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CompletedNCT03447249Updated Mar 13, 2020Results posted

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 interventional study of VX-659/TEZ/IVA and IVA in Cystic Fibrosis, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 101 sites in 11 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2020-03-13.

Sponsored by Vertex Pharmaceuticals Incorporated · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
385
Allocation
Randomized
Ages
12 Years and older
Sex
All
01

Study 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).

02

Conditions studied

  • Cystic Fibrosis

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03

In context

Cystic Fibrosis

1,605 studies on the registry are indexed under Cystic Fibrosis; 196 are open to participants now.

This study's enrollment of 385 is above the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Vertex Pharmaceuticals Incorporated is the lead sponsor of 249 studies on the registry; 20 are open to participants now.

Of its 106 completed or terminated interventional studies of FDA-regulated products, 53 (50%) have results posted.

Browse Vertex Pharmaceuticals Incorporated studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • 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

Key Exclusion Criteria:

  • 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

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
385 participants (actual)

Study arms

  • Placebo comparator
    Placebo

    Participants who received placebo matched to VX-659/TEZ/IVA for 24 weeks in the TC treatment period.

    Drug: Placebo

  • Experimental
    VX-659/TEZ/IVA TC

    Participants 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.

    Drug: VX-659/TEZ/IVA · Drug: IVA

Interventions

  • DrugVX-659/TEZ/IVA

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

    Also known as: VX-659/VX-661/VX-770, VX-659/tezacaftor/ivacaftor

  • DrugIVA

    Participants received IVA orally once daily in the evening.

    Also known as: VX-770, ivacaftor

  • DrugPlacebo

    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.

06

What researchers measure

Primary outcomes

  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.

    Time frame: From Baseline at Week 4

Secondary outcomes

  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.

    Time frame: 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.

    Time frame: From Baseline through Week 24

  3. Absolute Change in Sweat Chloride (SwCl)

    Sweat samples were collected using an approved collection device.

    Time frame: 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.

    Time frame: 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).

    Time frame: From Baseline at Week 24

  6. Absolute Change in Sweat Chloride

    Sweat samples were collected using an approved collection device.

    Time frame: 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.

    Time frame: 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.

    Time frame: 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.

    Time frame: From Baseline at Week 24

  10. Absolute Change in Body Weight

    Time frame: 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)

    Time frame: 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

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

07

Results

Posted Mar 13, 2020

Participant flow

A total of 385 participants were enrolled in the study, of which 3 participants were enrolled but were not dosed in the TC treatment period. Results are presented for 382 participants dosed in the TC treatment period.

Participant flow — Overall Study
MilestonePlaceboVX-659/TEZ/IVA TC
Started190192
Safety set189193
Completed185192
Not completed50
Withdrew: Adverse event10
Withdrew: Withdrawal of consent (not due to ae)10
Withdrew: Other30

Outcome measures

PrimaryAbsolute 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.

Time frame:
From Baseline at Week 4
Reported as:
Least squares mean · percentage points
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
percentage pointsPlaceboVX-659/TEZ/IVA TC
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)-1.0 ± 0.613.0 ± 0.6
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Mixed-effects model for repeated measure · p = <0.0001 · Least squares (ls) mean difference: 14.0 · 95% CI 12.4 to 15.7
SecondaryAbsolute 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.

Time frame:
From Baseline through Week 24
Reported as:
Least squares mean · percentage points
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
percentage pointsPlaceboVX-659/TEZ/IVA TC
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)-0.8 ± 0.613.4 ± 0.6
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Mixed-effects model for repeated measure · p = <0.0001 · Ls mean difference: 14.2 · 95% CI 12.6 to 15.7
SecondaryNumber 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.

Time frame:
From Baseline through Week 24
Reported as:
Number · pulmonary exacerbation events
Number of Pulmonary Exacerbations (PEx)
pulmonary exacerbation eventsPlaceboVX-659/TEZ/IVA TC
Number of Pulmonary Exacerbations (PEx)11617
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Negative binomial regression model · p = <0.0001 · Rate ratio: 0.14 · 95% CI 0.09 to 0.24
SecondaryAbsolute Change in Sweat Chloride (SwCl)

Sweat samples were collected using an approved collection device.

