AzTrials
AzTrials

Cystic FibrosisPhase 3NCT02565914

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

A Phase 3 study testing TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 154 sites in 16 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Cystic Fibrosis
What is being tested?
TEZ/IVA and IVA
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
1,131 participants
Where
154 sitesin 16 countries
Study dates
Aug 2015to 5 Dec 2022
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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:
Show all 25Show fewer
  • 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.

Who cannot join

  • 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.

Trial locations and contacts

154 sites in 16 countries.

154 sites in 16 countries

United States69 sites

  • Study site

    Akron, Ohio

  • Study site

    Albany, New York

  • Study site

    Albuquerque, New Mexico

  • Study site

    Ann Arbor, Michigan

  • Study site

    Atlanta, Georgia

  • Study site

    Aurora, Colorado

  • Study site

    Austin, Texas

  • Study site

    Baltimore, Maryland

  • Study site

    Birmingham, Alabama

  • Study site

    Boise, Idaho

  • Study site

    Boston, Massachusetts

  • Study site

    Buffalo, New York

  • Study site

    Chapel Hill, North Carolina

  • Study site

    Charleston, South Carolina

  • Study site

    Chicago, Illinois

  • Study site

    Cincinnati, Ohio

  • Study site

    Cleveland, Ohio

  • Study site

    Columbus, Ohio

  • Study site

    Dallas, Texas

  • Study site

    Denver, Colorado

  • Study site

    Detroit, Michigan

  • Study site

    Fort Worth, Texas

  • Study site

    Gainesville, Florida

  • Study site

    Glenview, Illinois

  • Study site

    Houston, Texas

  • Study site

    Indianapolis, Indiana

  • Study site

    Iowa City, Iowa

  • Study site

    Kansas City, Missouri

  • Study site

    Knoxville, Tennessee

  • Study site

    Lebanon, New Hampshire

  • Study site

    Lexington, Kentucky

  • Study site

    Little Rock, Arkansas

  • Study site

    Long Beach, California

  • Study site

    Long Branch, New Jersey

  • Study site

    Los Angeles, California

  • Study site

    Memphis, Tennessee

  • Study site

    Miami, Florida

  • Study site

    Milwaukee, Wisconsin

  • Study site

    Minneapolis, Minnesota

  • Study site

    Morgantown, West Virginia

  • Study site

    Nashville, Tennessee

  • Study site

    New Brunswick, New Jersey

  • Study site

    New Hyde Park, New York

  • Study site

    New Orleans, Louisiana

  • Study site

    New York, New York

  • Study site

    Niles, Illinois

  • Study site

    Oakland, California

  • Study site

    Oklahoma City, Oklahoma

  • Study site

    Omaha, Nebraska

  • Study site

    Orlando, Florida

  • Study site

    Palo Alto, California

  • Study site

    Pensacola, Florida

  • Study site

    Peoria, Illinois

  • Study site

    Philadelphia, Pennsylvania

  • Study site

    Pittsburgh, Pennsylvania

  • Study site

    Portland, Oregon

  • Study site

    Rochester, New York

  • Study site

    Sacramento, California

  • Study site

    Salt Lake City, Utah

  • Study site

    San Diego, California

  • Study site

    Seattle, Washington

  • Study site

    Sioux Falls, South Dakota

  • Study site

    Spokane, Washington

  • Study site

    St Louis, Missouri

  • Study site

    Syracuse, New York

  • Study site

    Tampa, Florida

  • Study site

    Toledo, Ohio

  • Study site

    Tucson, Arizona

  • Study site

    Tyler, Texas

Other trials in United States

United Kingdom14 sites

  • Study site

    Belfast

  • Study site

    Birmingham

  • Study site

    Exeter

  • Study site

    Glasgow

  • Study site

    Leeds, West Yorkshire

  • Study site

    Leeds

  • Study site

    Liverpool

  • Study site

    London

  • Study site

    Manchester

  • Study site

    Newcastle upon Tyne

  • Study site

    Penarth

  • Study site

    Sheffield

  • Study site

    Southampton

  • Study site

    Stoke-on-Trent

Other trials in United Kingdom

Germany12 sites

  • Study site

    Berlin

  • Study site

    Bochum

  • Study site

    Erlangen

  • Study site

    Essen

  • Study site

    Frankfurt

  • Study site

    Giesen

  • Study site

    Hannover

  • Study site

    Heidelberg

  • Study site

    Jena

  • Study site

    München

  • Study site

    Tübingen

  • Study site

    Würzburg

Other trials in Germany

France10 sites

  • Study site

    Benite Cedex

  • Study site

    Bordeaux

  • Study site

    Bron

  • Study site

    Lille

  • Study site

    Marseille

  • Study site

    Montpellier

  • Study site

    Paris

  • Study site

    Rennes

  • Study site

    Roscoff

  • Study site

    Rouen

Other trials in France

Italy10 sites

  • Study site

    Ancona

  • Study site

    Genova

  • Study site

    Milan

  • Study site

    Orbassano

  • Study site

    Palermo

  • Study site

    Potenza

  • Study site

    Roma

  • Study site

    Rome

  • Study site

    Torino

  • Study site

    Verona

Other trials in Italy

Canada5 sites

  • Study site

    Halifax

  • Study site

    Montreal

  • Study site

    Québec

  • Study site

    Toronto, Ontario

  • Study site

    Vancouver, British Columbia

Other trials in Canada

Israel5 sites

  • Study site

    Haifa, NAP

  • Study site

    Haifa

  • Study site

    Jerusalem

  • Study site

    Petah Tikva

  • Study site

    Tel HaShomer

Other trials in Israel

Netherlands5 sites

  • Study site

    Amsterdam

  • Study site

    Heidelberglaan

  • Study site

    Nijmegen

  • Study site

    Rotterdam

  • Study site

    The Hague

Other trials in Netherlands

Spain5 sites

  • Study site

    Barcelona

  • Study site

    Madrid

  • Study site

    Sabadell

  • Study site

    Seville

  • Study site

    Valencia

Other trials in Spain

Australia4 sites

  • Study site

    Chermside

  • Study site

    Melbourne

  • Study site

    South Brisbane

  • Study site

    Westmead

Other trials in Australia

Austria3 sites

  • Study site

    Graz

  • Study site

    Innsbruck

  • Study site

    Salzburg

Other trials in Austria

Belgium3 sites

  • Study site

    Brussels

  • Study site

    Ghent

