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Trial registered on ANZCTR


Registration number
ACTRN12611000461998
Ethics application status
Approved
Date submitted
28/04/2011
Date registered
4/05/2011
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 children with atypical hemolytic-uremic syndrome
Scientific title
Paediatric patients testing eculizumab for atypical hemolytic-uremic syndrome for safety and efficacy.
Secondary ID [1] 260072 0
none
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Atypical Hemolytic-Uremic Syndrome 265747 0
Condition category
Condition code
Renal and Urogenital 265891 265891 0 0
Kidney disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Eculizumab 30mL vials with a solution concentraion of 10mg/mL.
Given by intravenous infusion over 1-4 hours with the dose depending on body weight. Dosing is weekly infusion for 4 weeks and then IV infusion every 2 weeks. There will be 26 weeks of treatment to meet the study defined endpoints. Patients will be allowed to continue to receive the drug for 2 more year or until the product is registred in Australia unless the study is prematurely discontinued.
Intervention code [1] 264493 0
Treatment: Drugs
Comparator / control treatment
No comparator or control treatment. This is an open label study.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 266652 0
Primary Efficacy Endpoint: proportion of patients with complete TMA response as evidenced by normalisation of hematological parameters and improvement in serum creatinine from baseline.
Timepoint [1] 266652 0
Endpoints will be assessed after 26 weeks treatment for each patient.
Primary outcome [2] 266687 0
Primary Safety Endpoint: safety assesments are based on routine physical examinations, vitals signs, AEs, lab data and ECGs.
Timepoint [2] 266687 0
Endpoints will be assessed after 26 weeks treatment for each patient.
Secondary outcome [1] 276146 0
duration of complete TMA response
Timepoint [1] 276146 0
All endpoints will be assessed after 26 weeks treatment.
Secondary outcome [2] 276200 0
time to complete TMA response
Timepoint [2] 276200 0
All endpoints will be assessed after 26 weeks treatment.
Secondary outcome [3] 276201 0
assessment of hematologic response.
Timepoint [3] 276201 0
All endpoints will be assessed after 26 weeks treatment.

Eligibility
Key inclusion criteria
Signed informed consent.
Patients from 1 month to 18 years diagnosed with aHUS
Minimum age
1 Months
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Typical hemolytic-uremic syndrome (HUS), malignancy within 5 years of screening, HUS related to infection or bone marrow transplant or vitamin B12 deficiency, systemic lupus erythematosus (SLE), meningococcal/pneumococcal disease history.

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
Active, not recruiting
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 postcode(s) [1] 3961 0
3052
Recruitment outside Australia
Country [1] 3426 0
Austria
State/province [1] 3426 0
Country [2] 3427 0
France
State/province [2] 3427 0
Country [3] 3428 0
Germany
State/province [3] 3428 0
Country [4] 3429 0
United Kingdom
State/province [4] 3429 0
Country [5] 3430 0
Italy
State/province [5] 3430 0
Country [6] 3431 0
Netherlands
State/province [6] 3431 0
Country [7] 3432 0
United States of America
State/province [7] 3432 0

Funding & Sponsors
Funding source category [1] 264967 0
Commercial sector/Industry
Name [1] 264967 0
Alexion Pharmaceuticals Australasia Pty Ltd
Address [1] 264967 0
Suites 226-227, 117 Old Pittwater Road
Brookvale NSW Australia 2100
Country [1] 264967 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals Australasia Pty Ltd
Address
Suites 226-227, 117 Old Pittwater Road
Brookvale NSW Australia 2100
Country
Australia
Secondary sponsor category [1] 264060 0
None
Name [1] 264060 0
Address [1] 264060 0
Country [1] 264060 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 266931 0
The Royal Children's Hospital Human Research Ethics Committee
Ethics committee address [1] 266931 0
The Royal Children's Hospital Melbourne
50 Flemington Road
Parkville 3052 Victoria
Ethics committee country [1] 266931 0
Australia
Date submitted for ethics approval [1] 266931 0
31/05/2011
Approval date [1] 266931 0
09/09/2011
Ethics approval number [1] 266931 0
Ethics committee name [2] 269281 0
Women's and Children's Health Network
Ethics committee address [2] 269281 0
Women's and Children's Hospital, 72 King William Road, North Adelaide, SA 5006
Ethics committee country [2] 269281 0
Australia
Date submitted for ethics approval [2] 269281 0
20/05/2011
Approval date [2] 269281 0
26/07/2011
Ethics approval number [2] 269281 0
New ethics HREC. Please modify.

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 severely affected aHUS patients.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 32544 0
Address 32544 0
Country 32544 0
Phone 32544 0
Fax 32544 0
Email 32544 0
Contact person for public queries
Name 15791 0
Ms Nicola Cowlishaw
Address 15791 0
Regional Monitor
Alexion Pharmaceuticals Australasia Pty Ltd
Suites 226-227, 117 Old Pittwater Road
Brookvale NSW Australia 2100
Country 15791 0
Australia
Phone 15791 0
+61 2 9091 0500
Fax 15791 0
+61 2 9091 0511
Email 15791 0
CowlishawN@alxn.com
Contact person for scientific queries
Name 6719 0
Jean Young
Address 6719 0
Medical Affairs Manager
Alexion Pharmaceuticals Australasia Pty Ltd
Suites 226-227, 117 Old Pittwater Road
Brookvale NSW Australia 2100
Country 6719 0
Australia
Phone 6719 0
+61 2 9091 0500
Fax 6719 0
+61 2 9091 0511
Email 6719 0
YoungJ@alxn.com

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary