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756 : Phase 3 Study of Danicopan, an Oral Complement Factor D Inhibitor, As Add-on Therapy to a C5 Inhibitor in Patients with Paroxysmal Nocturnal Hemoglobinuria with Clinically Evident Extravascular Hemolysis

Researchers

Presenter

  • Austin G Kulasekararaj

Principal Investigators

  • Antonio Risitano

  • Jong Wook Lee

  • Mingjun Huang

  • Jun-Ichi Nishimura

  • Alejandra Ramirez-Santiago

  • Robert Brodsky

Medical Centers

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

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

  • Surgery, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Korea, Republic of

  • Kolltan Pharmaceuticals, New Haven, CT

  • Kinki University, Osaka, Japan

  • Division of Gastroenterology, Johns Hopkins University School of Medicine, Baltimore, MD

Locations

  • United Kingdom

  • Italy

  • Republic of South Korea

  • United States

  • Japan

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Paroxysmal nocturnal hemoglobinuria

  • Aplastic anemia

  • Anemia

Biomarkers

  • Alanine transaminase

  • Lactate dehydrogenase

  • Red Blood Cells

Drug/Treatment

  • N/A

Outcome

  • N/A


Study Design

  • Randomized

  • Placebo Control

  • Double Blind/Blinded

  • Controlled

Phase

  • III

Study Id's

  • NCT04469465

Sponsors

  • N/A

Result

  • N/A