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Paroxysmal Hemoglobinuria clinical trials recruiting in the U.S.
9 studies for Paroxysmal Hemoglobinuria are recruiting participants at sites across the United States.
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Recently updated studies
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
RecruitingPhase 2
Ages 4 to 80 · All sexes · Updated Sep 22, 2026 · NCT01174108Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
RecruitingPhase 2
Ages 4 to 75 · All sexes · Updated Sep 8, 2026 · NCT03520647Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
RecruitingPhase 3
Ages 2 to 18 · All sexes · Updated Aug 7, 2026 · NCT06934967A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
RecruitingPhase 2
Ages 12 to 17 · All sexes · Updated Jun 26, 2026 · NCT04901936MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
RecruitingPhase 2
Ages 0 to 75 · All sexes · Updated Jun 3, 2026 · NCT06412497Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
RecruitingObservational study
All ages · Women · Updated Apr 16, 2026 · NCT06312644Home Reported Outcomes in PNH
RecruitingObservational study
Ages 18 to 99 · All sexes · Updated Aug 27, 2025 · NCT06411626Global PNH Patient Registry
RecruitingObservational study
All ages · All sexes · Updated Feb 20, 2025 · NCT05755867The International PNH Interest Group PNH Registry
RecruitingObservational study
All ages · All sexes · Updated Jul 29, 2024 · NCT06524726From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.