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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02614898
Registration number
NCT02614898
Ethics application status
Date submitted
24/11/2015
Date registered
25/11/2015
Date last updated
2/01/2020
Titles & IDs
Public title
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
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Scientific title
Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
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Secondary ID [1]
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2015-003135-35
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Secondary ID [2]
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ECU-aHUS-403
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Universal Trial Number (UTN)
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Trial acronym
EVIDENCE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Atypical Hemolytic Uremic Syndrome
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Condition category
Condition code
Renal and Urogenital
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Other renal and urogenital disorders
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Blood
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Anaemia
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Blood
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Other blood disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Other interventions - Eculizumab
Eculizumab - The targeted population consists of pediatric and adult participants with aHUS who received eculizumab at the discretion of the treating physician.
Other interventions: 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.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline, 24 Months
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Secondary outcome [1]
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Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR)
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline, 24 Months
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Secondary outcome [2]
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Incidence Of Plasma Exchange And Plasma Infusion (PE/PI)
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline, 24 Months
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Eligibility
Key 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)
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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)
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
4/11/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
5/10/2017
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Sample size
Target
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Accrual to date
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Final
67
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Clinical Trial Site - Adelaide
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Recruitment hospital [2]
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Clinical Trial Site - Clayton
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Recruitment hospital [3]
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Clinical Trial Site - Heidelberg
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Recruitment hospital [4]
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Clinical Trial Site - Kingswood
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Recruitment hospital [5]
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Clinical Trial Site - Liverpool
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Recruitment hospital [6]
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Clinical Trial Site - Nedlands
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Recruitment hospital [7]
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Clinical Trial Site - Parkville
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Recruitment hospital [8]
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Clinical Trial Site - Perth
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Recruitment hospital [9]
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Clinical Trial Site - Westmead
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Recruitment hospital [10]
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Clinical Trial Site - Woolloongabba
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment postcode(s) [2]
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3168 - Clayton
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Recruitment postcode(s) [3]
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3084 - Heidelberg
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Recruitment postcode(s) [4]
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2747 - Kingswood
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Recruitment postcode(s) [5]
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2170 - Liverpool
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Recruitment postcode(s) [6]
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6109 - Nedlands
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Recruitment postcode(s) [7]
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3050 - Parkville
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Recruitment postcode(s) [8]
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3052 - Parkville
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Recruitment postcode(s) [9]
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6008 - Perth
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Recruitment postcode(s) [10]
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2145 - Westmead
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Recruitment postcode(s) [11]
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4102 - Woolloongabba
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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District of Columbia
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United States of America
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State/province [2]
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Georgia
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United States of America
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Illinois
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United States of America
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Massachusetts
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Country [5]
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State/province [5]
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New Jersey
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Country [6]
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United States of America
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State/province [6]
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North Carolina
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United States of America
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State/province [7]
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Ohio
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Country [8]
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United States of America
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State/province [8]
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Texas
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Country [9]
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Germany
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State/province [9]
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Hannover
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Germany
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State/province [10]
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Hannöver
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Country [11]
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Germany
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Kiel
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Germany
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State/province [12]
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Luebeck
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United Kingdom
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State/province [13]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02614898
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Email
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Contact person for public queries
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Fax
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02614898
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