Atypical Hemolytic Uremic Syndrome clinical trials
The registry lists 45 studies for Atypical Hemolytic Uremic Syndrome across more than 10 countries. 14 are recruiting or about to start. 37 of them are mainly about Atypical Hemolytic Uremic Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Who runs them
Alexion Pharmaceuticals, Inc. (16) · Mario Negri Institute for Pharmacological Research (4) · Novartis Pharmaceuticals (3) · AstraZeneca (3) · Omeros Corporation (2) · Hoffmann-La Roche (2) · Nantes University Hospital (1) · Alnylam Pharmaceuticals (1)
What is being tested here
Drugs under test in Atypical Hemolytic Uremic Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Iptacopan | 2 | 3 | 3 |
| Eculizumab | 1 | 1 | 10 |
| Ravulizumab | 1 | 2 | 3 |
| Ruxoprubart | 1 | 0 | 1 |
| Crovalimab | 0 | 2 | 2 |
| Narsoplimab | 0 | 1 | 2 |
| Avacopan | 0 | 0 | 2 |
| Cemdisiran | 0 | 0 | 2 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 3
- Dec 2026Complement Prospective Evaluation of Thrombotic Microangiopathy on Endothelium · Maastricht University Medical Center
- Dec 2026Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy · Mario Negri Institute for Pharmacological Research
- Jun 2027Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS · Alexion Pharmaceuticals, Inc.
Who these studies take
Of the 12 Atypical Hemolytic Uremic Syndrome studies recruiting now, 10 (83%) set no upper age limit and 0 (0%) stop at 75 or younger. 6 (50%) take children under 18. 1 (8%) are for women only and 0 (0%) for men only; 2 (17%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 6 (50%) | 5 (42%) |
| 18 | 12 (100%) | 11 (92%) |
| 30 | 12 (100%) | 11 (92%) |
| 40 | 12 (100%) | 11 (92%) |
| 50 | 12 (100%) | 11 (92%) |
| 65 | 12 (100%) | 11 (92%) |
| 75 | 12 (100%) | 11 (92%) |
| 85 | 12 (100%) | 11 (92%) |
Across all 45 studies, recruiting or not: 16% stop at 75 or younger and 16% at 65; 60% take children; 7% are for women only and 0% for men only; 11% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 15, 2026.
Studies
Recruiting first, then most recently updated. Showing 45 of 45. search them.
- Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUSRecruitingNCT05795140Phase 36 countries
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- Ravulizumab Outcomes in Polish Patients With aHUSRecruitingNCT073997301 country
- Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)RecruitingNCT05935215Phase 38 countries
- Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUSRecruitingNCT07308574Phase 41 country
- Study of Ultomiris® (Ravulizumab) Safety in PregnancyRecruitingNCT063126447 countries
- Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic MicroangiopathyNot yet recruitingNCT05684159Phase 2
- Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare DiseasesRecruitingNCT05996731Not applicable1 country
- Complement Prospective Evaluation of Thrombotic Microangiopathy on EndotheliumRecruitingNCT047451951 country
- Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized TherapyRecruitingNCT05805202Not applicable1 country
- Atypical Hemolytic-Uremic Syndrome (aHUS) RegistryRecruitingNCT0152218317 countries
- Eculizumab in Hypertensive Emergency-associated Hemolytic Uremic SyndromeRecruitingNCT05726916Phase 31 country
- National Registry of Rare Kidney DiseasesRecruitingNCT060658521 country
- OMS721 Compassionate Use in Patients With Thrombotic MicroangiopathyAvailableNCT02355782
- A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)Active, not recruitingNCT04861259Phase 316 countries
- A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome PatientsActive, not recruitingNCT060992361 country
- A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)Active, not recruitingNCT04958265Phase 310 countries
- Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUSCompletedNCT02574403Phase 41 country
- The Burden of Atypical Hemolytic Uremic Syndrome and The Clinical Characteristics of Patients in Egyptian Hospitals A Multicenter, Observational, Retrospective Cohort Study in EgyptCompletedNCT072185361 country
- Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor TherapyCompletedNCT04889430Phase 38 countries
- Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic SyndromeTerminatedNCT03205995Phase 34 countries
- Personalized Spacing of Eculizumab Infusions Based on Therapeutic Pharmacological Monitoring (EspacECU)CompletedNCT04859608Phase 31 country
- Safety and Efficacy Study of OMS721 in Patients With Thrombotic MicroangiopathiesCompletedNCT02222545Phase 212 countries
- Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)CompletedNCT03131219Phase 37 countries
- Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)CompletedNCT02949128Phase 314 countries
- Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant RecipientsCompletedNCT03518203Phase 21 country
- A Study in Healthy Volunteers to Evaluate the Drug-Drug Interaction Potential of CCX168 With Concomitant MedicationsCompletedNCT06004947Phase 11 country
- A Retrospective, Observational, Non-interventional Trial to Assess Eculizumab Treatment Effect in Patients With Atypical Hemolytic Uremic Syndrome (aHUS)CompletedNCT017709518 countries
- Observational Study of Elizaria® in aHUS PatientsCompletedNCT047498101 country
- Eculizumab to Cemdisiran Switch in aHUSWithdrawnNCT03999840Phase 21 country
- Eculizumab Use in the Postpartum Period for the Treatment of Pregnancy Associated aHUS: A Case SeriesStatus unknownNCT035745061 country
- Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and TreatmentTerminatedNCT026148984 countries
- The Role of Microparticles as a BiomarkerWithdrawnNCT026266631 country
- A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic SyndromeWithdrawnNCT03303313Phase 210 countries
- TTP and aHUS in Complicated PregnanciesStatus unknownNCT036055111 country
- Complement Inhibition in aHUS Dialysis PatientsTerminatedNCT02464891Phase 21 country
- An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic SyndromeCompletedNCT01194973Phase 27 countries
- The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)CompletedNCT01757431Phase 21 country
- aHUS Observational Long Term Follow-UpTerminatedNCT0152217012 countries
- Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUSCompletedNCT00844545Phase 25 countries
- Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUSCompletedNCT00844428Phase 27 countries
- Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS)CompletedNCT00838513Phase 27 countries
- Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUSCompletedNCT00844844Phase 25 countries
- An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic SyndromeCompletedNCT01193348Phase 29 countries
- To Characterize the Safety and Tolerability of Eculizumab in Two Japanese aHUS PatientsCompletedNCT017554291 country