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EP590 : PHARMACOKINETICS, PHARMACODYNAMICS, EFFICACY AND SAFETY OF RAVULIZUMAB IN CHILDREN AND ADOLESCENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: INTERIM ANALYSIS OF A PHASE 3, OPEN-LABEL STUDY

Researchers

Presenter

  • Alexander Kulagin

Principal Investigators

  • Satheesh Chonat

  • Alexey Maschan

  • Marije Bartels

  • Jochen Buechner

  • Rowena Punzalan

  • Michael Richards

  • Masayo Ogawa

  • Eden Hicks

  • Ji Yu

  • Andr Baruchel

  • Austin G Kulasekararaj

Medical Centers

  • Department of Pediatrics,Emory University School of Medicine, and Aflac Cancer and Blood Disorders Center Children’s Healthcare of Atlanta,Atlanta,United States

  • Department of Pediatric Oncology and Hematology, Essen University Hospital, Essen, Germany

  • Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI.

  • Université de Paris, Pediatric Hematology Department, Hôpital Robert-Debré (APHP),Paris,France

  • Blood Center of Wisconsin, Milwaukee, WI

  • Department of Paediatric Haematology, Sheffield Childrens Hospital, Sheffield, United Kingdom

  • Alexion Pharmaceuticals, Inc., Boston, United States

  • 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

  • Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation

  • Pediatric Infectious Diseases,Wilhelmina Childrens Hospital University Medical Center Utrecht, Utrecht,Netherlands

Locations

  • United States

  • Norway

  • France

  • United Kingdom

  • Russia

  • Netherlands

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Paroxysmal nocturnal hemoglobinuria

Biomarkers

  • Lactate dehydrogenase

Drug/Treatment

  • Eculizumab

Outcome

  • N/A


Study Design

  • Pharmacokinetics

  • Pharmacodynamics

  • Open Label

  • Single Arm

  • Multicenteric

Phase

  • III

Study Id's

  • NCT03406507

Sponsors

  • N/A

Result

  • N/A