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A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis Who Have an F508del-CFTR Mutation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02565914
Recruitment Status : Active, not recruiting
First Posted : October 1, 2015
Results First Posted : June 16, 2020
Last Update Posted : June 16, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE September 18, 2015
First Posted Date  ICMJE October 1, 2015
Results First Submitted Date  ICMJE May 28, 2020
Results First Posted Date  ICMJE June 16, 2020
Last Update Posted Date June 16, 2020
Actual Study Start Date  ICMJE August 2015
Actual Primary Completion Date May 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 28, 2020)
Part A: 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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: September 29, 2015)
Safety and tolerability of long-term treatment of VX-661 in combination with ivacaftor based on adverse events (AEs), ophthalmologic exams, clinical laboratory values, standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry [ Time Frame: from baseline through Study Completion (up to 3 years) ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 28, 2020)
  • Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline.
  • Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Number of Pulmonary Exacerbation (PEx) Events for 106/110 PEx Analysis Set [ Time Frame: From Baseline up to Study 110 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Number of Pulmonary Exacerbation (PEx) Events for 108/110 PEx Analysis Set [ Time Frame: From Baseline up to 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Body Mass Index (BMI) for 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Absolute Change in Body Mass Index (BMI) for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Body Mass Index (BMI) for 103/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Body Mass Index (BMI) for Study 111/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in BMI Z-score for 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    The 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Absolute Change in BMI Z-score for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    The 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/110 Efficacy Set [ Time Frame: From Baseline at Study 110 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. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Body Weight for Study 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Absolute Change in Body Weight for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Body Weight for 103/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Body Weight for 111/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Body Weight Z-score for 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    The 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Absolute Change in Body Weight Z-score for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    The 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Absolute Change in Height Z-score for 106/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    The 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline.
  • Part A: Absolute Change in Height Z-score for 108/110 Efficacy Set [ Time Frame: From Baseline at Study 110 Week 96 ]
    The 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline.
  • Part A: Time-to-first Pulmonary Exacerbation (PEx) for 106/110 PEx Analysis Set [ Time Frame: 96 weeks ]
    Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan.
  • Part A: Time-to-first Pulmonary Exacerbation (PEx) for 108/110 PEx Analysis Set [ Time Frame: 96 weeks ]
    Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan.
  • Part A: Plasma Concentrations of TEZ, TEZ Metabolite (M1-TEZ), Ivacaftor (IVA) and Ivacaftor Metabolite (M1-IVA) [ Time Frame: Week 24 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: September 29, 2015)
  • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline through Week 96 ]
  • Relative change from baseline in ppFEV1 [ Time Frame: from baseline through Week 96 ]
  • Number of pulmonary exacerbations [ Time Frame: from baseline through Week 96 ]
  • Absolute change from baseline in body mass index (BMI) [ Time Frame: from baseline through Week 96 ]
  • Absolute change from baseline in BMI z-score for subjects aged <20 years [ Time Frame: from baseline through Week 96 ]
  • Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through Week 96 ]
  • Absolute change from baseline in body weight [ Time Frame: from baseline through Week 96 ]
  • Absolute change from baseline in body weight z-score for subjects aged <20 years [ Time Frame: from baseline through Week 96 ]
  • Absolute change from baseline in height z-score for subjects aged <20 years [ Time Frame: from baseline through Week 96 ]
  • Time-to-first pulmonary exacerbation [ Time Frame: from baseline through Week 96 ]
  • Pharmacokinetic (PK) parameters: trough concentrations of VX-661 , a VX-661 metabolite (M1-661), ivacaftor, ivacaftor metabolite (M1-ivacaftor) [ Time Frame: from baseline through Week 96 ]
  • Observational Cohort: Safety, as determined by related serious adverse events (SAEs) [ Time Frame: from baseline through study Completion (up to 3 years) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis Who Have an F508del-CFTR Mutation
Official Title  ICMJE A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Brief Summary This is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744), VX14-661-106 (Study 106, NCT02347657), VX14-661-107 (Study 107, NCT02516410), VX14-661-108 (Study 108, NCT02392234), VX14-661-109 (Study 109, NCT02412111), and VX14-661-111 (Study 111, NCT02508207). The study is designed to evaluate the safety and efficacy of long-term treatment of VX-661 in combination with ivacaftor.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: TEZ/IVA
    Other Names:
    • VX-661/VX-770
    • tezacaftor/ivacaftor
  • Drug: IVA
    Other Names:
    • VX-770
    • ivacaftor
Study Arms  ICMJE
  • Experimental: Part A: TEZ/IVA
    TEZ 100 mg/IVA150 mg fixed dose tablet once daily in the morning and IVA150 mg mono tablet once daily in the evening.
    Interventions:
    • Drug: TEZ/IVA
    • Drug: IVA
  • Experimental: Part B: TEZ/IVA
    TEZ 100 mg/IVA150 mg fixed dose tablet once daily in the morning and IVA150 mg mono tablet once daily in the evening.
    Interventions:
    • Drug: TEZ/IVA
    • Drug: IVA
  • Experimental: Part C: TEZ/IVA
    TEZ 100 mg/IVA150 mg fixed dose tablet once daily in the morning and IVA150 mg mono tablet once daily in the evening.
    Interventions:
    • Drug: TEZ/IVA
    • Drug: IVA
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: March 4, 2020)
1044
Original Estimated Enrollment  ICMJE
 (submitted: September 29, 2015)
1375
Estimated Study Completion Date  ICMJE March 2023
Actual Primary Completion Date May 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Part A:

