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Condition: Paroxysmal Nocturnal Hemoglobinuria Synced from ClinicalTrials.gov · July 28, 2026

Recruiting studies: Paroxysmal Nocturnal Hemoglobinuria

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Phase 2 · 1 location(s) · Haisco Pharmaceutical Group Co., Ltd. · NCT06745622A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 1 · 2 location(s) · Shanghai Lanyi Therapeutics Co., Ltd. · NCT07256301Global PNH Patient Registry
Phase N/A · 1 location(s) · Aplastic Anemia and MDS International Foundation · NCT05755867Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Phase 3 · 13 location(s) · Novartis Pharmaceuticals · NCT06934967Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
Phase 2/Phase 3 · 1 location(s) · Wuhan Createrna Science and Technology Co., Ltd · NCT06933914Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Phase N/A · 7 location(s) · Alexion Pharmaceuticals, Inc. · NCT06312644REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Phase N/A · 12 location(s) · Novartis Pharmaceuticals · NCT07229235Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells a
Phase 2 · 1 location(s) · National Heart, Lung, and Blood Institute (NHLBI) · NCT03520647National Longitudinal Cohort of Hematological Diseases
Phase N/A · 1 location(s) · Institute of Hematology & Blood Diseases Hospital, China · NCT04645199A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobin
Phase 2 · 1 location(s) · S-INFINITY Pharmaceuticals Co., Ltd · NCT06978699Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complem
Phase 3 · 2 location(s) · Wuhan Createrna Science and Technology Co., Ltd · NCT06932744Home Reported Outcomes in PNH
Phase N/A · 1 location(s) · Novartis Pharmaceuticals · NCT06411626Danicopan PMS in Korea
Phase N/A · 4 location(s) · AstraZeneca · NCT07457151The International PNH Interest Group PNH Registry
Phase N/A · 1 location(s) · International PNH Interest Group · NCT06524726A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
Phase N/A · 3 location(s) · Novartis Pharmaceuticals · NCT07416162A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase N/A · 87 location(s) · Swedish Orphan Biovitrum · NCT05776472Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient
Phase 1/Phase 2 · 4 location(s) · ADARx Pharmaceuticals, Inc. · NCT05876312Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Us
Phase 2 · 2 location(s) · National Heart, Lung, and Blood Institute (NHLBI) · NCT01174108Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinical
Phase 3 · 4 location(s) · Alexion Pharmaceuticals, Inc. · NCT06449001A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in
Phase 1/Phase 2 · 8 location(s) · Regeneron Pharmaceuticals · NCT07187401A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hem
Phase 3 · 19 location(s) · Regeneron Pharmaceuticals · NCT07154745A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants
Phase 1 · 1 location(s) · Shenzhen Kangzhe Biotechnology Co., Ltd. · NCT07462780A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With P
Phase 3 · 45 location(s) · Regeneron Pharmaceuticals · NCT05744921A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 2 · 12 location(s) · Apellis Pharmaceuticals, Inc. · NCT04901936Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
Phase 3 · 1 location(s) · Wuhan Createrna Science and Technology Co., Ltd · NCT06932471Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Phase 1/Phase 2 · 1 location(s) · Jiangsu Hansoh Pharmaceutical Co., Ltd. · NCT07470762A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
Phase N/A · 14 location(s) · Novartis Pharmaceuticals · NCT06931691MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Se
Phase 2 · 1 location(s) · Masonic Cancer Center, University of Minnesota · NCT06412497Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment
Phase 1 · 1 location(s) · Haisco Pharmaceutical Group Co., Ltd. · NCT07152288
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.