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A PIVOTAL, OPEN-LABEL, MULTICENTER STUDY TO ASSESS THE EFFICACY AND SAFETY OF BIVV009 IN PATIENTS WITH PRIMARY COLD AGGLUTININ DISEASE WHO HAVE A RECENT HISTORY OF BLOOD TRANSFUSION

Part APrimary Objective:• The primary objective of Part A is to determine whether BIVV009 administration results in a >= 2 g/dL increase in hemoglobin (Hgb) levels or increases Hgb to >= 12 g/dL and obviates the need for blood…

An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase II and Phase III studies with iptacopan

This study has been transitioned to CTIS with ID 2023-509843-28-00 check the CTIS register for the current data. The purpose of this study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to…

rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

* The purpose of this study is to to determine the safety profile of long-termCoversin treatment.* To observe the long term safety and efficacy of Coversin over periods in excess of 6 months* To assess the long term patient acceptability of Coversin…

A PHASE 3, OPEN-LABEL STUDY OF ALXN1210
IN CHILDREN AND ADOLESCENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

The primary objective of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, andefficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).