  • Subjects entering the Treatment Cohort must meet all of the following criteria:
  • Elect to enroll in the Treatment Cohort
  • Completed study drug Treatment Period in a parent study (NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111) or study drug treatment and the Safety Follow up Visit for subjects from NCT02508207.
  • Willing to remain on a stable CF regimen through the Safety Follow-up Visit.
  • Subjects re-enrolling in the Part A Treatment Cohort must meet all of the following criteria:

    • Previously received at least 4 weeks of study drug before discontinuing in Part A of Study NCT02565914 to participate in another qualified Vertex study.
    • Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part A Study NCT02565914.
  • Subjects entering the Part A Observational Cohort must meet the following criteria:

    • <18 years of age (age on the date of informed consent/assent in the parent study)
    • Completed study drug Treatment Period in a parent study or study drug treatment and the Safety Follow up Visit for subjects from NCT02508207, but do not elect to enroll in the NCT02565914 Treatment Cohort; or
    • Received at least 4 weeks of study drug treatment and completed visits up to the last scheduled visit of the Treatment Period of a parent study (and the Safety Follow up Visit for subjects from NCT02508207), but do not meet eligibility criteria for enrollment into the Treatment Cohort

Part B:

Subjects who meet all of the following inclusion criteria will be eligible for Part B.

  • Did not withdraw consent from the parent study or Part A of Study NCT02565914.
  • Completed study drug treatment during the Treatment Period in Part A of - Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit of Study NCT02565914.

Subjects re enrolling in Part B must meet all of the following criteria:

  • Previously received at least 4 weeks of study drug before discontinuing Study NCT02565914 to participate in another qualified Vertex study, which is defined as a Vertex study of investigational CFTR modulators that allows participation of subjects in Study NCT02565914.
  • Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part B.
  • Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit in Part B.

Part C:

  • Subjects who meet all of the following inclusion criteria will be eligible for Part C.
  • Did not withdraw consent from Part B of Study NCT02565914.
  • Completed study drug treatment during Part B of NCT02565914.
  • Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit of Part C.

Exclusion Criteria:

  • History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk to the subject.
  • 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 parent study that would pose an additional risk to the subject.
  • Participation in an investigational drug trial (including studies investigating VX-661/ivacaftor or lumacaftor/ivacaftor) other than the parent studies of NCT02565914 or other eligible Vertex studies investigating VX-661 in combination with ivacaftor, or use of a commercially available CFTR modulator.

Other protocol defined Inclusion/Exclusion criteria may apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   Belgium,   Canada,   Denmark,   France,   Germany,   Ireland,   Israel,   Italy,   Netherlands,   Spain,   Sweden,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02565914
Other Study ID Numbers  ICMJE VX14-661-110
2014-004827-29 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date May 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP