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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 27, 2023
Sponsor:
Collaborator:
Syneos Health
Information provided by (Responsible Party):
Alexion

Study Description
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Brief Summary:
Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.

Condition or disease
Atypical Hemolytic-Uremic Syndrome

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
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 3000 participants
Observational Model: Other
Time Perspective: Other
Target Follow-Up Duration: 5 Years
Official Title: An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
Actual Study Start Date : April 30, 2012
Estimated Primary Completion Date : December 31, 2023
Estimated Study Completion Date : December 31, 2025

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. 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


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
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]).
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01522183


Contacts
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Contact: Caroline Collupy ahus-registry@syneoshealth.com

Locations
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Sponsors and Collaborators
Alexion
Syneos Health
Investigators
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Study Director: Katerina Anokhina, MD Alexion
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Alexion
ClinicalTrials.gov Identifier: NCT01522183    
Other Study ID Numbers: M11-001
First Posted: January 31, 2012    Key Record Dates
Last Update Posted: March 27, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Alexion:
Atypical Hemolytic-Uremic Syndrome
aHUS
Thrombotic Microangiopathy
TMA
Additional relevant MeSH terms:
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Azotemia
Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Syndrome
Hemolysis
Disease
Pathologic Processes
Uremia
Kidney Diseases
Urologic Diseases
 Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Male Urogenital Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders