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Home › Medications › eculizumab

eculizumab

4 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials of eculizumab

34 trials tracked of eculizumab: 16 in phase 3 or 4 and 10 with published results. The most-cited published study has 842 citations.

34Trials tracked
16Phase 3 & 4
0Recruiting
10With published results
Phase distribution
Phase 4 2 Phase 3 14 Phase 2 16 Phase 1 2
  1. Phase 3 Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study) Completed · 842 cited
  2. Phase 3 Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed · 579 cited
  3. Phase 3 ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Completed · 383 cited
  4. Phase 3 Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis Completed · 262 cited
  5. Phase 3 Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment Completed · 150 cited
  6. Phase 3 ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed · 129 cited
Show 28 more trials
  1. Phase 3 An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients Completed · 81 cited
  2. Phase 3 The De-novo Use of Eculizumab in Presensitized Patients Receiving Cardiac Transplantation Completed · 76 cited
  3. Phase 4 Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH) Completed · 42 cited
  4. Phase 3 A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH Completed · 9 cited
  5. Phase 4 Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab Completed
  6. Phase 3 Prevention of Delayed Graft Function Using Eculizumab Therapy (PROTECT Study) Completed
  7. Phase 3 A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG) Completed
  8. Phase 3 A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome Completed
  9. Phase 3 Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China Completed
  10. Phase 3 Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China Completed
  11. Phase 2 An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica Completed
  12. Phase 2 Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS Completed
  13. Phase 2 Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS Completed
  14. Phase 2 Complement Inhibition With Eculizumab for the Treatment of Non-Exudative Macular Degeneration (AMD) Completed
  15. Phase 2 An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome Completed
  16. Phase 2 Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Completed
  17. Phase 2 Dosing Regimen of Eculizumab Added to Conventional Treatment in Positive Cross Match Living Donor Kidney Transplant Completed
  18. Phase 2 Eculizumab to Enable Renal Transplantation in Patients With History of Catastrophic Antiphospholipid Antibody Syndrome Completed
  19. Phase 2 Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients Completed
  20. Phase 2 Complement Inhibition Using Eculizumab to Overcome Platelet Transfusion Refractoriness in Patients With Severe Thrombocytopenia Completed
  21. Phase 2 An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome Completed
  22. Phase 2 Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS Completed
  23. Phase 2 Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS) Completed
  24. Phase 2 Safety & Efficacy Of Eculizumab In The Prevention Of AMR In Sensitized Recipients Of A Kidney Transplant From A Deceased Donor Completed
  25. Phase 2 E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Completed
  26. Phase 2 Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab Completed
  27. Phase 1 Eculizumab Therapy for Chronic Complement-Mediated Injury in Kidney Transplantation Completed
  28. Phase 1 Eculizumab Therapy for Dense Deposit Disease and C3 Nephropathy Completed

Showing the 34 most-cited and recently-updated of 34 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.
Mechanistic-driven diagnosis and therapy shift focus from platelet counts to underlying pathophysiology of thrombocytopenia
Hematology Sys. Review
Mechanistic-driven diagnosis and therapy shift focus from platelet counts to underlying pathophysiology of thrombocytopenia New Framework Offers Precise Treatment for Low Blood Platelets
This systematic review synthesizes the multidimensional pathogenesis of thrombocytopenia across five axes including production, immune-media…
A new diagnostic framework moves beyond simple blood counts to identify the specific cause of low platelets for more precise treatment.
Frontiers Sep 29, 2026
Complement-targeted therapies show clinical activity in C3G and aHUS including 68% reduction in proteinuria
Drug Pipeline Meta-analysis
Complement-targeted therapies show clinical activity in C3G and aHUS including 68% reduction in proteinuria Newer therapies show promise for rare kidney and blood conditions
This narrative synthesis of 38 studies evaluates complement-targeted therapies for patients with C3G and aHUS. The review highlights positiv…
Newer treatments like iptacopan and pegcetacoplan may help manage serious kidney issues and blood disorders in specific patient groups.
Frontiers Aug 24, 2026
Switching therapy after rituximab resistance in AQP4-IgG+ NMOSD shows variable relapse outcomes
Oncology Cohort
Switching therapy after rituximab resistance in AQP4-IgG+ NMOSD shows variable relapse outcomes New Hope for Tough-to-Treat Nerve Disorder
This single-center, retrospective case series of 10 adults with AQP4-IgG+ NMOSD who were resistant to rituximab evaluated switching therapy.
Patients with a rare nerve disease who stopped responding to standard treatment found relief after switching to new therapies that prevented…
Frontiers Apr 19, 2026
Complement inhibitors and FcRn blockers improve outcomes in AChR-Ab+ generalized myasthenia gravis
Oncology Meta-analysis
Complement inhibitors and FcRn blockers improve outcomes in AChR-Ab+ generalized myasthenia gravis New Drugs Double Your Chance of Getting Better
A meta-analysis of 739 adults in RCTs and 588 in OLEs with acetylcholine receptor antibody-positive generalized myasthenia gravis found that…
New drugs for generalized myasthenia gravis double the odds of meaningful improvement while reducing the need for rescue therapy and severe …
Apr 14, 2026