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3278 : Safety of Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis: Results of the Primary Analysis of the Pivotal Phase 2/3 Study

Researchers

Presenter

  • Franco Locatelli

Principal Investigators

  • Michael B. Jordan

  • Carl Allen

  • Simone Cesaro

  • Carmelo Rizzari

  • Anupama Rao

  • Barbara Degar

  • Tim Garrington

  • Julian Sevilla

  • Maria Caterina Putti

  • Franca Fagioli

  • Martina Ahlmann

  • Jose-Luis Dapena Diaz

  • Michael Henry

  • Fabrizio De Benedetti

  • Alexei Grom

  • Cristina de Min

Medical Centers

  • Department of Pediatrics, University La Sapienza, Rome, Italy

  • Department of Pediatric Hematology and Oncology , IRCCS Ospedale Bambino Gesu, Rome , Italy

  • Division of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Univ. of Cincinnati, Cincinnati, OH, USA

  • Univ. of Cincinnati College of Medicine, Cincinnati, OH.

  • Department of Pediatric Medicine, Texas Childrens Hospital, Houston, TX

  • The Pediatric Hematology–Oncology, Woman and Child Hospital, Azienda Ospedaliera Universitaria Integrata, Verona, Italy

  • The Pediatric Hematology–Oncology Unit, Department of Pediatrics, University of Milano–Bicocca, Monza Brianza per il Bambino e la sua Mamma Foundation, Monza, Italy

  • Department of Haematology, Great Ormond Street Hospital For Children, London, United Kingdom

  • Department of Pediatric Hematology/Oncology, Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA

  • Center for Cancer and Blood Disorders, Childrens Hospital Colorado, Aurora, CO

  • The Departments of Pediatric Hematology–Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica, Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain

  • The Clinic of Pediatric Hematology–Oncology, University Hospital of Padova, Padua, Italy

  • Division of Pediatric Onco‐hematology, Regina Margherita‐S. Anna Hospital, Turin, Italy

  • Department of Pediatric Hematology and Oncology, University Children's Hospital Muenster, Muenster, Germany

  • Hospital Sant Joan de Deu de Barcelona, Barcelona, Spain

  • Center for Cancer & Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ

  • Division of Oncology, Bambino Gesu Childrens Hospital, Rome, Italy

  • Division of Nephrology, Cincinnati Childrens Hospital Medical Center, Cincinnati, OH

  • Swedish Orphan Biovitrum (Sobi) AG, Basel, Switzerland

Locations

  • Italy

  • United States

  • United Kingdom

  • Spain

  • Germany

  • Switzerland

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

Disease

  • Hemophagocytic lymphohistiocytosis

Biomarkers

  • N/A

Drug/Treatment

  • NI0501

Outcome

  • N/A


Study Design

  • Open Label

Phase

  • II/III

Study Id's

  • NCT01818492

Sponsors

  • N/A

Result

  • N/A