There are currently 3 active clinical trials seeking participants for Paroxysmal Nocturnal Hemoglobinuria (PNH) research studies. The states with the highest number of trials for Paroxysmal Nocturnal Hemoglobinuria (PNH) participants are California, Ontario, North Carolina and Maryland.
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Recruiting
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants. Objectives: To see how safely and effectively SAA, MDS and PNH are treated using per... Read More
Gender:
ALL
Ages:
Between 4 years and 75 years
Trial Updated:
08/15/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Recruiting
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).
Gender:
ALL
Ages:
Between 12 years and 17 years
Trial Updated:
06/06/2025
Locations: Children's Hospital of Atlanta, Atlanta, Georgia +11 locations
Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Recruiting
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.
Gender:
FEMALE
Ages:
All
Trial Updated:
01/06/2025
Locations: North American call center (NACC), Boston, Massachusetts
Conditions: Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), Generalized Myasthenia Gravis (gMG), Neuromyelitis Optica Spectrum Disorder (NMOSD)