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Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01522183
Recruitment Status : Recruiting
First Posted : January 31, 2012
Last Update Posted : March 16, 2022
Sponsor:
Collaborator:
Syneos Health
Information provided by (Responsible Party):
Alexion

Tracking Information
First Submitted Date January 18, 2012
First Posted Date January 31, 2012
Last Update Posted Date March 16, 2022
Actual Study Start Date April 30, 2012
Estimated Primary Completion Date December 31, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 2, 2022)
  • 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
Original Primary Outcome Measures
 (submitted: January 27, 2012)
  • 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 in aHUS patients.
  • Time to first and subsequent occurrence of specified events. [ Time Frame: 10 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 treatment or other disease management approaches
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
Official Title An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
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 Type Observational [Patient Registry]
Study Design Observational Model: Other
Time Perspective: Other
Target Follow-Up Duration 5 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Male or female patients of any age, including minors, who have been diagnosed with aHUS; clinical diagnosis of aHUS, patients with or without an identified complement regulatory factor genetic abnormality or anti-complement factor antibody, ADAMTS13 > 5% (if performed)
Condition Atypical Hemolytic-Uremic Syndrome
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: March 2, 2022)		 3000
Original Estimated Enrollment
 (submitted: January 27, 2012)		 2000
Estimated Study Completion Date December 31, 2025
Estimated Primary Completion Date December 31, 2023   (Final data collection date for primary outcome measure)
Eligibility 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]).
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Caroline Collupy ahus-registry@syneoshealth.com
Listed Location Countries Australia,   Belgium,   Canada,   Denmark,   France,   Germany,   Israel,   Italy,   Korea, Republic of,   Poland,   Russian Federation,   Spain,   Taiwan,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01522183
Other Study ID Numbers M11-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: No
Current Responsible Party Alexion
Original Responsible Party Same as current
Current Study Sponsor Alexion
Original Study Sponsor Same as current
Collaborators Syneos Health
Investigators
Study Director: Katerina Anokhina, MD Alexion
PRS Account Alexion
Verification Date March 2022