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Trial record 1 of 1 for:    VX15-809-110
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Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

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ClinicalTrials.gov Identifier: NCT02544451
Recruitment Status : Completed
First Posted : September 9, 2015
Results First Posted : October 29, 2019
Last Update Posted : May 24, 2021
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: LUM/IVA Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 246 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : August 2015
Actual Primary Completion Date : August 2018
Actual Study Completion Date : April 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: Treatment Period 1: LUM/IVA to LUM/IVA Drug: LUM/IVA

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

Experimental: Treatment Period 1: Placebo (PBO) to LUM/IVA Drug: LUM/IVA

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

No Intervention: Treatment Period 1: Observational Cohort
Experimental: Treatment Period 2: LUM/IVA Drug: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Other Names:
  • VX-809/VX-770
  • lumacaftor/ivacaftor




Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02544451


Locations
Show Show 61 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] September 15, 2015
Statistical Analysis Plan  [PDF] July 20, 2018

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02544451    
Other Study ID Numbers: VX15-809-110
First Posted: September 9, 2015    Key Record Dates
Results First Posted: October 29, 2019
Last Update Posted: May 24, 2021
Last Verified: February 2021
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action