Time frame:
From Baseline through Week 24
Reported as:
Least squares mean · millimole per liter (mmol/L)
Absolute Change in Sweat Chloride (SwCl)
millimole per liter (mmol/L)PlaceboVX-659/TEZ/IVA TC
Absolute Change in Sweat Chloride (SwCl)-0.1 ± 1.0-44.6 ± 0.9
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Mixed-effects model for repeated measure · p = <0.0001 · Ls mean difference: -44.6 · 95% CI -47.2 to -41.9
SecondaryAbsolute 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.

Time frame:
From Baseline through Week 24
Reported as:
Least squares mean · units on a scale
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
units on a scalePlaceboVX-659/TEZ/IVA TC
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score-1.5 ± 1.118.6 ± 1.0
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Mixed-effects model for repeated measure · p = <0.0001 · Ls mean difference: 20.1 · 95% CI 17.2 to 23.0
SecondaryAbsolute Change in Body Mass Index (BMI)

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

Time frame:
From Baseline at Week 24
Reported as:
Least squares mean · kilogram per meter square (kg/m^2)
Absolute Change in Body Mass Index (BMI)
kilogram per meter square (kg/m^2)PlaceboVX-659/TEZ/IVA TC
Absolute Change in Body Mass Index (BMI)-0.05 ± 0.071.06 ± 0.07
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Mixed-effects model for repeated measure · p = <0.0001 · Ls mean difference: 1.11 · 95% CI 0.91 to 1.31
SecondaryAbsolute Change in Sweat Chloride

Sweat samples were collected using an approved collection device.

Time frame:
From Baseline at Week 4
Reported as:
Least squares mean · mmol/L
Absolute Change in Sweat Chloride
mmol/LPlaceboVX-659/TEZ/IVA TC
Absolute Change in Sweat Chloride0.0 ± 1.0-43.3 ± 1.0
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Mixed-effects model for repeated measure · p = <0.0001 · Ls mean difference: -43.4 · 95% CI -46.3 to -40.5
SecondaryAbsolute 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.

Time frame:
From Baseline at Week 4
Reported as:
Least squares mean · units on a scale
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
units on a scalePlaceboVX-659/TEZ/IVA TC
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score0.1 ± 1.218.0 ± 1.2
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Mixed-effects model for repeated measure · p = <0.0001 · Ls mean difference: 17.9 · 95% CI 14.5 to 21.3
SecondaryTime-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.

Time frame:
From Baseline through Week 24
Reported as:
Median · days
Time-to-first Pulmonary Exacerbation (PEx)
daysPlaceboVX-659/TEZ/IVA TC
Time-to-first Pulmonary Exacerbation (PEx)NA (NA to NA)NA (NA to NA)
SecondaryAbsolute 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.

Time frame:
From Baseline at Week 24
Reported as:
Least squares mean · kg/m^2
Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline
kg/m^2PlaceboVX-659/TEZ/IVA TC
Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline-0.08 ± 0.050.31 ± 0.05
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Ls mean difference: 0.39 · 95% CI 0.24 to 0.54
SecondaryAbsolute Change in Body Weight
Time frame:
From Baseline at Week 24
Reported as:
Least squares mean · kg
Absolute Change in Body Weight
kgPlaceboVX-659/TEZ/IVA TC
Absolute Change in Body Weight0.1 ± 0.23.3 ± 0.2
Statistical analysis
  • Placebo vs VX-659/TEZ/IVA TC · Ls mean difference: 3.2 · 95% CI 2.7 to 3.8
SecondarySafety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame:
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)
Reported as:
Number · participants
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
participantsPlaceboVX-659/TEZ/IVA TC
Participants with TEAEs175173
Participants with Serious TEAEs5811
SecondaryObserved Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA
Time frame:
Pre-dose on Week 4, 8, 12, and 16
Reported as:
Mean · nanogram per milliliter (ng/mL)
Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA
nanogram per milliliter (ng/mL)VX-659/TEZ/IVA TC
VX-659 (Week 4)662 ± 528
VX-659 (Week 8)764 ± 1520
VX-659 (Week 12)614 ± 519
VX-659 (Week 16)638 ± 521
TEZ (Week 4)1220 ± 645
TEZ (Week 8)1390 ± 1250
TEZ (Week 12)1290 ± 766
TEZ (Week 16)1220 ± 654
M1-TEZ (Week 4)4380 ± 1560
M1-TEZ (Week 8)4580 ± 1480
M1-TEZ (Week 12)4580 ± 1530
M1-TEZ (Week 16)4420 ± 1520
IVA (Week 4)442 ± 277
IVA (Week 8)548 ± 1100
IVA (Week 12)429 ± 327
IVA (Week 16)416 ± 303