  • Study site

    Leuven

Other trials in Belgium

Ireland3 sites

  • Study site

    Cork

  • Study site

    Dublin

  • Study site

    Limerick

Other trials in Ireland

Sweden3 sites

  • Study site

    Gothenburg

  • Study site

    Stockholm

  • Study site

    Uppsala

Other trials in Sweden

Switzerland2 sites

  • Study site

    Bern

  • Study site

    Zurich

Other trials in Switzerland

Denmark1 site

  • Study site

    Copenhagen

Other trials in Denmark

About this study

Official title: 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.

Conditions
Cystic Fibrosis
Being tested
TEZ/IVA, IVA
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TEZ/IVADrug

    Also called VX-661/VX-770, tezacaftor/ivacaftor

    Fixed dose tablet for oral administration.

  • IVADrug

    Also called VX-770, ivacaftor

    Tablet for oral administration.

Study groups in NCT02565914
Study groupRoleWhat they receive
TEZ/IVAExperimentalPart 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.
Receives: Drug: TEZ/IVA; Drug: IVA

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Day 1 up to Week 100

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

    Measured: Day 1 up to Week 100

  3. Part C: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Measured: Day 1 up to Week 196

Secondary outcomes (34)

  1. Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  2. Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  3. Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  4. Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  5. Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  6. Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  7. Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  8. Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  9. Part A: Number of Pulmonary Exacerbation (PEx) Events for 106/110 PEx Analysis Set

    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.

    Measured: From Baseline up to Study 110 Week 96

  10. Part A: Number of Pulmonary Exacerbation (PEx) Events for 108/110 PEx Analysis Set

    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.

    Measured: From Baseline up to Week 96

  11. Part A: Absolute Change in Body Mass Index (BMI) for 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  12. Part A: Absolute Change in Body Mass Index (BMI) for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  13. Part A: Absolute Change in Body Mass Index (BMI) for 103/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  14. Part A: Absolute Change in Body Mass Index (BMI) for Study 111/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  15. Part A: Absolute Change in BMI Z-score for 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  16. Part A: Absolute Change in BMI Z-score for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  17. Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  18. Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  19. Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  20. Part A: Absolute Change in Body Weight for Study 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  21. Part A: Absolute Change in Body Weight for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  22. Part A: Absolute Change in Body Weight for 103/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  23. Part A: Absolute Change in Body Weight for 111/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  24. Part A: Absolute Change in Body Weight Z-score for 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  25. Part A: Absolute Change in Body Weight Z-score for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  26. Part A: Absolute Change in Height Z-score for 106/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  27. Part A: Absolute Change in Height Z-score for 108/110 Efficacy Set

    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.

    Measured: From Baseline at Study 110 Week 96

  28. Part A: Time-to-first Pulmonary Exacerbation (PEx) for 106/110 PEx Analysis Set

    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.

    Measured: 96 weeks

  29. Part A: Time-to-first Pulmonary Exacerbation (PEx) for 108/110 PEx Analysis Set

    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.

    Measured: 96 weeks

  30. Part A: Plasma Concentrations of TEZ, TEZ Metabolite (M1-TEZ), Ivacaftor (IVA) and Ivacaftor Metabolite (M1-IVA)

    Measured: Week 24

  31. Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

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

    Measured: From Baseline at Study 110 Week 96

  32. Part B: Absolute Change in Body Mass Index (BMI)

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

    Measured: From Baseline at Week 96

  33. Part B: Absolute Change in BMI Z-score

    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.

    Measured: From Baseline at Week 96

  34. Part B: Number of Pulmonary Exacerbation (PEx) Events

    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.

    Measured: From Baseline up to Week 96

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Oct 2015 (estimated)
  2. Study startAug 2015
  3. Primary completionMay 2019
  4. Study completion5 Dec 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
1,131 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02565914
Sponsor's protocol ID
VX14-661-110
Other IDs
2014-004827-29, 2014
Status last verified
Sep 2023
First submitted
18 Sep 2015
Last updated
28 Sep 2023
Data monitoring committee
Yes

Publications

  • Flume PA, Biner RF, Downey DG, Brown C, Jain M, Fischer R, De Boeck K, Sawicki GS, Chang P, Paz-Diaz H, Rubin JL, Yang Y, Hu X, Pasta DJ, Millar SJ, Campbell D, Wang X, Ahluwalia N, Owen CA, Wainwright CE; VX14-661-110 study group. Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study. Lancet Respir Med. 2021 Jul;9(7):733-746. doi: 10.1016/S2213-2600(20)30510-5. Epub 2021 Feb 10.

    PubMed 33581080

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 154 sites in 16 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 154 sites in United States, United Kingdom, Germany, France, Italy and 11 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Jun 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02565914, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2023.

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