Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

Recruiting

• Conditions: Atypical Hemolytic-Uremic Syndrome

• Registration Number: NCT01522183
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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 Timeline

• Study First Submitted: 2012-01-18
• Study First Submitted QC: 2012-01-27
• Study First Posted: 2012-01-31
• Study Start: 2012-04-26
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-13
• Last Update Posted: 2024-11-15
• Primary Completion: 2031-01-01
• Study Completion: 2031-01-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 3000

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]).

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Proportion of patients who experience specified events	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.	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

Trial Locations

Locations (12)

Clinical Trial Site; 🇬🇧 Wales, United Kingdom

Clinical Trial Site 1; 🇪🇸 Barcelona, Spain

Clinical Trial Site 2; 🇪🇸 Barcelona, Spain

Clinical Trial Site (Adult); 🇧🇪 Liège, Belgium

Clinical Trial Site (Pediatric); 🇧🇪 Woluwe-Saint-Lambert, Belgium

Clinical Trial Site (pediatric); 🇧🇪 Liège, Belgium

Clinical Trial Site (adult); 🇧🇪 Woluwe-Saint-Lambert, Belgium

Clinical Trial Site 4; 🇰🇷 Seoul, Korea, Republic of

Clinical Trial Site 5; 🇰🇷 Seoul, Korea, Republic of

Clinical Trial Site 3; 🇰🇷 Seoul, Korea, Republic of

Clinical Trial Site 6; 🇰🇷 Seoul, Korea, Republic of

Clinical Trial Site 7; 🇰🇷 Seoul, Korea, Republic of