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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02544451




Registration number
NCT02544451
Ethics application status
Date submitted
1/09/2015
Date registered
9/09/2015
Date last updated
2/10/2018

Titles & IDs
Public title
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Scientific title
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Secondary ID [1] 0 0
VX15-809-110
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 - Lumacaftor

Experimental: Treatment Cohort: lumacaftor/ivacaftor (6 through 11) - Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (subjects aged 6 through 11 years)

Experimental: Treatment Cohort: lumacaftor/ivacaftor (12 and older) - LUM 400 mg q12h/IVA 250 mg q12h (subjects aged 12 years and older)

No Intervention: Observational Cohort - Long-term Follow-up


Treatment: Drugs: Ivacaftor


Treatment: Drugs: Lumacaftor


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

Outcomes
Primary outcome [1] 0 0
Treatment Cohort: Assess safety and tolerability of long term treatment of lumacaftor in combination with ivacaftor, based on treatment emergent adverse events and changes in clinical laboratory values, vital signs, and spirometry
Timepoint [1] 0 0
up to 4 weeks after last dose [last dose = Week 96]
Secondary outcome [1] 0 0
Treatment Cohort: Absolute change in Lung Clearance Index 2.5 (LCI2.5) (subjects from Study 109 and the Study 011B LCI Substudy only)
Timepoint [1] 0 0
from baseline to Week 96
Secondary outcome [2] 0 0
Treatment Cohort: Absolute change in sweat chloride
Timepoint [2] 0 0
from baseline to Week 96
Secondary outcome [3] 0 0
Treatment Cohort: Absolute change in body mass index (BMI)
Timepoint [3] 0 0
from baseline to Week 96
Secondary outcome [4] 0 0
Treatment Cohort: Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score
Timepoint [4] 0 0
from baseline to Week 96
Secondary outcome [5] 0 0
Observational Cohort: Safety, as determined by serious adverse events (SAEs)
Timepoint [5] 0 0
2 years
Secondary outcome [6] 0 0
Treatment Cohort: Absolute change in Lung Clearance Index 5.0 (LCI5.0) (subjects from Study 109 and the Study 011B LCI Substudy only)
Timepoint [6] 0 0
from baseline to Week 96
Secondary outcome [7] 0 0
Treatment Cohort: Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)
Timepoint [7] 0 0
from baseline to Week 96
Secondary outcome [8] 0 0
Treatment Cohort: Relative change in ppFEV1
Timepoint [8] 0 0
from baseline to Week 96
Secondary outcome [9] 0 0
Treatment Cohort: Absolute change in body mass index (BMI)-for-age-z-score
Timepoint [9] 0 0
from baseline to Week 96
Secondary outcome [10] 0 0
Treatment Cohort: Absolute change in weight
Timepoint [10] 0 0
from baseline to Week 96
Secondary outcome [11] 0 0
Treatment Cohort: Absolute change in weight-for-age-z-score
Timepoint [11] 0 0
from baseline to Week 96
Secondary outcome [12] 0 0
Treatment Cohort: Absolute change in height
Timepoint [12] 0 0
from baseline to Week 96
Secondary outcome [13] 0 0
Treatment Cohort: Absolute change in height-for-age-z-score
Timepoint [13] 0 0
from baseline to Week 96
Secondary outcome [14] 0 0
Treatment Cohort: Absolute change in Treatment Satisfaction Questionnaire for Medication (TSQM) domains
Timepoint [14] 0 0
from baseline to Week 96
Secondary outcome [15] 0 0
Treatment Cohort: Time-to-first pulmonary exacerbation (subjects from Study 109 only)
Timepoint [15] 0 0
from baseline to Week 96
Secondary outcome [16] 0 0
Treatment Cohort: Event of having at least 1 pulmonary exacerbation (subjects from Study 109 only)
Timepoint [16] 0 0
to Week 96
Secondary outcome [17] 0 0
Treatment Cohort: Number of pulmonary exacerbations (subjects from Study 109 only)
Timepoint [17] 0 0
from baseline to Week 96
Secondary outcome [18] 0 0
Treatment Cohort: Rate of change in LCI2.5 (subjects from Study 109 and the Study 011B LCI Substudy only)
Timepoint [18] 0 0
from baseline to Week 96
Secondary outcome [19] 0 0
Treatment Cohort: Rate of change in LCI5.0 (subjects from Study 109 and the Study 011B LCI Substudy only)
Timepoint [19] 0 0
from baseline to Week 96
Secondary outcome [20] 0 0
Treatment Cohort: Rate of change in ppFEV1
Timepoint [20] 0 0
from baseline to Week 96

Eligibility
Key inclusion criteria
Subjects entering the Treatment Cohort must meet both of the following criteria:

- Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not
permanently discontinue treatment

- Willing to remain on a stable CF medication through the Safety Follow-up Visit.

Subjects entering the Observational Cohort must meet 1 of the following criteria:

- Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study
drug treatment and the Week 26 Safety Follow up in Study 011B.

- Received at least 4 weeks of study drug and completed visits up to Week 24 of Study
109 or Week 26 of Study 011B.
Minimum age
6 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria (Treatment Cohort Only):

- History of any comorbidity or laboratory abnormality that, in the opinion of the
investigator, might confound the results of the study or pose an additional risk in
administering study drug to the subject (e.g., cirrhosis with portal hypertension).

- Pregnant and nursing females.

- Sexually active subjects of reproductive potential who are not willing to follow the
contraception requirements.

- History of drug intolerance in the prior study that would pose an additional risk to
the subject in the opinion of investigator

- History of poor compliance with study drug and/or procedure in the previous study as
deemed by the investigator.

- Participation in an investigational drug trial (including studies investigating
lumacaftor and/or ivacaftor).

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
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
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
- Herston
Recruitment hospital [3] 0 0
- New Lambton Heights
Recruitment hospital [4] 0 0
- Subiaco
Recruitment hospital [5] 0 0
- Westmead
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment postcode(s) [3] 0 0
- New Lambton Heights
Recruitment postcode(s) [4] 0 0
- Subiaco
Recruitment postcode(s) [5] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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Arizona
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California
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Colorado
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Delaware
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Florida
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Georgia
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Illinois
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Indiana
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Iowa
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Massachusetts
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Minnesota
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Missouri
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Nebraska
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New Hampshire
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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South Carolina
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Texas
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Utah
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Vermont
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Virginia
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Washington
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Wisconsin
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Belgium
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Brussels
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Belgium
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Leuven
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Canada
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British Columbia
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Canada
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Ontario
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Canada
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Quebec
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Denmark
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Copenhagen
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France
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Cedex
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France
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Bordeaux Cedex
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France
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Paris Cedex 15
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France
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Paris
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Germany
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Berlin
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Germany
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Giessen
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Germany
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Hanover
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Germany
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Koeln
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Sweden
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Stockholm
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United Kingdom
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West Yorkshire
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United Kingdom
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Belfast
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Edinburgh
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United Kingdom
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London

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

Ethics approval
Ethics application status

Summary
Brief summary
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic
fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator
(CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233).
Study 110 is designed to evaluate the safety and efficacy of long term treatment of
lumacaftor in combination with ivacaftor.
Trial website
https://clinicaltrials.gov/show/NCT02544451
Trial related presentations / publications
Public notes

Contacts
Principal investigator
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Address 0 0
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Email 0 0
Contact person for public queries
Name 0 0
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Contact person for scientific queries

Summary results
Other publications