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S174 : FORTY-EIGHT WEEK EFFICACY AND SAFETY OF PEGCETACOPLAN IN ADULT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AND SUBOPTIMAL RESPONSE TO PRIOR ECULIZUMAB TREATMENT

Researchers

Presenter

  • Regis Peffault de Latour

Principal Investigators

  • Jeffrey Szer

  • Ilene Weitz

  • Alexander Rth

  • Britta Hchsmann

  • Jens Panse

  • Kensuke Usuki

  • Morag Griffin

  • Jean-Jacques Kiladijan

  • Carlos M. de Castro

  • Hisakazu Nishimori

  • Lisa Tan

  • Mohammed Al-Adhami

  • Pascal Deschatelets

  • Cedric Francois

  • Federico Grossi

  • Antonio Risitano

  • Peter Hillmen

Medical Centers

  • Department of Hematology and Stem Cell Transplantation,University Hospital Essen, University of Duisburg-Essen,Essen,Germany

  • Department of Clinical Medicine and Surgery, Federico II University, Naples, Italy

  • Department of Clinical Haematology,Peter MacCallum Cancer Centre &amp

  • Hematology and BMT Unit,AORN San Giuseppe Moscati,Avellino,Italy

  • Royal Melbourne Hospital, Melbourne, Australia

  • Jane Anne Nohl Division of Hematology,Keck-USC School of Medicine,Los Angeles, CA,United States

  • French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Assistance Publique – Hôpitaux de Paris, Université de Paris, Paris, France

  • Department of Obstetrics and Gynecology, Tokai Univeresity Hospital, Kanagawa, Japan

  • University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics,German Red Cross Blood Transfusion Service and University Hospital Ulm, Institute of Transfusion Medicine,Ulm,Germany

  • Department of Oncology, Hematology and Stem Cell Transplantation, University Hospital of the RWTH Aachen, Aachen, Germany

  • Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan

  • Lisa Tan Pharma Consulting, Cambridge, United Kingdom

  • Assistance Publique Hopitaux de Paris,

  • Université de Paris,Paris,France

  • Centre d'Investigations Cliniques,Hôpital Saint-Louis

  • Duke University, Durham, North Carolina, United States

  • Apellis Pharmaceuticals, Inc.,Waltham,United States

Locations

  • Germany

  • Italy

  • United Kingdom

  • Australia

  • United States

  • France

  • Japan

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Blood/haematological Diseases

Disease

  • Paroxysmal nocturnal hemoglobinuria

Biomarkers

  • Complement Factor B

  • Lactate dehydrogenase

Drug/Treatment

  • Eculizumab

Outcome

  • N/A

Study Design

  • Randomized

  • Open Label

Phase

  • III

Study Id's

  • NCT03500549

Sponsors

  • N/A

Result

  • N/A

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