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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. Identifier: NCT01522183
Recruitment Status : Recruiting
First Posted : January 31, 2012
Last Update Posted : May 3, 2019
Syneos Health
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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

Condition or disease
Atypical Hemolytic-Uremic Syndrome

Detailed Description:
The study will capture post-marketing safety data on patients treated with eculizumab. Additionally, the study will collect information on the progression of disease in all patients.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 2000 participants
Observational Model: Other
Time Perspective: Other
Target Follow-Up Duration: 10 Years
Official Title: An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
Study Start Date : April 2012
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2025

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 in aHUS patients.

  2. 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

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)

Inclusion Criteria:

  • Male or female patients of any age, including minors, who have been diagnosed with aHUS
  • 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

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 identifier (NCT number): NCT01522183

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Contact: Caroline Collupy

  Show 128 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
Syneos Health
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Study Director: Benjamin Miller, MD Alexion Pharmaceuticals

Additional Information:

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Alexion Pharmaceuticals Identifier: NCT01522183     History of Changes
Other Study ID Numbers: M11-001
First Posted: January 31, 2012    Key Record Dates
Last Update Posted: May 3, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Alexion Pharmaceuticals:
Atypical Hemolytic-Uremic Syndrome
Thrombotic Microangiopathy
Additional relevant MeSH terms:
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Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Pathologic Processes
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Hematologic Diseases
Thrombotic Microangiopathies
Blood Platelet Disorders