Phase 3
N=25
Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China
Atypical Hemolytic Uremic
Bottom Line
View on ClinicalTrials.gov: NCT05876351 ↗Enrolled (actual)
25
Serious AEs
32.0%
Results posted
Dec 2025
Primary outcome: Primary: Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response — 64.0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Eculizumab (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- May 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response |
64.0 | — |
| SECONDARY Number of Participants With an Adverse Event (AE) |
24; 8 | — |
| SECONDARY Mean Serum Concentration of Eculizumab |
4.690; 373.423; 153.190; 498.262; 353.726; 727.862 | — |
| SECONDARY Change From Baseline in Serum Free Complement 5 (C5) |
-79.8842; -79.6539; -79.6636; -80.4277; -80.4284; -80.0632 | — |
| SECONDARY Change From Baseline in Serum Total C5 |
-12.0267; 37.9293; 40.2831; 65.9100; 64.4783; 71.2418 | — |
| SECONDARY Number of Participants With an Anti-drug Antibody (ADA) Response |
— | — |
| SECONDARY Time to Complete TMA Response |
75.0 | — |
| SECONDARY Proportion of Participants On or Off Dialysis at Each Timepoint |
0.440; 0.560; 0.261; 0.739; 0.235; 0.765 | — |
| SECONDARY Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit |
39.82; 32.72; 40.33; 37.53; 35.88; 35.85 | — |
| SECONDARY Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline |
0.545; 0.455; 0; 0.688; 0.313; 0 | — |
| SECONDARY Change From Baseline in Platelets |
76.5; 65.5; 71.3; 79.4; 60.1; 61.8 | — |
| SECONDARY Change From Baseline in LDH |
-9.445; -6.147; -7.083; -5.861; -5.769; -5.444 | — |
| SECONDARY Change From Baseline in Hemoglobin |
17.0; 25.7; 30.5; 30.3; 29.8; 25.2 | — |
Summary
This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China
Eligibility Criteria
Inclusion Criteria
- Any age weighing ≥ 5 kg
- Complement treatment naïve with evidence of TMA.
- History of aHUS prior to kidney transplant,or persistent evidence of TMA at least 4 days after modifying the immunosuppressive regimen.
- Among participants with onset of TMA postpartum, persistent evidence of TMA for > 3 days after the day of childbirth
- All participants must be vaccinated against N meningitidis if not already vaccinated within the time period of active coverage specified by the vaccine manufacturer.
- Participants < 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to local vaccination schedule guidelines.
- In participants receiving treatment with medications known to cause TMA, persistent evidence of TMA at least 4 days after modifying the excluded medication
Exclusion Criteria
- Known familial or acquired ADAMTS13deficiency (activity < 5%).
- ST-HUS as demonstrated by local guidelines.
- Positive direct Coombs test which is indicative of a clinically significant immune-mediated hemolysis not due to aHUS.
- HIV infection, and /or unresolved meningococcal disease
- Ongoing sepsis, and / or presence or suspicion of active and untreated systemic infection
- Organ transplantation history, and/or Bone marrow transplant/hematopoietic stem cell transplant within 6 months prior to the start of Screening.
- Among participants with a kidney transplant, acute kidney dysfunction within 4 weeks of transplant consistent with the diagnosis of acute antibody-mediated rejection.
- Among participants without a kidney transplant, history of kidney disease other than aHUS
- Identified drug exposure-related HUS, and / or HUS related to vitamin B12 deficiency and / or known genetic defects of cobalamin C metabolism.
- History of malignancy within 5 years of Screening.
- Known systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
- Chronic dialysis.
- Prior use of complement inhibitors.
- Use of tranexamic acid within 7 days prior to the start of Screening.
- Other immunosuppressive therapies.
- Receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to the start of Screening.
- Received vasopressors or inotropes within 7 days prior to Screening.
- Previously or currently treated with a complement inhibitor.
- Has participated in another interventional treatment study or used any experimental therapy.
- Hypersensitivity to any excipient in eculizumab.
- Pregnant or breastfeeding.
Data sourced from ClinicalTrials.gov (NCT05876351). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.