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Trial registered on ANZCTR


Registration number
ACTRN12614000356662
Ethics application status
Approved
Date submitted
24/03/2014
Date registered
3/04/2014
Date last updated
3/04/2014
Type of registration
Retrospectively registered

Titles & IDs
Public title
The effect of ivacaftor (VX-770, registered in Australia as Kalydeco) on exocrine pancreatic function in patients 6 years
and older with cystic fibrosis and at least one copy of the G551D CFTR mutation and who are naive for ivacaftor
Scientific title
The effect of ivacaftor (VX-770, registered in Australia as Kalydeco) on exocrine pancreatic function in patients 6 years
and older with cystic fibrosis and at least one copy of the G551D CFTR mutation and who are naive for ivacaftor
Secondary ID [1] 284328 0
nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 291480 0
Condition category
Condition code
Human Genetics and Inherited Disorders 291845 291845 0 0
Cystic fibrosis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Kalydeco (ivacaftor) 150mg administered orally twice daily for 34 weeks. Tablet counts will be used to assess adherence to study medicine dosage.
Intervention code [1] 289047 0
Treatment: Drugs
Comparator / control treatment
nil
Control group
Uncontrolled

Outcomes
Primary outcome [1] 291760 0
Exocrine Pancreatic Function as measured by faecal fat excretion
Timepoint [1] 291760 0
Assessed at baseline at 16 weeks and at 34 weeks
Secondary outcome [1] 307439 0
body weight measured by scales
Timepoint [1] 307439 0
every 4 weeks from Visit 1 to Visit 10

Eligibility
Key inclusion criteria
Participants 6 years and older with cystic fibrosis and at least one copy of the G551D CFTR mutation, requiring pancreatic enzyme replacement therapy and who are naive for ivacaftor
Minimum age
6 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. known hypersensitivity to ivacaftor, its metabolites, or formulation excipients

2. galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption

3. have undergone organ transplantation

4.Use of any inhibitors (itraconazole, posaconazole, voriconazole, ketoconazole, telithromycin, clarithromycin) or inducers of (rifampicin, rifabutin, phenobarbital, carbamazepine, phenytoin, St Johns Wort) CYP450 3A, within 14 days prior to the first dose of ivacaftor or during the study period

5. hepatic insufficiency defined as Child-Pugh Class C impairment (a score of 10)

6. severe renal impairment, as defined by creatinine clearance less than or equal to 30mL/min or end stage renal disease

7. Pregnant, planning a pregnancy during the course of the study , or breast-feeding

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)
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
Efficacy
Statistical methods / analysis
Observational - no sample size calculation performed as this is a Phase IV study and sufficient medication for 20 participants only has been provided due to the very high cost of the medication

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
3/03/2014
Actual
24/03/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 2231 0
The Children's Hospital at Westmead - Westmead

Funding & Sponsors
Funding source category [1] 288964 0
Commercial sector/Industry
Name [1] 288964 0
Vertex Pharmaceuticals (partial funding)
Country [1] 288964 0
United States of America
Primary sponsor type
Hospital
Name
Sydney Children's Hospitals Network
Address
The Children's Hospital at Westmead
Locked Bag 4001
westmead NSW 2145
Country
Australia
Secondary sponsor category [1] 287645 0
None
Name [1] 287645 0
Address [1] 287645 0
Country [1] 287645 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 290775 0
Sydney Children's Hospitals Network Human Research Ethics Committee
Ethics committee address [1] 290775 0
Ethics committee country [1] 290775 0
Australia
Date submitted for ethics approval [1] 290775 0
Approval date [1] 290775 0
20/12/2013
Ethics approval number [1] 290775 0
230

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 47230 0
Prof Kevin Gaskin
Address 47230 0
James Fairfax Institute of Paediatric Nutrition
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 47230 0
Australia
Phone 47230 0
+61 2 98450000
Fax 47230 0
Email 47230 0
[email protected]
Contact person for public queries
Name 47231 0
Karen McKay
Address 47231 0
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 47231 0
Australia
Phone 47231 0
+61 2 98450000
Fax 47231 0
Email 47231 0
[email protected]
Contact person for scientific queries
Name 47232 0
Karen McKay
Address 47232 0
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 47232 0
Australia
Phone 47232 0
+61 2 98450000
Fax 47232 0
Email 47232 0
[email protected]

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
No additional documents have been identified.