Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02875366
Registration number
NCT02875366
Ethics application status
Date submitted
15/08/2016
Date registered
23/08/2016
Date last updated
17/06/2019
Titles & IDs
Public title
A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
Query!
Scientific title
A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Study of the Effect of Lumacaftor/Ivacaftor Combination Therapy on Exercise Tolerance in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Query!
Secondary ID [1]
0
0
2016-000066-34
Query!
Secondary ID [2]
0
0
VX15-809-112
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Cystic fibrosis
Query!
Respiratory
0
0
0
0
Query!
Other respiratory disorders / diseases
Query!
Oral and Gastrointestinal
0
0
0
0
Query!
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Connective tissue diseases
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Placebo comparator: Placebo - Placebo matched to LUM/IVA fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
Experimental: LUM/IVA - LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24
Query!
Assessment method [1]
0
0
CPET was used to assess change in exercise tolerance, as measured by VO2max.
Query!
Timepoint [1]
0
0
Baseline, Week 24
Query!
Secondary outcome [1]
0
0
Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24
Query!
Assessment method [1]
0
0
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
Query!
Timepoint [1]
0
0
Baseline, Week 24
Query!
Secondary outcome [2]
0
0
Absolute Change From Baseline in Exercise Duration During CPET at Week 24
Query!
Assessment method [2]
0
0
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
Query!
Timepoint [2]
0
0
Baseline, Week 24
Query!
Secondary outcome [3]
0
0
Absolute Change From Baseline in VO2max During CPET at Week 24
Query!
Assessment method [3]
0
0
CPET was used to assess change in exercise tolerance, as measured by VO2max.
Query!
Timepoint [3]
0
0
Baseline, Week 24
Query!
Secondary outcome [4]
0
0
Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24
Query!
Assessment method [4]
0
0
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
Query!
Timepoint [4]
0
0
Baseline, Week 24
Query!
Secondary outcome [5]
0
0
Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24
Query!
Assessment method [5]
0
0
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
Query!
Timepoint [5]
0
0
Baseline, Week 24
Query!
Secondary outcome [6]
0
0
Absolute Change From Baseline in Functional VO2 Gain at Week 24
Query!
Assessment method [6]
0
0
Query!
Timepoint [6]
0
0
Baseline, Week 24
Query!
Secondary outcome [7]
0
0
Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
Baseline, Week 24
Query!
Secondary outcome [8]
0
0
Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
Query!
Assessment method [8]
0
0
Query!
Timepoint [8]
0
0
Baseline, Week 24
Query!
Secondary outcome [9]
0
0
Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
Baseline, Week 24
Query!
Secondary outcome [10]
0
0
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
Query!
Assessment method [10]
0
0
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Query!
Timepoint [10]
0
0
Baseline, Week 24
Query!
Secondary outcome [11]
0
0
Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
Query!
Assessment method [11]
0
0
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Query!
Timepoint [11]
0
0
Baseline, Week 24
Query!
Secondary outcome [12]
0
0
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Query!
Assessment method [12]
0
0
BMI was defined as weight in kilograms (kg) divided by height in square meter (m\^2).
Query!
Timepoint [12]
0
0
Baseline, Week 24
Query!
Secondary outcome [13]
0
0
Relative (Percent) Change From Baseline in BMI at Week 24
Query!
Assessment method [13]
0
0
BMI was defined as weight in kg divided by height in m\^2.
Query!
Timepoint [13]
0
0
Baseline, Week 24
Query!
Secondary outcome [14]
0
0
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
Query!
Assessment method [14]
0
0
The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.
Query!
Timepoint [14]
0
0
Baseline, Week 24
Query!
Secondary outcome [15]
0
0
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Query!
Assessment method [15]
0
0
The PHQ-8 is an eight item self-reported measure of depression. Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day). Total score is the sum of individual eight items and ranges from 0 to 24, with higher scores indicating more severe depression symptoms. Total score of 0 to 5 indicates none to minimal depression, 6 to 10 indicates mild depression, 11 to 15 indicates moderate depression, 16 to 20 indicates moderately severe depression and 21 to 24 indicates severe depression.
Query!
Timepoint [15]
0
0
Baseline, Week 24
Query!
Secondary outcome [16]
0
0
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Query!
Assessment method [16]
0
0
The GAD-7 is a seven item, self-reported measurement of GAD severity. Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day). Total score is the sum of individual seven items and ranges from 0 to 21, with higher scores indicating more severe anxiety symptoms. Total score of 0 to 5 indicates none to minimal anxiety, 6 to 10 indicates mild anxiety, 11 to 15 indicates moderate anxiety, 16 to 21 indicates severe anxiety.
Query!
Timepoint [16]
0
0
Baseline, Week 24
Query!
Secondary outcome [17]
0
0
Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24
Query!
Assessment method [17]
0
0
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activity counts.
Query!
Timepoint [17]
0
0
Baseline, Week 24
Query!
Secondary outcome [18]
0
0
Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24
Query!
Assessment method [18]
0
0
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activities.
Query!
Timepoint [18]
0
0
Baseline, Week 24
Query!
