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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
Scientific title
Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
Secondary ID [1] 0 0
2015-003135-35
Secondary ID [2] 0 0
ECU-aHUS-403
Universal Trial Number (UTN)
Trial acronym
EVIDENCE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Atypical Hemolytic Uremic Syndrome 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
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.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment
Timepoint [1] 0 0
Baseline, 24 Months
Secondary outcome [1] 0 0
Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR)
Timepoint [1] 0 0
Baseline, 24 Months
Secondary outcome [2] 0 0
Incidence Of Plasma Exchange And Plasma Infusion (PE/PI)
Timepoint [2] 0 0
Baseline, 24 Months

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)
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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)

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/11/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/10/2017
Sample size
Target
Accrual to date
Final
67
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Adelaide
Recruitment hospital [2] 0 0
Clinical Trial Site - Clayton
Recruitment hospital [3] 0 0
Clinical Trial Site - Heidelberg
Recruitment hospital [4] 0 0
Clinical Trial Site - Kingswood
Recruitment hospital [5] 0 0
Clinical Trial Site - Liverpool
Recruitment hospital [6] 0 0
Clinical Trial Site - Nedlands
Recruitment hospital [7] 0 0
Clinical Trial Site - Parkville
Recruitment hospital [8] 0 0
Clinical Trial Site - Perth
Recruitment hospital [9] 0 0
Clinical Trial Site - Westmead
Recruitment hospital [10] 0 0
Clinical Trial Site - Woolloongabba
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
3084 - Heidelberg
Recruitment postcode(s) [4] 0 0
2747 - Kingswood
Recruitment postcode(s) [5] 0 0
2170 - Liverpool
Recruitment postcode(s) [6] 0 0
6109 - Nedlands
Recruitment postcode(s) [7] 0 0
3050 - Parkville
Recruitment postcode(s) [8] 0 0
3052 - Parkville
Recruitment postcode(s) [9] 0 0
6008 - Perth
Recruitment postcode(s) [10] 0 0
2145 - Westmead
Recruitment postcode(s) [11] 0 0
4102 - Woolloongabba
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
District of Columbia
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
New Jersey
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Germany
State/province [9] 0 0
Hannover
Country [10] 0 0
Germany
State/province [10] 0 0
Hannöver
Country [11] 0 0
Germany
State/province [11] 0 0
Kiel
Country [12] 0 0
Germany
State/province [12] 0 0
Luebeck
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT02614898
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02614898