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PNH
Clinical trial pipeline · Data from ClinicalTrials.gov
See which PNH trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which PNH trials you may qualify forBackground: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem…
This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs". Researchers are look…
The first-in-human Phase 1/Phase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in…
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Da…
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed…
The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whethe…
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to…
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatm…
The complement system is an important component of the innate immune system. Abnormal activation, inadequate regulation and control of the complement system, as…