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PB1476 : PH3 STUDY OF EFFICACY AND SAFETY OF IPTACOPAN (LNP023), AN ORAL COMPLEMENT FACTOR B INHIBITOR, IN PTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AND RESIDUAL ANEMIA DESPITE ANTI-C5 ANTIBODY TREATMENT

Researchers

Presenter

  • Antonio M. Risitano

Principal Investigators

  • Austin Kulasekararaj

  • Carlos M. De Castro

  • Shinji Nakao

  • Phillip Scheinberg

  • Rafael Levitch

  • Georgina Bermann

  • Marion Dahlke

  • Rgis Peffault de Latour

Medical Centers

  • Hospital Beneficencia Portuguesa, Sao Paulo, Brazil

  • University of Naples Federico II, Naples, Italy

  • AORN Moscati,Avellino,Italy

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

  • Novartis Pharma Corp, Basel, Switzerland

  • Kings College London, London, United Kingdom

  • Aphp Saint-Louis Hospital, Paris, France

  • Duke University, Durham, North Carolina, United States

  • Kanazawa University, Kanazawa, Japan

Locations

  • Brazil

  • Italy

  • United Kingdom

  • Switzerland

  • France

  • United States

  • Japan

Companies

  • N/A

Study Components

Therapeutic Area

  • Pulmonary/Respiratory Diseases

  • Cardiovascular (CVS)

  • Blood/haematological Diseases

Disease

  • Paroxysmal nocturnal hemoglobinuria

  • Venous thrombosis

  • Anemia

Biomarkers

  • Red Blood Cells

Drug/Treatment

  • Eculizumab

Outcome

  • N/A


Study Design

  • Randomized

  • Superiority

  • Controlled

Phase

  • NA

Study Id's

  • NCT03439839

  • NCT04558918

Sponsors

  • N/A

Result

  • N/A