Atypical Hemolytic-Uremic Syndrome (aHU… | Clinical Trial

Key information

Conditions

Atypical Hemolytic-Uremic Syndrome Anemia Anemia, Hemolytic Atypical Hemolytic Uremic Syndrome Blood Platelet Disorders Cytopenia Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Hematologic Diseases Hemic and Lymphatic Diseases Hemolytic-Uremic Syndrome Kidney Diseases Male Urogenital Diseases Thrombocytopenia Thrombotic Microangiopathies Uremia Urogenital Diseases Urologic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Trial Site 1, Westmead, New South Wales, Australia

About this study

The study will capture post-marketing safety data on patients treated with eculizumab or ravulizumab. Additionally, the study will collect information on the progression of disease in all patients.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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]).

Treatment and study plan

Primary outcomes

Proportion of patients who experience specified events

Time frame: 10 years

To collect and evaluate safety and effectiveness data specific to the use of eculizumab or ravulizumab in aHUS patients.
Time to first and subsequent occurrence of specified events.

Time frame: 5 years

To assess the long term manifestations of thrombotic microangiopathy (TMA) complications of aHUS as well as other clinical outcomes, including morbidity and mortality in aHUS patients, receiving eculizumab or ravulizumab treatment or other disease management approaches

Study contacts

Contact information is provided by the study sponsor or research team.

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

[email protected]

1-855-752-2356

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Collaborators

Syneos Health

Registry information

Official study title

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

Important dates

Study start: 2013
Primary completion: 2031
Study completion: 2031
First posted: Jan 31, 2012
Registry last updated: Sep 16, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed