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Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment (EVIDENCE)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02614898
Recruitment Status : Terminated (Terminated for administrative reasons before enrollment was complete.)
First Posted : November 25, 2015
Results First Posted : January 2, 2020
Last Update Posted : January 2, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician. The purpose of this study was to assess disease manifestations of complement-mediated thrombotic microangiopathy (TMA) and evaluate potential clinical predictors of disease manifestations and progression in participants with aHUS with or without eculizumab treatment in the clinical setting.

Condition or disease Intervention/treatment
Atypical Hemolytic Uremic Syndrome Other: Eculizumab

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Study Type : Observational
Actual Enrollment : 67 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
Actual Study Start Date : November 4, 2015
Actual Primary Completion Date : October 5, 2017
Actual Study Completion Date : October 5, 2017


Group/Cohort Intervention/treatment
Eculizumab
The targeted population consists of pediatric and adult participants with aHUS who received eculizumab at the discretion of the treating physician.
Other: Eculizumab
This study was an observational study. Therefore, the study drug was not provided as part of this study, and the dosing regimen was based solely at the discretion of the treating physician.
Other Name: Soliris®




Primary Outcome Measures :
  1. Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment [ Time Frame: Baseline, 24 Months ]

    A TMA manifestation was defined as one of following: Hematologic or renal events due to aHUS; extra-renal clinical signs and symptoms of aHUS; tissue (for example, kidney transplant) biopsy demonstrating TMA due to aHUS.

    The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.



Secondary Outcome Measures :
  1. Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline, 24 Months ]

    Change in estimated eGFR over time using the chronic kidney disease-epidemiology (CKD-EPI) formula.

    The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.


  2. Incidence Of Plasma Exchange And Plasma Infusion (PE/PI) [ Time Frame: Baseline, 24 Months ]

    The number of occurrences of PE/PI per participant-years was calculated and summarized by treatment status.

    The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.




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:   Probability Sample
Study Population
Participants with aHUS enrolled in the M11-001 aHUS Registry and currently receiving eculizumab with no prior intentional eculizumab discontinuations at any time (for example, not occasional, unplanned, or temporary missed doses of eculizumab, but intended long-term discontinuation).
Criteria

Inclusion Criteria:

  1. Currently receiving eculizumab treatment in the M11-001 aHUS Registry
  2. Two normal platelet counts at least 4 weeks apart
  3. Two normal lactate dehydrogenase levels at least 4 weeks apart
  4. Willing, committed, and able to return for all clinic visits and complete all study-related procedures
  5. Participant or participant's parent/legal guardian must have been willing and able to give written informed consent. Participant (if minor) must have been willing to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees)

Exclusion Criteria:

  1. Any prior eculizumab treatment discontinuation
  2. On chronic dialysis (defined as ≥3 months on dialysis)
  3. Currently participating in another complement inhibitor trial
  4. Life expectancy of <6 months
  5. Participant or participant's parent/legal guardian was unable to give written informed consent. Participant (if minor) was unable to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees)

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): NCT02614898


Locations
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United States, District of Columbia
Clinical Trial Site
Washington, District of Columbia, United States, 20007
United States, Georgia
Clinical Trial Site
Atlanta, Georgia, United States, 30322
United States, Illinois
Clinical Trial Site
Chicago, Illinois, United States, 60611
United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States, 02115
United States, New Jersey
Clinical Trial Site
Hackensack, New Jersey, United States, 07601
United States, North Carolina
Clinical Trial Site
Charlotte, North Carolina, United States, 28203
United States, Ohio
Clinical Trial Site
Columbus, Ohio, United States, 43210
United States, Texas
Clinical Trial Site
Houston, Texas, United States, 77030
Australia
Clinical Trial Site
Adelaide, Australia, 5000
Clinical Trial Site
Clayton, Australia, 3168
Clinical Trial Site
Heidelberg, Australia, 3084
Clinical Trial Site
Kingswood, Australia, 2747
Clinical Trial Site
Liverpool, Australia, 2170
Clinical Trial Site
Nedlands, Australia, 6109
Clinical Trial Site
Parkville, Australia, 3050
Clinical Trial Site
Parkville, Australia, 3052
Clinical Trial Site
Perth, Australia, 6008
Clinical Trial Site
Westmead, Australia, 2145
Clinical Trial Site
Woolloongabba, Australia, 4102
Germany
Clinical Trial Site
Hannover, Germany, 30625
Clinical Trial Site
Hannöver, Germany, 30625
Clinical Trial Site
Kiel, Germany, 24105
Clinical Trial Site
Luebeck, Germany, 23538
United Kingdom
Clinical Trial Site
London, United Kingdom, NW3 2PF
Sponsors and Collaborators
Alexion Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alexion Pharmaceuticals:
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02614898    
Other Study ID Numbers: ECU-aHUS-403
2015-003135-35 ( EudraCT Number )
First Posted: November 25, 2015    Key Record Dates
Results First Posted: January 2, 2020
Last Update Posted: January 2, 2020
Last Verified: December 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Syndrome
Disease Progression
Hemolysis
Disease
Pathologic Processes
Disease Attributes
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Eculizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs