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Registry for patients with Atypical Hemolytic-Uremic Syndrome

An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)

Alexion Pharmaceuticals, Inc. · NCT01522183

This study is collecting information from people with Atypical Hemolytic-Uremic Syndrome to see how the disease progresses in those treated with eculizumab or ravulizumab and those who aren't treated at all.

Quick facts

Study typeObservational
Enrollment3000 (estimated)
SexAll
SponsorAlexion Pharmaceuticals, Inc. (industry)
Drugs / interventionseculizumab, ravulizumab
Locations141 sites (Aurora, Colorado and 140 other locations)
Trial IDNCT01522183 on ClinicalTrials.gov

What this trial studies

This registry collects post-marketing safety data on patients diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS) who are treated with eculizumab or ravulizumab, as well as those who are untreated. The study aims to gather comprehensive information on the progression of the disease in all enrolled patients, regardless of their treatment status. By documenting patient outcomes and safety data, the registry seeks to enhance understanding of aHUS and improve future treatment strategies.

Who should consider this trial

Good fit: Ideal candidates include males and females of any age diagnosed with aHUS, regardless of whether they have an identified complement pathogenic variant.

Not a fit: Patients with Hemolytic Uremic Syndrome solely due to Shiga Toxin producing Escherichia coli (STEC) may not benefit from this registry.

Why it matters

Potential benefit: If successful, this registry could provide valuable insights into the safety and efficacy of treatments for aHUS, ultimately leading to better patient management.

How similar studies have performed: Other studies have successfully utilized registries to gather safety and efficacy data for rare diseases, indicating that this approach is both valid and beneficial.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Male or female patients of any age, including minors, who have been diagnosed with aHUS
* Patients with or without an identified complement pathogenic variant or anti-complement factor antibody
* Able to give written informed consent. Patient or patient's parent/legal guardian must be willing and able to given written informed consent and the patient (if minor) must be willing to give written informed assent \[if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)\].
* ADAMTS13 \> 5%, if performed.

Exclusion Criteria:

* Hemolytic Uremic Syndrome (HUS) only due to Shiga Toxin producing Escherichia coli (STEC).
* Unable to give written informed consent. Patient or patient's parent/legal guardian unable to give written informed consent. Patient (if minor) unable to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees \[IRB/IEC\]).

Where this trial is running

Aurora, Colorado and 140 other locations

+91 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Atypical Hemolytic-Uremic Syndrome, aHUS, Thrombotic Microangiopathy, TMA

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.