RecruitingPhase 1NCT07760467
Blood
Paroxysmal Nocturnal Hemoglobinuria
Also known as PNH, Marchiafava-Micheli syndrome
Paroxysmal nocturnal hemoglobinuria is an acquired clonal disorder of hematopoietic stem cells caused by somatic mutations in the PIGA gene, resulting in deficiency of GPI-anchored complement regulatory proteins (CD55 and CD59) on blood cel
ORPHA:447 ↗Gene PIG-APrevalence 1-5 per millionOnset Young to middle-aged adults; median age of diagnosis 35-40 yearsAcquired somatic mutation
29
studies recruiting now
as of 7 Sept 2026
189
studies registered in total
as of 7 Sept 2026
8
countries with a recruiting site
as of 7 Sept 2026
12 Aug 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2NCT03520647
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
RecruitingPhase 2NCT01174108
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
RecruitingNCT07457151
Danicopan PMS in Korea
RecruitingPhase 3NCT06934967
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 29 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Registry: International PNH Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria is an acquired clonal disorder of hematopoietic stem cells caused by somatic mutations in the PIGA gene, resulting in deficiency of GPI-anchored complement regulatory proteins (CD55 and CD59) on blood cell surfaces. The loss of these proteins renders red blood cells, white blood cells, and platelets vulnerable to complement-mediated destruction, leading to intravascular hemolysis, thrombosis in unusual sites, and cytopenias. PNH is closely associated with aplastic anemia and carries a significantly elevated risk of life-threatening venous thromboembolism.
Common clinical features
Hemoglobinuria (dark urine, especially in the morning)Chronic intravascular hemolysis with elevated LDHVenous thrombosis in unusual sites (hepatic, mesenteric, cerebral veins)Abdominal pain and dysphagia from smooth muscle dystoniaSevere fatigue and anemiaPulmonary hypertensionRecurrent infections from neutropenia in aplastic anemia-associated PNHErectile dysfunction in males
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
7 approved treatments and 9 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Ravulizumab (Ultomiris)Approved: Pegcetacoplan (Aspaveli)Approved: Crovalimab (Piasky)Approved: Iptacopan Hydrochloride (Fabhalta)Approved: Danicopan (Voydeya)Approved: IptacopanApproved: Eculizumab (Bekemv)
Phase 3Cemdisiran
Phase 3Pozelimab (Veopoz)
Phase 2Zilucoplan
Phase 2Zaltenibart
Phase 2Vensobafusp Alfa
Phase 2Ruxoprubart
Phase 2Nomacopan
Phase 2Vemircopan
+ 1 more in development
Before you apply
Things trial teams commonly ask about for Paroxysmal Nocturnal Hemoglobinuria. Not eligibility rules; those are set by each study.
- Flow cytometry quantifying the PNH clone size (percentage of GPI-deficient granulocytes and red cells) is essential for most trial eligibility criteria; a clone size greater than 10% in granulocytes is typically required.
- Current use of complement inhibitors (eculizumab, ravulizumab) affects eligibility for trials of novel complement pathway agents; document your treatment history and any breakthrough hemolysis events.
- LDH levels, transfusion history, thrombotic event history, and FACIT-Fatigue scores are commonly used outcome measures in PNH trials and should be documented prospectively.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).