Browse Studies — Arctuva

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)

Estimated

~$10 – $5,625

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)

Estimated

~$10 – $8,750

Novartis PharmaceuticalsNCT06934967

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

Estimated

~$10 – $8,750

Apellis Pharmaceuticals, Inc.NCT04901936

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

Estimated

~$10 – $1,050

Alexion Pharmaceuticals, Inc.NCT06312644