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AzTrials

Cystic FibrosisPhase 3NCT03447262

A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

A Phase 3 study testing VX-659/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 100 sites in 11 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation. 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?
VX-659/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
484 participants
Where
100 sitesin 11 countries
Study dates
13 Jul 2018to 9 Sep 2020
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

  • Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.

Who cannot join

  • History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
  • Current participation in an investigational drug trial (other than a parent study)
  • Other protocol defined Inclusion/Exclusion criteria may apply.

Trial locations and contacts

100 sites in 11 countries.

100 sites in 11 countries

United States48 sites

  • Albany Medical College

    Albany, New York, 12208

  • Michigan Medicine

    Ann Arbor, Michigan, 48109-5212

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21287

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233

  • St. Luke's CF Center of Idaho

    Boise, Idaho, 83712

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • CF Therapeutics Development Center of Western New York

    Buffalo, New York, 14203

  • Clinical Research of Charlotte

    Charlotte, North Carolina, 28277

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104

  • Cystic Fibrosis Center of Chicago

    Glenview, Illinois, 60025

  • Spectrum Health Medical Group Adult Cystic Fibrosis Care Center

    Grand Rapids, Michigan, 49546

  • Hartford Hospital

    Hartford, Connecticut, 06102

  • Texas Children's Hospital

    Houston, Texas, 77030

  • Indiana Clinical Research Center, IU Health University Hospital

    Indianapolis, Indiana, 46202

  • The University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216

  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • University of Tennessee Medical Center- Adult Cystic Fibrosis Clinic

    Knoxville, Tennessee, 37920

  • University of Kentucky

    Lexington, Kentucky, 40536

  • Kosair Charities Pediatric Clinical Research Unit

    Louisville, Kentucky, 40202

  • Dartmouth Hitchcock, Manchester

    Manchester, New Hampshire, 03104

  • Children's Foundation Research Center / Le Bonheur Children's Hospital

    Memphis, Tennessee, 38103

  • University of Miami Miller School of Medicine

    Miami, Florida, 33136

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Rutgers-Robert Wood Johnson Medical School

    New Brunswick, New Jersey, 08901

  • Yale New Haven Medical Center

    New Haven, Connecticut, 06511

  • Northwell Health, Long Island Jewish Medical Center

    New Hyde Park, New York, 11040

  • Columbia University Medical Center

    New York, New York, 10032

  • Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants

    Niles, Illinois, 60714

  • Santiago Reyes, M.D.

    Oklahoma City, Oklahoma, 73112

  • Orlando Health, Inc.- Arnold Palmer Hospital for Children (APH)

    Orlando, Florida, 32806

  • Stanford University

    Palo Alto, California, 94304

  • Drexel University College of Medicine / Drexel Adult Cystic Fibrosis Center

    Philadelphia, Pennsylvania, 19107

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • University of Utah/ Primary Children's Medical Center

    Salt Lake City, Utah, 84132

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Sanford Children's Specialty Clinic

