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4 recruiting studies matching your filters
Phase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Ste…
Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)Paroxysmal Nocturnal Hemoglobinuria (PNH)
National Heart, Lung, and Blood Institute (NHLBI)NCT03520647
Phase 3
An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age
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…
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Novartis PharmaceuticalsNCT06934967
Observational Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants expose…
Ultomiris-exposed Pregnant/ PostpartumPregnancyParoxysmal Nocturnal Hemoglobinuria (PNH)+3 more
Alexion Pharmaceuticals, Inc.NCT06312644
Phase 2
An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
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…
Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria
Apellis Pharmaceuticals, Inc.NCT04901936