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

September 1, 2023 updated by: Vertex Pharmaceuticals Incorporated

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

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), VX14-661-111 (Study 111, NCT02508207), VX15-661-112 (NCT02730208), and VX16-661-114 (NCT03150719). The study is designed to evaluate the safety and efficacy of long-term treatment of VX-661 in combination with ivacaftor.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

1131

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Chermside, Australia
      • Melbourne, Australia
      • South Brisbane, Australia
      • Westmead, Australia
  • Austria
      • Graz, Austria
      • Innsbruck, Austria
      • Salzburg, Austria
  • Belgium
      • Brussels, Belgium
      • Gent, Belgium
      • Leuven, Belgium
  • Canada
      • Halifax, Canada
      • Montreal, Canada
      • Montréal, Canada
      • Québec, Canada
    • British Columbia
      • Vancouver, British Columbia, Canada
    • Ontario
      • Toronto, Ontario, Canada
  • Denmark
      • Copenhagen, Denmark
  • France
      • Benite Cedex, France
      • Bordeaux Cedex, France
      • Bron Cedex, France
      • Lille, France
      • Marseilles, France
      • Montpellier Cedex 5, France
      • Paris, France
      • Paris Cedex 14, France
      • Paris Cedex 15, France
      • Rennes Cedex, France
      • Roscoff Cedex, France
      • Rouen Cedex, France
  • Germany
      • Berlin, Germany
      • Bochum, Germany
      • Erlangen, Germany
      • Essen, Germany
      • Frankfurt, Germany
      • Giesen, Germany
      • Hannöver, Germany
      • Heidelberg, Germany
      • Jena, Germany
      • Muenchen, Germany
      • Munchen, Germany
      • Tuebingen, Germany
      • Würzburg, Germany
  • Ireland
      • Cork, Ireland
      • Dublin, Ireland
      • Dublin 4, Ireland
      • Limerick, Ireland
  • Israel
      • Haifa, Israel
      • Jerusalem, Israel
      • Petah Tikva, Israel
      • Tel Hashomer, Israel
    • NAP
      • Haifa, NAP, Israel
  • Italy
      • Ancona, Italy
      • Genova, Italy
      • Milano, Italy
      • Orbassano, Italy
      • Palermo, Italy
      • Potenza, Italy
      • Roma, Italy
      • Rome, Italy
      • Torino, Italy
      • Verona, Italy
  • Netherlands
      • Amsterdam, Netherlands
      • Den Haag, Netherlands
      • Heidelberglaan, Netherlands
      • Nijmegen, Netherlands
      • Rotterdam, Netherlands
  • Spain
      • Barcelona, Spain
      • Madrid, Spain
      • Sabadell, Spain
      • Sevilla, Spain
      • Valencia, Spain
  • Sweden
      • Goteborg, Sweden
      • Stockholm, Sweden
      • Uppsala, Sweden
  • Switzerland
      • Bern, Switzerland
      • Zurich, Switzerland
  • United Kingdom
      • Belfast, United Kingdom
      • Birmingham, United Kingdom
      • Exeter, United Kingdom
      • Glasgow, United Kingdom
      • Leeds, United Kingdom
      • Liverpool, United Kingdom
      • London, United Kingdom
      • Manchester, United Kingdom
      • Newcastle Upon Tyne, United Kingdom
      • Penarth, United Kingdom
      • Sheffield, United Kingdom
      • Southampton, United Kingdom
      • Stoke-on-Trent, United Kingdom
    • West Yorkshire
      • Leeds, West Yorkshire, United Kingdom
  • United States
    • Alabama
      • Birmingham, Alabama, United States
    • Arizona
      • Tucson, Arizona, United States
    • Arkansas
      • Little Rock, Arkansas, United States
    • California
      • Long Beach, California, United States
      • Los Angeles, California, United States
      • Oakland, California, United States
      • Palo Alto, California, United States
      • Sacramento, California, United States
      • San Diego, California, United States
    • Colorado
      • Aurora, Colorado, United States
      • Denver, Colorado, United States
    • Florida
      • Gainesville, Florida, United States
      • Miami, Florida, United States
      • Orlando, Florida, United States
      • Pensacola, Florida, United States
      • Tampa, Florida, United States
    • Georgia
      • Atlanta, Georgia, United States
    • Idaho
      • Boise, Idaho, United States
    • Illinois
      • Chicago, Illinois, United States
      • Glenview, Illinois, United States
      • Niles, Illinois, United States
      • Peoria, Illinois, United States
    • Indiana
      • Indianapolis, Indiana, United States
    • Iowa
      • Iowa City, Iowa, United States
    • Kentucky
      • Lexington, Kentucky, United States
    • Louisiana
      • New Orleans, Louisiana, United States
    • Maryland
      • Baltimore, Maryland, United States
    • Massachusetts
      • Boston, Massachusetts, United States
    • Michigan
      • Ann Arbor, Michigan, United States
      • Detroit, Michigan, United States
    • Minnesota
      • Minneapolis, Minnesota, United States
    • Missouri
      • Kansas City, Missouri, United States
      • Saint Louis, Missouri, United States
    • Nebraska
      • Omaha, Nebraska, United States
    • New Hampshire
      • Lebanon, New Hampshire, United States
    • New Jersey
      • Long Branch, New Jersey, United States
      • New Brunswick, New Jersey, United States
    • New Mexico
      • Albuquerque, New Mexico, United States
    • New York
      • Albany, New York, United States
      • Buffalo, New York, United States
      • New Hyde Park, New York, United States
      • New York, New York, United States
      • Rochester, New York, United States
      • Syracuse, New York, United States
    • North Carolina
      • Chapel Hill, North Carolina, United States
    • Ohio
      • Akron, Ohio, United States
      • Cincinnati, Ohio, United States
      • Cleveland, Ohio, United States
      • Columbus, Ohio, United States
      • Toledo, Ohio, United States
    • Oklahoma
      • Oklahoma City, Oklahoma, United States
    • Oregon
      • Portland, Oregon, United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States
      • Pittsburgh, Pennsylvania, United States
    • South Carolina
      • Charleston, South Carolina, United States
    • South Dakota
      • Sioux Falls, South Dakota, United States
    • Tennessee
      • Knoxville, Tennessee, United States
      • Memphis, Tennessee, United States
      • Nashville, Tennessee, United States
    • Texas
      • Austin, Texas, United States
      • Dallas, Texas, United States
      • Fort Worth, Texas, United States
      • Houston, Texas, United States
      • Tyler, Texas, United States
    • Utah
      • Salt Lake City, Utah, United States
    • Washington
      • Seattle, Washington, United States
      • Spokane, Washington, United States
    • West Virginia
      • Morgantown, West Virginia, United States
    • Wisconsin
      • Milwaukee, Wisconsin, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