Adverse events

Collected over 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). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Placebo0/189 (0%)58/189 (30.7%)166/189 (87.8%)
VX-659/TEZ/IVA TC0/193 (0%)11/193 (5.7%)141/193 (73.1%)
Most frequent serious events
Showing 10 of 32
Most frequent serious events
EventPlaceboVX-659/TEZ/IVA TC
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations45/1893/193
HaemoptysisRespiratory, thoracic and mediastinal disorders3/1890/193
Distal intestinal obstruction syndromeGastrointestinal disorders2/1890/193
Lower respiratory tract infection bacterialInfections and infestations2/1890/193
Intentional self-injuryPsychiatric disorders1/1890/193
Pneumothorax traumaticInjury, poisoning and procedural complications1/1890/193
Forced expiratory volume decreasedInvestigations1/1890/193
Weight decreasedInvestigations1/1890/193
Pericardial effusionCardiac disorders1/1890/193
Right-to-left cardiac shuntCongenital, familial and genetic disorders1/1890/193
Most frequent other events
Showing 10 of 22
Most frequent other events
EventPlaceboVX-659/TEZ/IVA TC
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations79/18921/193
CoughRespiratory, thoracic and mediastinal disorders68/18934/193
Sputum increasedRespiratory, thoracic and mediastinal disorders38/18928/193
Upper respiratory tract infectionInfections and infestations7/18935/193
HeadacheNervous system disorders31/18926/193
NasopharyngitisInfections and infestations16/18926/193
NauseaGastrointestinal disorders24/1898/193
Oropharyngeal painRespiratory, thoracic and mediastinal disorders13/18920/193
HaemoptysisRespiratory, thoracic and mediastinal disorders19/1896/193
FatigueGeneral disorders19/1899/193

Baseline characteristics

All randomized participants who carried the intended CF transmembrane conductance regulator gene (CFTR) allele mutation and received at least 1 dose of study drug in the TC Treatment Period.

Age, Continuous
Age, Continuous(years)PlaceboVX-659/TEZ/IVA TCTotal
Mean27.1 ± 10.026.7 ± 9.826.9 ± 9.9
Sex: Female, Male
Sex: Female, Male(Participants)PlaceboVX-659/TEZ/IVA TCTotal
Female8385168
Male107107214
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)PlaceboVX-659/TEZ/IVA TCTotal
Hispanic or Latino7613
Not Hispanic or Latino181184365
Unknown or Not Reported224
Race
Race(Participants)PlaceboVX-659/TEZ/IVA TCTotal
White187188375
Black or African American123
Both White and Black/African American224
Forced Expiratory Volume in 1 Second (ppFEV1)
Forced Expiratory Volume in 1 Second (ppFEV1)(percentage points)PlaceboVX-659/TEZ/IVA TCTotal
Mean60.4 ± 14.560.7 ± 15.460.6 ± 14.9
08

