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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 579 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 · 579 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 · 86 cited
  3. Phase 3 Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Completed · 25 cited
  4. Phase 3 Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed · 18 cited
  5. Phase 3 A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria Completed · 13 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.

What the trials found Updated — new results For clinicians

Paroxysmal nocturnal hemoglobinuria: what the trials found

Several C5 inhibitors have demonstrated significant efficacy in reducing hemolysis markers and improving clinical outcomes. Eculizumab showed a significant reduction in Lactate Dehydrogenase (LDH) levels [1, 17, 20] and was associated with reductions in packed red blood cell (pRBC) transfusions [17, 20]. Ravulizumab also demonstrated significant LDH reductions 18 and improvements in free hemoglobin levels 18, while a separate study showed mixed results for LDH percentage change but high rates of transfusion avoidance 7.

Other targeted therapies showed robust clinical activity. Danicopan was associated with significant increases in hemoglobin (Hb) and higher rates of transfusion avoidance compared to placebo 4. Pegcetacoplan demonstrated significant reductions in LDH levels and marked improvements in transfusion avoidance during the rapid clinical progression period 6, as well as a substantial reduction in LDH at one year 22. APL-2 showed superior results in Hb stabilization and LDH concentration reduction compared to placebo 9. Iptacopan met non-inferiority and superiority endpoints for Hb levels 10.

Emerging agents like Pozelimab demonstrated 100% transfusion-free rates in a small cohort 12 and high rates of adequate hemolysis control 13. SB12 (a proposed eculizumab biosimilar) was evaluated with LDH measurements at week 26 11.

Recent results — preliminary, needs further review

  • LFG316 showed significant reduction in serum LDH levels over the treatment period, though not yet corroborated as established 14.
  • LNP023 demonstrated a substantial percentage decrease in LDH levels 15.
  • ACH-0144471 was associated with significant reductions in LDH levels at weeks 49 and 169 16.

For the clinician treating this condition

  • C5 inhibitors (Eculizumab, Ravulizumab) consistently reduce LDH levels and decrease the requirement for packed red blood cell transfusions.
  • Targeted therapies such as Danicopan, Pegcetacoplan, and APL-2 have shown statistically significant improvements in hemoglobin stability and reduction of hemolysis markers.
  • Iptacopan demonstrated superior and non-inferiority outcomes for hemoglobin levels compared to baseline.

AI synthesis of 13 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
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