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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01807923




Registration number
NCT01807923
Ethics application status
Date submitted
4/03/2013
Date registered
8/03/2013
Date last updated
31/08/2015

Titles & IDs
Public title
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
Scientific title
A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Secondary ID [1] 0 0
VX12-809-103
Universal Trial Number (UTN)
Trial acronym
TRAFFIC
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation 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 - Lumacaftor Plus Ivacaftor Combination
Treatment: Drugs - Ivacaftor
Treatment: Drugs - Placebo

Placebo Comparator: Placebo - Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24.

Experimental: LUM 600 mg qd/IVA 250 mg q12h - LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24.

Experimental: LUM 400 mg q12h/ IVA 250 mg q12h - LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.


Treatment: Drugs: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet

Treatment: Drugs: Ivacaftor
Film-coated tablet

Treatment: Drugs: Placebo
Matching placebo tablet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24
Timepoint [1] 0 0
Baseline, Week 16 and 24
Secondary outcome [1] 0 0
Relative Change From Baseline in Percent Predicted FEV1 at Week 24
Timepoint [1] 0 0
Baseline, Week 16 and 24
Secondary outcome [2] 0 0
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Timepoint [2] 0 0
Baseline, Week 24
Secondary outcome [3] 0 0
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
Timepoint [3] 0 0
Baseline, Week 24
Secondary outcome [4] 0 0
Percentage of Participants With Response Based on Percent Predicted FEV1
Timepoint [4] 0 0
Week 16 and 24
Secondary outcome [5] 0 0
Number of Pulmonary Exacerbation Events
Timepoint [5] 0 0
through Week 24
Secondary outcome [6] 0 0
Absolute Change From Baseline in Weight at Week 24
Timepoint [6] 0 0
Baseline, Week 24
Secondary outcome [7] 0 0
Absolute Change From Baseline in BMI-for-age Z-score at Week 24
Timepoint [7] 0 0
Baseline, Week 24
Secondary outcome [8] 0 0
Time-to-First Pulmonary Exacerbation
Timepoint [8] 0 0
through Week 24
Secondary outcome [9] 0 0
Percentage of Participants With At Least 1 Pulmonary Exacerbation Event
Timepoint [9] 0 0
through Week 24
Secondary outcome [10] 0 0
Absolute Change From Baseline in Euro Quality of Life Scale (EuroQol) 5-Dimension-3 Level (EQ-5D-3L) Index Score at Week 24
Timepoint [10] 0 0
Baseline, Week 24
Secondary outcome [11] 0 0
Absolute Change From Baseline in EQ-5D-3L VAS Score at Week 24
Timepoint [11] 0 0
Baseline, Week 24
Secondary outcome [12] 0 0
Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Scores at Week 24
Timepoint [12] 0 0
Baseline, Week 24
Secondary outcome [13] 0 0
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Treatment-Emergent Adverse Events (SAEs)
Timepoint [13] 0 0
up to Week 28
Secondary outcome [14] 0 0
Pre-dose Concentration (Ctrough), Average Pre-dose Concentration (Ctrough,Avg), 3 to 6 Hours Post-dose Concentration (C3-6h), and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Avg)
Timepoint [14] 0 0
For C3-6h: 3 to 6 hours after morning dose on Day 1 and 15, Week 4 and 8; For C3-6h,avg 3 to 6 hours after morning dose on Day 15, Week 4 and 8; For Ctrough and Ctrough,avg: before morning dose on Week 4, 8, and 16

Eligibility
Key inclusion criteria
- Confirmed diagnosis of CF

- Homozygous for the F508del CFTR mutation

- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent
(%) and less than or equal to (=<) 90% of predicted normal for age, sex, and height

- Willing to remain on a stable CF medication regimen through Week 24 or, if applicable,
the Safety Follow up Visit
Minimum age
12 Years
Maximum age
No limit
Sex
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 first dose
of study drug

- History of solid organ or hematological transplantation

- History of alcohol or drug abuse in the past year

- Ongoing or prior participation in an investigational drug study (including studies
investigating lumacaftor and/or ivacaftor) within 30 days of screening

- Use of strong inhibitors, moderate inducers or strong inducers of Cytochrome P450 3A
(CYP3A) within 14 days before Day 1 of dosing

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

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/05/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/04/2014
Sample size
Target
Accrual to date
Final
559
Recruitment in Australia
Recruitment state(s)
NSW,SA
Recruitment hospital [1] 0 0
- Broadmeadow
Recruitment hospital [2] 0 0
- Westmead
Recruitment hospital [3] 0 0
- Adelaide
Recruitment postcode(s) [1] 0 0
- Broadmeadow
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment postcode(s) [3] 0 0
- Adelaide
Recruitment outside Australia
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United States of America
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Alabama
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Alaska
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Arizona
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Georgia
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Idaho
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Illinois
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Belfast

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

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective of the study was to evaluate the efficacy of lumacaftor in combination
with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF)
who are homozygous for the F508del mutation on the CF transmembrane conductance regulator
(CFTR) gene.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01807923
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
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Phone 0 0
Fax 0 0
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Contact person for scientific queries