CompletedPhase 3

Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

China25 participantsStarted 2023-07-14

Plain-language 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

Who can participate

Age range0 Years – 99 Years

SexALL

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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

What they're measuring

Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response

Timeframe: Up to Week 26

Trial details

NCT IDNCT05876351

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-05-07

Results submitted2025-11-17

View on ClinicalTrials.gov More Atypical Hemolytic Uremic trials