    Sioux Falls, South Dakota, 57105

  • Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

    Spokane, Washington, 99204

  • Washington University School of Medicine/ St. Louis Children's Hospital

    St Louis, Missouri, 63110

  • Johns Hopkins All Children's Hospital Outpatient Care Center

    St. Petersburg, Florida, 33701

  • SUNY Upstate Medical University

    Syracuse, New York, 13210

  • UMass Memorial Medical Center

    Worcester, Massachusetts, 01655

Other trials in United States

Germany9 sites

  • Charite Paediatric Pulmonology Department

    Berlin

  • Universitaetsklinkum Koeln, CF-Studienzentrum

    Cologne

  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

    Essen

  • Clinic of J.W. Goethe University

    Frankfurt

  • Medizinische Hochschule Hannover

    Hanover

  • Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin

    Jena

  • Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin

    Lübeck

  • Klinikum Innenstadt, University of Munich

    München

  • Pneumologische Praxis Pasing

    München

Other trials in Germany

United Kingdom9 sites

  • Papworth Hospital NHS Foundation Trust, Papworth Everard

    Cambridge

  • Clinical Research Facility, Queen Elizabeth University Hospital

    Glasgow

  • St. James University Hospital

    Leeds

  • Liverpool Head and Chest Hospital

    Liverpool

  • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

    London

  • Wythenshaw e Hospital

    Manchester

  • The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary

    Newcastle upon Tyne

  • Wolfson Cystic Fibrosis Unit, City Campus

    Nottingham

  • All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

    Penarth

Other trials in United Kingdom

Australia8 sites

  • Royal Adelaide Hospital

    Adelaide

  • The Prince Charles Hospital

    Chermside

  • Alfred Hospital

    Melbourne

  • Institute for Respiratory Health, Sir Charles Gairdner Hospital

    Nedlands

  • John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital

    New Lambton

  • Telethon Kids Institute

    Perth

  • Sydney Children's Hospital

    Randwick

  • Lady Cilento Children's Hospital

    South Brisbane

Other trials in Australia

Ireland7 sites

  • Cork University Hospital

    Cork

  • Beaumont Hospital

    Dublin

  • Our Lady's Children's Hospital

    Dublin

  • St. Vincent's University Hospital

    Dublin

  • Temple Street Children's University Hospital

    Dublin

  • University Hospital Galway

    Galway

  • University Hospital Limerick

    Limerick

Other trials in Ireland

Spain7 sites

  • Hospital Universitari Vall d Hebron

    Barcelona

  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

    Barcelona

  • Hospital Universitario 12 de Octubre

    Madrid

  • Hospital Universitario Infantil La Paz

    Madrid

  • Parc Tauli Sabadell Hospital Universitari

    Sabadell

  • Hospital Universitario Virgen del Rocio

    Seville

  • Hospital Universitario y Politecnico La Fe

    Valencia

Other trials in Spain

Israel5 sites

  • Lady Davis Carmel Medical Center

    Haifa

  • Rambam Health Care Campus, Liver Unit

    Haifa

  • Pediatrics Hadassah Medical Center

    Jerusalem

  • Schneider Children's Medical Center

    Petah Tikva

  • Sheba Medical Center

    Tel HaShomer

Other trials in Israel

Canada3 sites

  • Stollery Children's Hospital

    Edmonton

  • Queen Elizabeth II Health Sciences Center

    Halifax

  • St. Michael's Hospital

    Toronto

Other trials in Canada

Switzerland2 sites

  • Lindenhofspital - Quartier Bleu

    Bern

  • Kinderspital Zuerich

    Zurich

Other trials in Switzerland

Denmark1 site

  • Juliane Marie Center, Rigshospitalet

    Copenhagen

Other trials in Denmark

Poland1 site

  • Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY

    Łomianki

Other trials in Poland

About this study

Official title: A Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation

This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.

Conditions
Cystic Fibrosis
Being tested
VX-659/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.

  • VX-659/TEZ/IVADrug

    Also called VX-659/VX-661/VX-770, VX-659/tezacaftor/ivacaftor

    Fixed-dose combination tablets for oral administration qd in the morning.

  • IVADrug

    Also called VX-770, ivacaftor

    IVA tablet qd in the evening.

Study groups in NCT03447262
Study groupRoleWhat they receive
VX-659/TEZ/IVA TCExperimentalParticipants from parent studies VX17-659-102 (NCT03447249) or VX17-659-103 (NCT03460990) were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study VX17-659-105.
Receives: Drug: VX-659/TEZ/IVA; Drug: IVA

What is being measured

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

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

    Measured: From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100)

Secondary outcomes (16)

  1. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

  2. Absolute Change in ppFEV1 for Participants From the Parent Study 659-103

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

  3. Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102

    Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 24 (Study 659-105)

  4. Absolute Change in SwCl for Participants From the Parent Study 659-103

    Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 24 (Study 659-105)

  5. Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102

    PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

    Measured: From Baseline up to Week 96 (Study 659-105)

  6. Number of PEx for Participants From the Parent Study 659-103

    PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported for overall participants from the parent study 659-103, that is combined for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

    Measured: From Baseline up to Week 96 (Study 659-105)

  7. Number of Participants With at Least One PEx for Participants From the Parent Study 659-102

    PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the Kaplan-Meier (KM) method. However, because way less than 50% of participants had events, median time-to-first event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported separately for those in Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105). Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

    Measured: From Baseline up to Week 96 (Study 659-105)

  8. Number of Participants With at Least One PEx for Participants From the Parent Study 659-103

    PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the KM method. However, because way less than 50% of participants had events, median time-to-first-event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported for all participants from the parent study 659-103, that is combined for those in the TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105). Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

    Measured: From Baseline up to Week 96 (Study 659-105)

  9. Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102

    BMI was defined as weight in kilograms (kg) divided by squared height in meters (m^2). The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

  10. Absolute Change in BMI for Participants From the Parent Study 659-103

    BMI was defined as weight in kg divided by squared height in meters (m^2). The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

  11. Absolute Change in BMI Z-score for Participants From the Parent Study 659-102 (Participants <=20 Years Old at Parent Study Baseline)

    BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 60 (Study 659-105)

  12. Absolute Change in BMI Z-score for Participants From The Parent Study 659-103 (Participants <=20 Years Old at Parent Study Baseline)

    BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 60 (Study 659-105)

  13. Absolute Change in Body Weight for Participants From the Parent Study 659-102

    The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

  14. Absolute Change in Body Weight for Participants From the Parent Study 659-103

    The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

  15. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for Participants From the Parent Study 659-102

    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. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

  16. Absolute Change in CFQ-R Respiratory Domain Score for Participants From the Parent Study 659-103

    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. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

    Measured: From Baseline at Week 72 (Study 659-105)

Timeline

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

  1. First posted27 Feb 2018
  2. Study start13 Jul 2018
  3. Primary completion9 Sep 2020
  4. Study completion9 Sep 2020

Why it stopped: At Sponsor's Discretion

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
484 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03447262
Sponsor's protocol ID
VX17-659-105
Other IDs
2017-004134-29, 2017
Status last verified
Dec 2021
First submitted
21 Feb 2018
Last updated
25 Jan 2022
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.

    PubMed 37983082

  • Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

    PubMed 33331662

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing VX-659/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 100 sites in 11 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 1 inclusion and 3 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: stopped early, as of the sponsor's last update on 25 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 100 sites in United States, Germany, United Kingdom, Australia, Ireland and 6 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 25 Jan 2022). 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?

Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100)). 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 NCT03447262, which also lists 2 linked publications.

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 25 Jan 2022.

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