An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Enrollment
- 3,000
- Locations
- 12
- Primary Endpoint
- Proportion of patients who experience specified events
Study Overview
Brief Summary
Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.
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.
Study Design
- Study Type
- Observational
- Observational Model
- Other
- Time Perspective
- Other
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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]).
Outcomes
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
Secondary Outcomes
No secondary outcomes reported