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Cystic FibrosisPhase 3NCT03447249

A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

A Phase 3 study testing VX-659/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 101 sites in 11 countries.

Completed

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

Official record
Search all trials

Trial summary

This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects). 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?
Yes, some participants may receive a placebo
How many people
385 participants
Where
101 sitesin 11 countries
Study dates
7 Mar 2018to 5 Feb 2019
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

  • Heterozygous for F508del and an MF mutation (as defined in the protocol)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Who cannot join

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Solid organ or hematological transplantation
  • Other protocol defined Inclusion/Exclusion criteria may apply

Trial locations and contacts

101 sites in 11 countries.

101 sites in 11 countries

United States48 sites

  • Albany Medical College

    Albany, New York, 12208

  • University of Michigan Health System

    Ann Arbor, Michigan, 48109

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • The 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

  • Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo

    Buffalo, New York, 14222

  • Clinical Research of Charlotte

    Charlotte, North Carolina, 28277

  • Cincinnati Children's Hospital Medical Center

    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

  • Helen DeVos Children's Hospital CF Center

    Grand Rapids, Michigan, 49503

  • Hartford Health

    Hartford, Connecticut, 06106

  • Baylor College of Medicine

    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

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • University of Tennessee Medical Center - Adult Cystic Fibrosis Clinic

    Knoxville, Tennessee, 37920

  • Dartmouth Hitchcock Medical Center, Lebanon

    Lebanon, New Hampshire, 03756

  • Kentucky Clinic

    Lexington, Kentucky, 40536

  • Kosair Charities Pediatric Clinical Research Unit

    Louisville, Kentucky, 40202

  • 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, 37920

  • Rutgers-Robert Wood Johnson Medical School

    New Brunswick, New Jersey, 80901

  • 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

  • Respiratory Diseases of Children and Adolescents

    Oklahoma City, Oklahoma, 73112

  • Arnold Palmer Hospital

    Orlando, Florida, 32806

  • Stanford University

    Palo Alto, California, 94304

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

    Philadelphia, Pennsylvania, 19017

  • Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

    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 Research/ USD

    Sioux Falls, South Dakota, 57105

  • Providence Pediatric Pulmonary & Allergy/Immunology 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

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts, 01655

Other trials in United States

Germany9 sites

  • Charite Paediatric Pulmonology Department

    Berlin

  • University Hospital Cologne

    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 fur 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

  • Queen Elizabeth University Hospital

    Glasgow

  • The Leeds Teaching Hospitals NHS Trust, St. James University Hospital

    Leeds

  • Liverpool Heart and Chest Hospital

    Liverpool

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

    London

  • Wythenshawe Hospital

    Manchester

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

    Newcastle upon Tyne

  • Nottingham University Hospitals NHS Trust, Queens Medical Center

    Nottingham

  • University Hospital Llandough

    Penarth

Other trials in United Kingdom

Australia8 sites

  • Royal Adelaide Hospital

    Adelaide

  • Prince Charles Hospital

    Chermside

  • Royal Brisbane & Women's Hospital

    Herston

  • The Alfred Hospital

    Melbourne, Victoria

  • Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital

    Nedlands

  • John Hunter Hospital & Hunter Medical Research Institute

    New Lambton Heights

  • Sydney Children's Hospital, Randwick

    Randwick

  • Princess Margaret Hospital for Children

    Subiaco

Other trials in Australia

Ireland7 sites

  • Beaumont Hospital

    Dublin

  • Children's University Hospital Temple Street

    Dublin

  • Cork University Hospital

    Dublin

  • Our Lady's Children's Hospital

    Dublin

  • St. Vincent's University Hospital

    Dublin

  • National University of Ireland

    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

  • Coporacio Sanitaria Parc Tauli

    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

  • Pediatric Pulmonary Unit Rambam Medical Center

    Haifa

  • Hadassah Medical Organization

    Jerusalem

  • Schneider Children's Medical Center

    Petah Tikva

  • Sheba Medical Center

    Tel Litwinsky

Other trials in Israel

Canada3 sites

  • Stollery Children's Hospital

    Edmonton, Alberta

  • Queen Elizabeth II Health Sciences Center

    Halifax, Nova Scotia

  • St. Michael's Hospital

    Toronto

Other trials in Canada

Switzerland3 sites

  • Lindenhofspital - Quartier Bleu

    Bern

  • Kinderspital Zuerich

    Zurich

  • Universitaetsspital Zuerich

    Zurich

Other trials in Switzerland

Denmark1 site

  • Juliane Marie Center, Rigshospitalet

    Copenhagen

Other trials in Denmark

Poland1 site

  • Instytut Matki i Dziecka

    Warsaw

Other trials in Poland

About this study

Official title: A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

Conditions
Cystic Fibrosis
Being tested
VX-659/TEZ/IVA, IVA, Placebo
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

    Participants received VX-659/TEZ/IVA orally once daily in the morning.

  • IVADrug

    Also called VX-770, ivacaftor

    Participants received IVA orally once daily in the evening.

  • PlaceboDrug

    Participants received placebo matched VX-659/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening.

Study groups in NCT03447249
Study groupRoleWhat they receive
PlaceboPlacebo comparatorParticipants who received placebo matched to VX-659/TEZ/IVA for 24 weeks in the TC treatment period.
Receives: Drug: Placebo
VX-659/TEZ/IVA TCExperimentalParticipants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period.
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. 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 Week 4

Secondary outcomes (12)

  1. 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 through Week 24

  2. Number of Pulmonary Exacerbations (PEx)

    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 through Week 24

  3. Absolute Change in Sweat Chloride (SwCl)

    Sweat samples were collected using an approved collection device.

    Measured: From Baseline through Week 24

  4. Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score

    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.

    Measured: From Baseline through Week 24

  5. 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 24

  6. Absolute Change in Sweat Chloride

    Sweat samples were collected using an approved collection device.

    Measured: From Baseline at Week 4

  7. Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score

    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.

    Measured: From Baseline at Week 4

  8. Time-to-first Pulmonary Exacerbation (PEx)

    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 through Week 24

  9. Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline

    BMI was defined as weight in kg divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. 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. Higher values are indicative of higher BMI.

    Measured: From Baseline at Week 24

  10. Absolute Change in Body Weight

    Measured: From Baseline at Week 24

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

    Measured: From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks)

  12. Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA

    Measured: Pre-dose on Week 4, 8, 12, and 16

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 start7 Mar 2018
  3. Primary completion5 Feb 2019
  4. Study completion5 Feb 2019

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
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
385 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03447249
Sponsor's protocol ID
VX17-659-102
Other IDs
2017-004132-11, 2017
Status last verified
Mar 2020
First submitted
21 Feb 2018
Last updated
13 Mar 2020
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

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 101 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 2 inclusion and 4 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 13 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 101 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. Some participants in this study may receive a placebo. 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 13 Mar 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?

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) (time frame: From Baseline at Week 4). 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 NCT03447249.

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 13 Mar 2020; AzTrials last refreshed its copy on 30 Oct 2026.

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