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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01937325




Registration number
NCT01937325
Ethics application status
Date submitted
4/09/2013
Date registered
9/09/2013
Date last updated
25/07/2016

Titles & IDs
Public title
CPET in CF Patients With One G551D Mutation Taking VX770
Scientific title
Airway Infection, Inflammatory Markers and Exercise Capacity in Patients With Cystic Fibrosis and at Least One G551D Mutation Taking VX770 (Ivacaftor)
Secondary ID [1] 0 0
CPET in CF
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ivacaftor
Treatment: Drugs - placebo

Active Comparator: Ivacaftor - 150mg orally twice daily

Placebo Comparator: Placebo - Matching placebo


Treatment: Drugs: ivacaftor
active arm

Treatment: Drugs: placebo
active arm

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
exercise capacity - Respiratory exercise testing, including spirometry and V02 max.
Timepoint [1] 0 0
one month, 3 months
Secondary outcome [1] 0 0
Inflammatory profile - Cytokine levels (IL-1ß, IL-6, TNFa, IL-8, VEGF & Activin A) determined using cytometric bead analysis and / or ELISA
Timepoint [1] 0 0
One month, 3 months

Eligibility
Key inclusion criteria
- All participants will have CF proven based on established criteria (sweat test,
genotype and phenotype).

- All participants will have at least one copy of the G551D mutation.

- All will be able to perform an exercise study and complete study questionnaires and
assessments.

- Age range will be between 16 and 75 years of age.

- Lung function inclusion will be above 25% predicted FEV1.
Minimum age
16 Years
Maximum age
70 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Participants will not be included if they are unable to complete study assessments or
have had a known adverse reaction to Ivacaftor.

- Female participants will be excluded if found to return a positive pregnancy test at
screening.

- Participants will be excluded if using St. John's Wort or rifampicin (strong CYP3A
inducers).

- Participants with significant liver dysfunction will be excluded (ALT or ALT above 5
times upper limit of normal).

Study design
Purpose of the study
Basic Science
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 4
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Unknown status
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)
VIC
Recruitment hospital [1] 0 0
The Alfred - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Other
Name
The Alfred
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Ivacaftor will restore CFTR function in treated CF patients with the G551D mutation.
Improvement in ventilation, salt balance and well-being will contribute to better exercise
capacity at all levels of lung function. While potential improvements may be variable across
the spectrum of lung function, even small gains at low levels of FEV1 may have significant
benefit for some subjects.
Trial website
https://clinicaltrials.gov/show/NCT01937325
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
John Wilson
Address 0 0
Alfred Health and Monash University
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/show/NCT01937325