Study locations

101 sites
  • University of Alabama at Birmingham
    Birmingham, Alabama 35233, United States
  • Stanford University
    Palo Alto, California 94304, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Hartford Health
    Hartford, Connecticut 06106, United States
  • Yale New Haven Medical Center
    New Haven, Connecticut 06511, United States
  • University of Miami/ Miller School of Medicine
    Miami, Florida 33136, United States
  • Nicklaus Children's Hospital
    Miami, Florida 33155, United States
  • Arnold Palmer Hospital
    Orlando, Florida 32806, United States
  • Johns Hopkins All Children's Hospital Outpatient Care Center
    St. Petersburg, Florida 33701, United States
  • St. Luke's CF Center of Idaho
    Boise, Idaho 83712, United States
  • Cystic Fibrosis Center of Chicago
    Glenview, Illinois 60025, United States
  • Advocate Children's Hospital - Park Ridge/ North Suburban Pulmonary and Critical Care Consultants
    Niles, Illinois 60714, United States
  • Indiana Clinical Research Center, IU Health University Hospital
    Indianapolis, Indiana 46202, United States
  • The University of Iowa Hospitals and Clinics
    Iowa City, Iowa 52242, United States
  • Kentucky Clinic
    Lexington, Kentucky 40536, United States
  • Kosair Charities Pediatric Clinical Research Unit
    Louisville, Kentucky 40202, United States
  • The Johns Hopkins Hospital
    Baltimore, Maryland 21287, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • University of Massachusetts Memorial Medical Center
    Worcester, Massachusetts 01655, United States
  • University of Michigan Health System
    Ann Arbor, Michigan 48109, United States
  • Helen DeVos Children's Hospital CF Center
    Grand Rapids, Michigan 49503, United States
  • University of Minnesota
    Minneapolis, Minnesota 55455, United States
  • University of Mississippi Medical Center
    Jackson, Mississippi 39216, United States
  • Children's Mercy Hospital
    Kansas City, Missouri 64108, United States
  • Washington University School of Medicine/ St. Louis Children's Hospital
    St Louis, Missouri 63110, United States
  • Dartmouth Hitchcock Medical Center, Lebanon
    Lebanon, New Hampshire 03756, United States
  • Rutgers-Robert Wood Johnson Medical School
    New Brunswick, New Jersey 80901, United States
  • Albany Medical College
    Albany, New York 12208, United States
  • Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
    Buffalo, New York 14222, United States
  • Northwell Health, Long Island Jewish Medical Center
    New Hyde Park, New York 11040, United States
  • Columbia University Medical Center
    New York, New York 10032, United States
  • SUNY Upstate Medical University
    Syracuse, New York 13210, United States
  • Clinical Research of Charlotte
    Charlotte, North Carolina 28277, United States
  • Duke University Medical Center
    Durham, North Carolina 27710, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Respiratory Diseases of Children and Adolescents
    Oklahoma City, Oklahoma 73112, United States
  • Oregon Health & Science University
    Portland, Oregon 97239, United States
  • Drexel University College of Medicine/ Drexel Adult Cystic Fibrosis Center
    Philadelphia, Pennsylvania 19017, United States
  • Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center
    Pittsburgh, Pennsylvania 15224, United States
  • Sanford Research/ USD
    Sioux Falls, South Dakota 57105, United States
  • University of Tennessee Medical Center - Adult Cystic Fibrosis Clinic
    Knoxville, Tennessee 37920, United States
  • Children's Foundation Research Center/ Le Bonheur Children's Hospital
    Memphis, Tennessee 38103, United States
  • Vanderbilt University Medical Center
    Nashville, Tennessee 37920, United States
  • Cook Children's Medical Center
    Fort Worth, Texas 76104, United States
  • Baylor College of Medicine
    Houston, Texas 77030, United States
  • University of Utah / Primary Children's Medical Center
    Salt Lake City, Utah 84132, United States
  • Seattle Children's Hospital
    Seattle, Washington 98105, United States
  • Providence Pediatric Pulmonary & Allergy/Immunology Clinic
    Spokane, Washington 99204, United States
