrezenmeier

  • Yuji Yonemura

  • Talha Munir

  • Rodrigo Pavani

  • Jimmy Wang

  • Alexander D. Kulagin

  • Austin G Kulasekararaj

  • Flore Sicre de Fontbrune

  • Alexander Rth

Medical Centers

  • Department of clinical medicine and surgery, University of Naples Federico II, Naples, Italy

  • Sungkyunkwan university school of medicine, samsung medical center, Seoul, Korea (the Republic of)

  • Division of Hematology/Oncology, Department of Internal Medicine, Gyeongsang National University Hospital, Gyeongsang National University School of Medicine, Jinju, South Korea

  • Department of Internal Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand

  • Institute of Transfusion Medicine, University of Ulm, and Institute for Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen and University Hospital Ulm,Ulm,Germany

  • Kumamoto University School of Medicine, Kumamoto, Japan

  • St. James's University Hospital, Leeds, United Kingdom

  • Vertex Pharmaceuticals Inc., Boston, MA

  • Vertex Pharmaceuticals Inc., Boston, MA

  • RM Gorbacheva Research Institute, Pavlov University,St Petersburg,Russia

  • King’s College Hospital, National Institute of Health Research/Wellcome King’s Clinical Research Facility and King’s College London,London,United Kingdom

  • Centre de Référence Aplasie Médullaire, Service d'Hématologie Greffe,Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis,Paris,France

  • Department of Surgery, University Hospital Essen, Essen, Germany

Locations

  • Italy

  • Republic of South Korea

  • Thailand

  • Germany

  • Japan

  • United Kingdom

  • United States

  • Russia

  • France

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Paroxysmal nocturnal hemoglobinuria

Biomarkers

  • Lactate dehydrogenase

  • Tyrosine 3-Monooxygenase/Tryptophan 5-Monooxygenase Activation Protein Epsilon

  • Bone Mineral Density

Drug/Treatment

  • Eculizumab

Outcome

  • N/A

Study Design

  • Randomized

  • Open Label

  • Non Inferiority

  • Multicenteric

  • Controlled

Phase

  • III

Study Id's

  • NCT02946463

Sponsors

  • N/A

Result

  • N/A

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