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NCT Number: NCT02544451

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.

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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

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Parkville, 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

Subjects entering the Treatment Cohort must meet both of the following criteria:

  • Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
  • Willing to remain on a stable CF medication through the Safety Follow-up Visit.

Subjects entering the Observational Cohort must meet 1 of the following criteria:

  • Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
  • Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.

Exclusion criteria

(Treatment Cohort Only):

  • History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension).
  • Pregnant and nursing females.
  • Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
  • History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
  • History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
  • Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).

Treatment and study plan

LUM/IVA

Drug

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).

LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

Other names: VX-809/VX-770, lumacaftor/ivacaftor

Primary outcomes

  1. Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 100

Secondary outcomes

  1. Absolute Change in Lung Clearance Index (LCI) 2.5

    Time frame: From Parent Study Baseline at Week 96

    LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

  2. Absolute Change in Sweat Chloride

    Time frame: From Parent Study Baseline at Week 96

    Sweat samples were collected using an approved collection device.

  3. Absolute Change in Body Mass Index (BMI)

    Time frame: From Parent Study Baseline at Week 96

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

  4. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

    Time frame: From Parent Study Baseline at Week 96

    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  5. Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 100

  6. Absolute Change in LCI 5.0

    Time frame: From Parent Study Baseline at Week 96

    LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.

  7. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

    Time frame: From Parent Study Baseline at Week 96

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

  8. Relative Change in ppFEV1

    Time frame: From Parent Study Baseline at Week 96

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

  9. Absolute Change in BMI-for-age Z-score

    Time frame: From Parent Study Baseline at Week 96

    BMI was defined as weight in kilograms divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

  10. Absolute Change in Weight

    Time frame: From Parent Study Baseline at Week 96

  11. Absolute Change in Weight-for-age Z-score

    Time frame: From Parent Study Baseline at Week 96

    z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

  12. Absolute Change in Height

    Time frame: From Parent Study Baseline at Week 96

  13. Absolute Change in Height-for-age Z-score

    Time frame: From Parent Study Baseline at Week 96

    z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

  14. Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score

    Time frame: From Parent Study Baseline at Week 96

    The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction.

  15. Time-to-first Pulmonary Exacerbation

    Time frame: From Parent Study Baseline through Week 96

    Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.

  16. Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event

    Time frame: From Parent Study Baseline through Week 96

    Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.

  17. Number of Pulmonary Exacerbation Events Per Patient-year

    Time frame: From Parent Study Baseline through Week 96

    Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.

  18. Rate of Change in LCI 2.5

    Time frame: Day 15 after first dose of LUM/IVA through Week 96

    Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.

  19. Rate of Change in LCI 5.0

    Time frame: Day 15 after first dose of LUM/IVA through Week 96

    Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.

  20. Rate of Change in ppFEV1

    Time frame: Day 15 after first dose of LUM/IVA through Week 96

    Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.

  21. Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 168

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Important dates

Study start
2015
Primary completion
2018
Study completion
2020
First posted
Sep 9, 2015
Registry last updated
May 24, 2021

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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