Part A:

  • Participants 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 participants from NCT02508207.
  • Willing to remain on a stable CF regimen through the Safety Follow-up Visit.

  • Participants 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.

  • Participants 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 participants from NCT02508207), but do not meet eligibility criteria for enrollment into the Treatment Cohort

Part B:

Participants 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.

Participants 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 participants 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:

  • Participants 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TEZ/IVA

Part A: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 103,106,107,108,109 and 111 were administered TEZ 100 milligram (mg)/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks.

Part B: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 106,108,109,112 and 114 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks.

Part C: Participants who received TEZ/IVA, IVA monotherapy or Placebo in parent studies 106,108, and 114 were administered TEZ 100 mg/IVA 150 mg fixed dose tablet in the morning and IVA 150 mg mono tablet in the evening for 192 weeks.
Drug: IVA
Tablet for oral administration.
Other Names:
  • VX-770
  • ivacaftor
Drug: TEZ/IVA
Fixed dose tablet for oral administration.
Other Names:
  • VX-661/VX-770
  • tezacaftor/ivacaftor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Day 1 up to Week 100
Part B: 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
Day 1 up to Week 100
Part C: 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 196
Day 1 up to Week 196

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline up to Study 110 Week 96
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.
From Baseline up to Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
From Baseline at Study 110 Week 96
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.
96 weeks
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.
96 weeks
Part A: Plasma Concentrations of TEZ, TEZ Metabolite (M1-TEZ), Ivacaftor (IVA) and Ivacaftor Metabolite (M1-IVA)
Time Frame: Week 24
Week 24
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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.
From Baseline at Study 110 Week 96
Part B: Absolute Change in Body Mass Index (BMI)
Time Frame: From Baseline at Week 96
BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).
From Baseline at Week 96
Part B: Absolute Change in BMI Z-score
Time Frame: From Baseline at 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.
From Baseline at Week 96
Part B: Number of Pulmonary Exacerbation (PEx) Events
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.
From Baseline up to Week 96

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

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General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2015

Primary Completion (Actual)

May 1, 2019

Study Completion (Actual)

December 5, 2022

Study Registration Dates

First Submitted

September 18, 2015

First Submitted That Met QC Criteria

September 29, 2015

First Posted (Estimated)

October 1, 2015

Study Record Updates

Last Update Posted (Actual)

September 28, 2023

Last Update Submitted That Met QC Criteria

September 1, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX14-661-110
  • 2014-004827-29 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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