Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12611000498998
Ethics application status
Approved
Date submitted
4/05/2011
Date registered
12/05/2011
Date last updated
7/10/2015
Type of registration
Prospectively registered

Titles & IDs
Public title
Study to evaluate if eculizumab is efficient and safe enough to be used for treatment of adults with atypical hemolytic-uremic syndrome
Scientific title
Adult patients testing eculizumab for atypical hemolytic-uremic syndrome for safety and efficacy
Secondary ID [1] 260106 0
ClinicalTrials.gov reference NCT01194973
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Atypical Hemolytic-Uremic Syndrome 265782 0
Condition category
Condition code
Renal and Urogenital 265949 265949 0 0
Kidney disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Eculizumab 900mg administered intravenously weekly for 4 weeks via infusion over 30 minutes then 1200mg intravenous infusion every two weeks for 26 weeks with the possibility of continued treatment for up to 2 years or until marketing approval.
Intervention code [1] 264522 0
Treatment: Drugs
Comparator / control treatment
None. This is an open label study
Control group
Uncontrolled

Outcomes
Primary outcome [1] 266688 0
Proportion of patients with complete TMA response
Timepoint [1] 266688 0
endpoints to be assessed after 26 weeks treatment based on blood results from routine blood testing.
Secondary outcome [1] 276202 0
duration of complete TMA response
Timepoint [1] 276202 0
endpoints to be assessed after 26 weeks treatment based on blood results from routine blood testing.

Eligibility
Key inclusion criteria
Patients must be willing to give written informed consent.
Patients must be over 18 years of age with a diagnosis of atyipcal hemolytic-uremic syndrome.
Patients exhibit thrombocytopenia, hemolysis and elevated serum creatinine.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Chronic dialysis.
Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Once a patient has been screened and meets all eligibility criteria and has signed the consent form, they can be enrolled in the study. This is an open label study so all patients enrolled will be given active study drug.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Date of first participant enrolment
Anticipated
1/07/2011
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 3570 0
Belgium
State/province [1] 3570 0
Country [2] 3571 0
France
State/province [2] 3571 0
Country [3] 3572 0
Germany
State/province [3] 3572 0
Country [4] 3573 0
Italy
State/province [4] 3573 0
Country [5] 3574 0
Spain
State/province [5] 3574 0
Country [6] 3575 0
Switzerland
State/province [6] 3575 0
Country [7] 3576 0
United States of America
State/province [7] 3576 0
Country [8] 3577 0
United Kingdom
State/province [8] 3577 0

Funding & Sponsors
Funding source category [1] 265011 0
Commercial sector/Industry
Name [1] 265011 0
Alexion Pharmaceuticals Australasia Pty Ltd
Country [1] 265011 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals Australasia Pty Ltd
Address
Suites 226-227, 117 Old Pittwater Road
Brookvale NSW 2100
Country
Australia
Secondary sponsor category [1] 264105 0
None
Name [1] 264105 0
Address [1] 264105 0
Country [1] 264105 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 266983 0
Melbourne Health Human Research Ethics Committee
Ethics committee address [1] 266983 0
Office for Research
Level 6 East, Main Building
300 Grattan Street
The Royal Melbourne Hospital
Parkville VIC 3050
Ethics committee country [1] 266983 0
Australia
Date submitted for ethics approval [1] 266983 0
29/04/2011
Approval date [1] 266983 0
11/07/2011
Ethics approval number [1] 266983 0
2011.092

Summary
Brief summary
Atypical hemolytic-uremic syndrome is a serious, life-threatening rare and chronic disease believed to be caused by genetic mutations. Current treatment for the disease is inadequate.
Due to the uncontrolled complement activation seen in aHUS patients and the previously shown activity of eculizumab to selectively inhibit terminal complement activation, it has been decided to look in to the use of eculizumab in the treatment of serverely affected aHUS patients.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 32566 0
Address 32566 0
Country 32566 0
Phone 32566 0
Fax 32566 0
Email 32566 0
Contact person for public queries
Name 15813 0
Ms Nicola Cowlishaw
Address 15813 0
Regional Monitor
Alexion Pharmaceuticals Australasia Pty Ltd
Suites 226-227, 117 Old Pittwater Road
Brookvale NSW 2100
Country 15813 0
Australia
Phone 15813 0
+61 2 9091 0500
Fax 15813 0
+61 2 9091 0511
Email 15813 0
CowlishawN@alxn.com
Contact person for scientific queries
Name 6741 0
Jean Young
Address 6741 0
Medical Affairs Manager
Alexion Pharmaceuticals Australasia Pty Ltd
Suites 226-227, 117 Old Pittwater Road
Brookvale NSW 2100
Country 6741 0
Australia
Phone 6741 0
+61 2 9091 0500
Fax 6741 0
+61 2 9091 0511
Email 6741 0
YoungJ@alxn.com

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
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