Secondary outcome [19]
0
0
Absolute Change From Baseline in Duration of Sleep Time at Week 24
Query!
Assessment method [19]
0
0
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
Query!
Timepoint [19]
0
0
Baseline, Week 24
Query!
Secondary outcome [20]
0
0
Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24
Query!
Assessment method [20]
0
0
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
Query!
Timepoint [20]
0
0
Baseline, Week 24
Query!
Secondary outcome [21]
0
0
Absolute Change From Baseline in Time Above Sedentary Duration at Week 24
Query!
Assessment method [21]
0
0
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
Query!
Timepoint [21]
0
0
Baseline, Week 24
Query!
Secondary outcome [22]
0
0
Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24
Query!
Assessment method [22]
0
0
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
Query!
Timepoint [22]
0
0
Baseline, Week 24
Query!
Secondary outcome [23]
0
0
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Query!
Assessment method [23]
0
0
Query!
Timepoint [23]
0
0
Day 1 up to Week 28
Query!
Eligibility
Key inclusion criteria
* Homozygous for the F508del-CFTR mutation
* Confirmed diagnosis of CF defined as a sweat chloride value =60 mmol/L by quantitative pilocarpine iontophoresis
* Stable CF disease as judged by the investigator
* Forced expiratory volume in 1 second (FEV1) at least 40% and not greater than 90% of predicted
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* History of any comorbidity that might confound the results of the study, interfere with the use of cardiopulmonary exercise tests (CPETs) as an assessment, or pose an additional risk in administering study drug to the subject
* Any previous exposure to LUM or IVA
* History of cardiac arrhythmia, ischemic heart disease, congestive heart failure, or other clinically significant cardiac condition, or medical condition requiring chronic use of a beta blocker, non-dihydropyridine calcium channel blocker, or other cardiac medication known to affect exercise tolerance
* History of solid organ or hematological transplantation
* For subjects under 18 years of age at Screening, except those who have had bilateral lens removal, selected findings on a screening ophthalmologic examination will be exclusionary
* Using or expected to require any concomitant medication that is prohibited in this study
* History of alcohol or drug abuse, as deemed by the investigator, in the past year, including but not limited to cannabis, cocaine, and opiates
* Participation in an investigational drug study within 30 days before the Screening Visit
* Pregnant or nursing females; males with a female partner who is pregnant or nursing
* Colonization with organisms associated with a more rapid decline in pulmonary status
Query!
Study design
Purpose of the study
Other
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 4
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/09/2016
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/10/2017
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
70
Query!
Recruitment in Australia
Recruitment state(s)
VIC
Query!
Recruitment hospital [1]
0
0
- Melbourne
Query!
Recruitment hospital [2]
0
0
- Parkville
Query!
Recruitment hospital [3]
0
0
- Adelaide
Query!
Recruitment hospital [4]
0
0
- Camperdown
Query!
Recruitment hospital [5]
0
0
- Clayton
Query!
Recruitment hospital [6]
0
0
- Nedlands
Query!
Recruitment hospital [7]
0
0
- New Lambton Heights
Query!
Recruitment hospital [8]
0
0
- Randwick
Query!
Recruitment hospital [9]
0
0
- South Brisbane
Query!
Recruitment hospital [10]
0
0
- Subiaco
Query!
Recruitment hospital [11]
0
0
- Westmead
Query!
Recruitment postcode(s) [1]
0
0
- Melbourne
Query!
Recruitment postcode(s) [2]
0
0
- Parkville
Query!
Recruitment postcode(s) [3]
0
0
- Adelaide
Query!
Recruitment postcode(s) [4]
0
0
- Camperdown
Query!
Recruitment postcode(s) [5]
0
0
- Clayton
Query!
Recruitment postcode(s) [6]
0
0
- Nedlands
Query!
Recruitment postcode(s) [7]
0
0
- New Lambton Heights
Query!
Recruitment postcode(s) [8]
0
0
- Randwick
Query!
Recruitment postcode(s) [9]
0
0
- South Brisbane
Query!
Recruitment postcode(s) [10]
0
0
- Subiaco
Query!
Recruitment postcode(s) [11]
0
0
- Westmead
Query!
Recruitment outside Australia
Country [1]
0
0
United Kingdom
Query!
State/province [1]
0
0
Edinburgh
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Vertex Pharmaceuticals Incorporated
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation. This study is designed to evaluate the effect of LUM/IVA on exercise tolerance in subjects with CF, homozygous for the F508del-CFTR mutation.
Query!
Trial website
https://clinicaltrials.gov/study/NCT02875366
Query!
Trial related presentations / publications
Wilson J, You X, Ellis M, Urquhart DS, Jha L, Duncan M, Tian S, Harris RA, Kotsimbos T, Keating D. VO2max as an exercise tolerance endpoint in people with cystic fibrosis: Lessons from a lumacaftor/ivacaftor trial. J Cyst Fibros. 2021 May;20(3):499-505. doi: 10.1016/j.jcf.2020.12.006. Epub 2020 Dec 24.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/66/NCT02875366/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/66/NCT02875366/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02875366
Download to PDF