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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02875366
• Date of registration: 15/08/2016
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• 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
• 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
• Date of first enrolment: September 2016
• Target sample size: 70
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02875366
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Other. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 4

Countries of recruitment

Australia, United Kingdom

Key inclusion & exclusion criteria

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

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



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: LUM/IVA

Primary Outcome(s)

Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24 [Time Frame: Baseline, Week 24]

Secondary Outcome(s)

Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in VO2max During CPET at Week 24 [Time Frame: Baseline, Week 24]

Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8) [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in BMI at Week 24 [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Functional VO2 Gain at Week 24 [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24 [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24 [Time Frame: Baseline, Week 24]

Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24 [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Duration of Sleep Time at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Exercise Duration During CPET at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Time Above Sedentary Duration at Week 24 [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24 [Time Frame: Baseline, Week 24]

Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24 [Time Frame: Baseline, Week 24]

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Week 28]

Secondary ID(s)

VX15-809-112

2016-000066-34

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 17/06/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02875366