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
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
2 other identifiers
interventional
70
2 countries
12
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_4
Started Sep 2016
Shorter than P25 for phase_4
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 15, 2016
CompletedFirst Posted
Study publicly available on registry
August 23, 2016
CompletedStudy Start
First participant enrolled
September 1, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2017
CompletedResults Posted
Study results publicly available
June 17, 2019
CompletedJune 17, 2019
March 1, 2019
1 year
August 15, 2016
September 30, 2018
March 18, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24
CPET was used to assess change in exercise tolerance, as measured by VO2max.
Baseline, Week 24
Secondary Outcomes (23)
Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24
Baseline, Week 24
Absolute Change From Baseline in Exercise Duration During CPET at Week 24
Baseline, Week 24
Absolute Change From Baseline in VO2max During CPET at Week 24
Baseline, Week 24
Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24
Baseline, Week 24
Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24
Baseline, Week 24
- +18 more secondary outcomes
Study Arms (2)
Placebo
PLACEBO COMPARATORPlacebo matched to LUM/IVA fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
LUM/IVA
EXPERIMENTALLUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
Unknown Facility
Melbourne, Victoria, Australia
Unknown Facility
Parkville, Victoria, Australia
Unknown Facility
Adelaide, Australia
Unknown Facility
Camperdown, Australia
Unknown Facility
Clayton, Australia
Unknown Facility
Nedlands, Australia
Unknown Facility
New Lambton Heights, Australia
Unknown Facility
Randwick, Australia
Unknown Facility
South Brisbane, Australia
Unknown Facility
Subiaco, Australia
Unknown Facility
Westmead, Australia
Unknown Facility
Edinburgh, United Kingdom
Related Publications (1)
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.
PMID: 33358691DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- OTHER
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 15, 2016
First Posted
August 23, 2016
Study Start
September 1, 2016
Primary Completion
September 1, 2017
Study Completion
October 1, 2017
Last Updated
June 17, 2019
Results First Posted
June 17, 2019
Record last verified: 2019-03