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Home › Conditions › Paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria

Part of Anemia

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Paroxysmal nocturnal hemoglobinuria

23 trials tracked for Paroxysmal nocturnal hemoglobinuria: 11 in phase 3 or 4 and 7 with published results. The most-cited published study has 578 citations.

23Trials tracked
11Phase 3 & 4
0Recruiting
7With published results
Phase distribution
Phase 4 1 Phase 3 10 Phase 2 10 Phase 1 1 Other / NA 1
  1. Phase 3 Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed · 578 cited
  2. Phase 3 Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) Completed · 84 cited
  3. Phase 3 Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Completed · 23 cited
  4. Phase 3 Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed · 17 cited
  5. Phase 3 A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria Completed · 11 cited
  6. Phase 3 A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH Completed · 6 cited
Show 17 more trials
  1. Phase 3 A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH Completed · 6 cited
  2. Phase 4 Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab Completed
  3. Phase 3 A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria Completed
  4. Phase 3 Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. Completed
  5. Phase 3 Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China Completed
  6. Phase 2 Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed
  7. Phase 2 A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed
  8. Phase 2 Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Completed
  9. Phase 2 Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Completed
  10. Phase 2 Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Completed
  11. Phase 2 Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy Completed
  12. Phase 2 Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria Completed
  13. Phase 2 Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy Completed
  14. Phase 2 Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders Completed
  15. Phase 2 E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Completed
  16. Phase 1 Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. Completed
  17. N/A Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders Completed

Showing the 23 most-cited and recently-updated of 23 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Small PNH clones in SLE linked to thrombotic events and hemolysis in case series
Allergy & Immunology Sys. Review
Small PNH clones in SLE linked to thrombotic events and hemolysis in case series Small blood cell issues can impact patients with lupus and PNH
A systematic review of 8 cases found that 37.5% of SLE patients with PNH had thrombotic events and 87.5% had detectable hemolysis. The findi…
A review of cases shows that even small clusters of abnormal cells can cause serious blood complications in people with lupus and PNH.
Frontiers Jun 26, 2026
FDA Approves Epysqli (eculizumab-sbja) for PNH, aHUS, and gMG
Hematology FDA Approval
FDA Approves Epysqli (eculizumab-sbja) for PNH, aHUS, and gMG FDA approves new drug Epysqli for three rare blood and nerve conditions.
The FDA approved Epysqli (eculizumab-sbja) for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and gen…
The FDA approved Epysqli (eculizumab-sbja) for paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and generalized myas…
FDA May 2, 2026