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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02614898




Registration number
NCT02614898
Ethics application status
Date submitted
24/11/2015
Date registered
25/11/2015
Date last updated
7/11/2017

Titles & IDs
Public title
Eval. of Potential Predictors of Disease Progression in Patients With aHUS Including Genetics, Biomarkers and Treatment
Scientific title
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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Clinical assessments and laboratory tests.

Other: Clinical assessments & laboratory tests - Clinical assessments and laboratory tests for patients already taking an approved therapy Soliris® (eculizumab).


Other interventions: Clinical assessments and laboratory tests.
Clinical assessments and laboratory tests required at a minimum monthly. Frequency is increased at enrollment and any time there is a change in Soliris® regimen. Treatment for aHUS is observational and remains 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 TMA manifestations during eculizumab treatment compared to off- treatment - TMA is defined as one of following:
Hematologic or renal events due to aHUS
Extra-renal clinical signs and symptoms of aHUS
Tissue (eg, kidney transplant) biopsy demonstrating TMA
Timepoint [1] 0 0
Up to 47 Months
Secondary outcome [1] 0 0
Change in estimated Glomerular Filtration Rate (eGFR) over time - Change in estimated Glomerular Filtration Rate (eGFR) over time using the chronic kidney disease-epidemiology (CKD-EPI) formula
Timepoint [1] 0 0
Up to 47 Months
Secondary outcome [2] 0 0
Rate of TMA manifestations in subsets of patients - While patient is on a labeled eculizumab dosing regimen
While patient is on a non-labeled eculizumab dosing regimen (e.g. reduced dose and or reduced frequency)
Timepoint [2] 0 0
Up to 47 Months
Secondary outcome [3] 0 0
Incidence of Plasma Exchange and Plasma Infusion (PE/PI)
Timepoint [3] 0 0
Up to 47 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 LDH levels at least 4 weeks apart

4. Willing, committed, and able to return for ALL clinic visits and complete all
study-related procedures

5. Patient or patient's parent/legal guardian must be willing and able to give written
informed consent. Patient (if minor) must be 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
Gender
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. Patient or patient's parent/legal guardian unable to give written informed consent.
Patient (if minor) unable to give written informed assent (if applicable as determined
by the central Institutional Review Boards/Independent Ethics Committees)

Study design
Purpose of the study
Other
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 4
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Adelaide
Recruitment hospital [2] 0 0
Clinical Trial Site - Cairns
Recruitment hospital [3] 0 0
Clinical Trial Site - Clayton
Recruitment hospital [4] 0 0
Clinical Trial Site - Heidelberg
Recruitment hospital [5] 0 0
Clinical Trial Site - Kingswood
Recruitment hospital [6] 0 0
Clinical Trial Site - Liverpool
Recruitment hospital [7] 0 0
Clinical Trial Site - Nedlands
Recruitment hospital [8] 0 0
Clinical Trial Site - Palmyra
Recruitment hospital [9] 0 0
Clinical Trial Site - Parkville
Recruitment hospital [10] 0 0
Clinical Trial Site - Perth
Recruitment hospital [11] 0 0
Clinical Trial Site - Subiaco
Recruitment hospital [12] 0 0
Clinical Trial Site - Westmead
Recruitment hospital [13] 0 0
Clinical Trial Site - Woolloongabba
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
4870 - Cairns
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
3084 - Heidelberg
Recruitment postcode(s) [5] 0 0
2747 - Kingswood
Recruitment postcode(s) [6] 0 0
2170 - Liverpool
Recruitment postcode(s) [7] 0 0
6109 - Nedlands
Recruitment postcode(s) [8] 0 0
6961 - Palmyra
Recruitment postcode(s) [9] 0 0
3052 - Parkville
Recruitment postcode(s) [10] 0 0
6008 - Perth
Recruitment postcode(s) [11] 0 0
6008 - Subiaco
Recruitment postcode(s) [12] 0 0
2145 - Westmead
Recruitment postcode(s) [13] 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
Hannöver
Country [10] 0 0
Germany
State/province [10] 0 0
Heidelberg
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
Germany
State/province [13] 0 0
Tübingen
Country [14] 0 0
United Kingdom
State/province [14] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a prospective, open-label study with no patient randomization. Treatment for atypical
hemolytic uremic syndrome (aHUS) will remain observational and at the discretion of the
treating physician. The purpose of this study is to assess disease manifestations of
complement mediated thrombotic microangiopathy (TMA) and potential clinical predictors of
disease manifestations and progression in patients with aHUS with or without eculizumab
treatment in the clinical setting.
Trial website
https://clinicaltrials.gov/show/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
Other publications