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NCT Number: NCT02875366

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

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Melbourne, Victoria, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

LUM/IVA

Drug

Other names: Lumacaftor/Ivacaftor

Placebo

Drug

Primary outcomes

  1. Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24

    Time frame: Baseline, Week 24

    CPET was used to assess change in exercise tolerance, as measured by VO2max.

Secondary outcomes

  1. Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24

    Time frame: Baseline, Week 24

    Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.

  2. Absolute Change From Baseline in Exercise Duration During CPET at Week 24

    Time frame: Baseline, Week 24

    Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.

  3. Absolute Change From Baseline in VO2max During CPET at Week 24

    Time frame: Baseline, Week 24

    CPET was used to assess change in exercise tolerance, as measured by VO2max.

  4. Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24

    Time frame: Baseline, Week 24

    Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.

  5. Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24

    Time frame: Baseline, Week 24

    Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.

  6. Absolute Change From Baseline in Functional VO2 Gain at Week 24

    Time frame: Baseline, Week 24

  7. Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24

    Time frame: Baseline, Week 24

  8. Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24

    Time frame: Baseline, Week 24

  9. Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24

    Time frame: Baseline, Week 24

  10. Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24

    Time frame: Baseline, Week 24

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  11. Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24

    Time frame: Baseline, Week 24

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  12. Absolute Change From Baseline in Body Mass Index (BMI) at Week 24

    Time frame: Baseline, Week 24

    BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2).

  13. Relative (Percent) Change From Baseline in BMI at Week 24

    Time frame: Baseline, Week 24

    BMI was defined as weight in kg divided by height in m^2.

  14. Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24

    Time frame: Baseline, Week 24

    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.

  15. Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)

    Time frame: Baseline, Week 24

    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.

  16. Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores

    Time frame: Baseline, Week 24

    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.

  17. Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24

    Time frame: Baseline, Week 24

    Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activity counts.

  18. Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24

    Time frame: Baseline, Week 24

    Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activities.

  19. Absolute Change From Baseline in Duration of Sleep Time at Week 24

    Time frame: Baseline, Week 24

    Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.

  20. Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24

    Time frame: Baseline, Week 24

    Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.

  21. Absolute Change From Baseline in Time Above Sedentary Duration at Week 24

    Time frame: Baseline, Week 24

    Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.

  22. Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24

    Time frame: Baseline, Week 24

    Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.

  23. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 28

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study 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

Important dates

Study start
2016
Primary completion
2017
Study completion
2017
First posted
Aug 23, 2016
Registry last updated
Jun 17, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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