Study Stopped
Terminated for administrative reasons before enrollment was complete.
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
EVIDENCE
Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
2 other identifiers
observational
67
4 countries
24
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Nov 2015
24 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 4, 2015
CompletedFirst Submitted
Initial submission to the registry
November 24, 2015
CompletedFirst Posted
Study publicly available on registry
November 25, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 5, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
October 5, 2017
CompletedResults Posted
Study results publicly available
January 2, 2020
CompletedJanuary 2, 2020
December 1, 2019
1.9 years
November 24, 2015
May 14, 2018
December 12, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment
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.
Baseline, 24 Months
Secondary Outcomes (2)
Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR)
Baseline, 24 Months
Incidence Of Plasma Exchange And Plasma Infusion (PE/PI)
Baseline, 24 Months
Study Arms (1)
Eculizumab
The targeted population consists of pediatric and adult participants with aHUS who received eculizumab at the discretion of the treating physician.
Interventions
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.
Eligibility Criteria
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).
You may qualify if:
- Currently receiving eculizumab treatment in the M11-001 aHUS Registry
- Two normal platelet counts at least 4 weeks apart
- Two normal lactate dehydrogenase levels at least 4 weeks apart
- Willing, committed, and able to return for all clinic visits and complete all study-related procedures
- 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)
You may not qualify if:
- Any prior eculizumab treatment discontinuation
- On chronic dialysis (defined as ≥3 months on dialysis)
- Currently participating in another complement inhibitor trial
- Life expectancy of \<6 months
- 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)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (24)
Clinical Trial Site
Washington D.C., District of Columbia, 20007, United States
Clinical Trial Site
Atlanta, Georgia, 30322, United States
Clinical Trial Site
Chicago, Illinois, 60611, United States
Clinical Trial Site
Boston, Massachusetts, 02115, United States
Clinical Trial Site
Hackensack, New Jersey, 07601, United States
Clinical Trial Site
Charlotte, North Carolina, 28203, United States
Clinical Trial Site
Columbus, Ohio, 43210, United States
Clinical Trial Site
Houston, Texas, 77030, United States
Clinical Trial Site
Adelaide, 5000, Australia
Clinical Trial Site
Clayton, 3168, Australia
Clinical Trial Site
Heidelberg, 3084, Australia
Clinical Trial Site
Kingswood, 2747, Australia
Clinical Trial Site
Liverpool, 2170, Australia
Clinical Trial Site
Nedlands, 6109, Australia
Clinical Trial Site
Parkville, 3050, Australia
Clinical Trial Site
Parkville, 3052, Australia
Clinical Trial Site
Perth, 6008, Australia
Clinical Trial Site
Westmead, 2145, Australia
Clinical Trial Site
Woolloongabba, 4102, Australia
Clinical Trial Site
Hannover, 30625, Germany
Clinical Trial Site
Hanover, 30625, Germany
Clinical Trial Site
Kiel, 24105, Germany
Clinical Trial Site
Lübeck, 23538, Germany
Clinical Trial Site
London, NW3 2PF, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
As the study was terminated for administrative reasons, before enrollment was complete, and the program subsequently lost funding, all collected data could not be analyzed to generate summary-level data.
Results Point of Contact
- Title
- Alexion Pharmaceuticals, Inc.
- Organization
- Alexion Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 24, 2015
First Posted
November 25, 2015
Study Start
November 4, 2015
Primary Completion
October 5, 2017
Study Completion
October 5, 2017
Last Updated
January 2, 2020
Results First Posted
January 2, 2020
Record last verified: 2019-12