Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

Overview

Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.

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

Study Type

  • Study Type: Observational [Patient Registry]
  • Study Design
    • Time Perspective: Other
  • Study Primary Completion Date: December 31, 2023

Detailed Description

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.

Clinical Trial Outcome Measures

Primary Measures

  • 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

Participating in This Clinical Trial

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]).

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Alexion Pharmaceuticals, Inc.
  • Collaborator
    • Syneos Health
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Katerina Anokhina, MD, Study Director, Alexion Pharmaceuticals, Inc.
  • Overall Contact(s)
    • Caroline Collupy, ahus-registry@syneoshealth.com

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