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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02742519




Registration number
NCT02742519
Ethics application status
Date submitted
14/04/2016
Date registered
19/04/2016
Date last updated
19/11/2018

Titles & IDs
Public title
A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
Scientific title
A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
Secondary ID [1] 0 0
2015-001267-39
Secondary ID [2] 0 0
VX15-770-123
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
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
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

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

Experimental: Part 1-Sequence 1 - ivacaftor in Treatment Period 1 ?washout?placebo in Treatment Period 2

Experimental: Part 1 - Sequence 2 - placebo in Treatment Period 1?washout?ivacaftor in Treatment Period 2

Experimental: Part 2: ivacaftor - open label period


Treatment: Drugs: ivacaftor


Treatment: Drugs: Placebo


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

Outcomes
Primary outcome [1] 0 0
Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5) - LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Timepoint [1] 0 0
Baseline Through Week 8 for each treatment period, Up to 24 Weeks
Secondary outcome [1] 0 0
Absolute Change From Baseline in Immunoreactive Trypsinogen Levels at Week 8 - Serum samples were collected for evaluation of change in immunoreactive trypsinogen levels at Week 8.
Timepoint [1] 0 0
Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary outcome [2] 0 0
Absolute Change From Baseline in Fecal Elastase-1 Levels at Week 8 - Fecal elastase-1 was used clinically to diagnose pancreatic exocrine insufficiency in participants with cystic fibrosis.
Timepoint [2] 0 0
Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary outcome [3] 0 0
Absolute Change From Baseline in Weight at Week 8
Timepoint [3] 0 0
Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary outcome [4] 0 0
Absolute Change From Baseline in Body Mass Index (BMI) at Week 8 - BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).
Timepoint [4] 0 0
Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary outcome [5] 0 0
Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [5] 0 0
Baseline up to Month 15

Eligibility
Key inclusion criteria
- Male or female with confirmed diagnosis of CF.

- Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R,
S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.

- Hematology, serum chemistry, and coagulation at Screening with no clinically
significant abnormalities or concomitant diagnosis that would interfere with the LCI
and CT scan study assessments, as judged by the investigator.
Minimum age
3 Years
Maximum age
5 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1

- Any clinically significant laboratory abnormalities at the Screening Visit that would
interfere with the study assessments or pose an undue risk for the subject (in the
opinion of the investigator)

- Abnormal liver function, at Screening, defined as =3 × upper limit of normal (ULN), of
any 3 or more of the following: serum aspartate transaminase (AST), serum alanine
transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase
(ALP), and total bilirubin

- History of solid organ or hematological transplantation

- Any clinically significant "non-CF-related" illness within 2 weeks before Day 1

- Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A
within 2 weeks before Day 1

- Participation in a clinical study involving administration of either an
investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever
is longer or as determined by the local requirements) before Screening

Study design
Purpose of the study
Treatment
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 receiving the treatment/s


The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
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
- Parkville
Recruitment hospital [2] 0 0
- South Brisbane
Recruitment hospital [3] 0 0
- Subiaco
Recruitment hospital [4] 0 0
- Westmead
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment postcode(s) [3] 0 0
- Subiaco
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Ontario
Country [2] 0 0
United Kingdom
State/province [2] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI),
in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance
regulator (CFTR) gating mutation
Trial website
https://clinicaltrials.gov/show/NCT02742519
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
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02742519