8 recruiting studies matching your filters
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Ste…
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 wheth…
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pe…
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiop…
The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history st…
The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH cov…
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants expose…
The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced b…