  • The Alfred Hospital
    Melbourne, Victoria, Australia
  • Royal Adelaide Hospital
    Adelaide, Australia
  • Prince Charles Hospital
    Chermside, Australia
  • Royal Brisbane & Women's Hospital
    Herston, Australia
  • Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital
    Nedlands, Australia
  • John Hunter Hospital & Hunter Medical Research Institute
    New Lambton Heights, Australia
  • Sydney Children's Hospital, Randwick
    Randwick, Australia
  • Princess Margaret Hospital for Children
    Subiaco, Australia
  • Stollery Children's Hospital
    Edmonton, Alberta, Canada
  • Queen Elizabeth II Health Sciences Center
    Halifax, Nova Scotia, Canada
  • St. Michael's Hospital
    Toronto, Canada
  • Juliane Marie Center, Rigshospitalet
    Copenhagen, Denmark
  • Charite Paediatric Pulmonology Department
    Berlin, Germany
  • University Hospital Cologne
    Cologne, Germany
  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
    Essen, Germany
  • Clinic of J.W Goethe University
    Frankfurt, Germany
  • Medizinische Hochschule Hannover
    Hanover, Germany
  • Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder - und Jugendmedizin
    Jena, Germany
  • Universitatsklinikum Schleswig-Holstein, Klinik fur Kinder- und Jugendmedizin
    Lübeck, Germany
  • Klinikum Innenstadt, University of Munich
    München, Germany
  • Pneumologische Praxis Pasing
    München, Germany
  • Beaumont Hospital
    Dublin, Ireland
  • Children's University Hospital Temple Street
    Dublin, Ireland
  • Cork University Hospital
    Dublin, Ireland
  • Our Lady's Children's Hospital
    Dublin, Ireland
  • St. Vincent's University Hospital
    Dublin, Ireland
  • National University of Ireland
    Galway, Ireland
  • University Hospital Limerick
    Limerick, Ireland
  • Lady Davis Carmel Medical Center
    Haifa, Israel
  • Pediatric Pulmonary Unit Rambam Medical Center
    Haifa, Israel
  • Hadassah Medical Organization
    Jerusalem, Israel
  • Schneider Children's Medical Center
    Petah Tikva, Israel
  • Sheba Medical Center
    Tel Litwinsky, Israel
  • Instytut Matki i Dziecka
    Warsaw, Poland
  • Hospital Universitari Vall d Hebron
    Barcelona, Spain
  • Hospital Universitari Vall d'Hebron Servicio de Broncoscopia
    Barcelona, Spain
  • Hospital Universitario 12 de Octubre
    Madrid, Spain
  • Hospital Universitario Infantil La Paz
    Madrid, Spain
  • Coporacio Sanitaria Parc Tauli
    Sabadell, Spain
  • Hospital Universitario Virgen del Rocio
    Seville, Spain
  • Hospital Universitario y Politecnico La Fe
    Valencia, Spain
  • Lindenhofspital - Quartier Bleu
    Bern, Switzerland
  • Kinderspital Zuerich
    Zurich, Switzerland
  • Universitaetsspital Zuerich
    Zurich, Switzerland
  • Papworth Hospital NHS Foundation Trust, Papworth Everard
    Cambridge, United Kingdom
  • Queen Elizabeth University Hospital
    Glasgow, United Kingdom
  • The Leeds Teaching Hospitals NHS Trust, St. James University Hospital
    Leeds, United Kingdom
  • Liverpool Heart and Chest Hospital
    Liverpool, United Kingdom
  • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
    London, United Kingdom
  • Wythenshawe Hospital
    Manchester, United Kingdom
  • The Newcastle Upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
    Newcastle upon Tyne, United Kingdom
  • Nottingham University Hospitals NHS Trust, Queens Medical Center
    Nottingham, United Kingdom

Showing the first 100 of 101 sites across 11 countries.

09

References and documents

Study documents

  • Study protocol · Apr 27, 2018
  • Statistical analysis plan · Feb 22, 2019

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 13, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03447249
Lead sponsor
Vertex Pharmaceuticals Incorporated
Responsible party
Sponsor
First posted
Feb 27, 2018
Start date
Mar 7, 2018
Primary completion
Feb 5, 2019
Completion
Feb 5, 2019
Results posted
Mar 13, 2020
Last update
Mar 13, 2020

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

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Other studies from this sponsor

Vertex Pharmaceuticals Incorporated →

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