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A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation

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ClinicalTrials.gov Identifier: NCT02392234
Recruitment Status : Completed
First Posted : March 18, 2015
Results First Posted : June 12, 2018
Last Update Posted : June 12, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation predicted to have residual function.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-661/Ivacaftor Drug: Ivacaftor Drug: Placebo matched to VX-661/ ivacaftor Drug: Placebo matched to Ivacaftor Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 248 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a Second Allele With a CFTR Mutation Predicted to Have Residual Function
Study Start Date : March 2015
Actual Primary Completion Date : February 2017
Actual Study Completion Date : February 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: VX-661/Ivacaftor combination Drug: VX-661/Ivacaftor
Fixed dose combination tablet, oral use
Other Name: VX-661+VX-770

Drug: Ivacaftor
Tablet, oral use
Other Name: IVA, VX-770

Drug: Placebo matched to Ivacaftor
Tablet, oral use

Experimental: Ivacaftor monotherapy Drug: Ivacaftor
Tablet, oral use
Other Name: IVA, VX-770

Drug: Placebo matched to VX-661/ ivacaftor
Fixed dose combination tablet, oral use

Placebo Comparator: Placebo Drug: Placebo matched to VX-661/ ivacaftor
Fixed dose combination tablet, oral use

Drug: Placebo matched to Ivacaftor
Tablet, oral use




Primary Outcome Measures :
  1. Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8 [ Time Frame: Baseline, Week 4 and Week 8 of each treatment period ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.


Secondary Outcome Measures :
  1. Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8 [ Time Frame: Baseline, Week 4 and Week 8 of each treatment period ]
    The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.

  2. Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Day 1 up to Week 28 ]
  3. Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8 [ Time Frame: Baseline, Week 4 and Week 8 of each treatment period ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  4. Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8 [ Time Frame: Baseline, Week 4 and Week 8 of each treatment period ]
  5. Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy [ Time Frame: Pre-morning dose on Week 8 of each treatment period ]
  6. Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy [ Time Frame: Pre-morning dose on Week 8 of each treatment period ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Heterozygous for F508del-CFTR and a second allele with a CFTR mutation predicted to have residual function
  • Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (≥) 40 percent (%) and less than or equal to (≤) 90% of predicted normal for age, sex, and height during screening
  • Sweat chloride value ≥60 millimole per liter (mmol/L) during screening OR as documented in the participant's medical record
  • Stable CF disease as judged by the investigator

Exclusion Criteria:

  • History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant
  • An acute upper or lower respiratory infection, pulmonary exacerbation
  • History of solid organ or hematological transplantation
  • Ongoing or prior participation in an investigational drug study (including studies investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of screening
  • Pregnant and nursing females
  • Sexually active participants of reproductive potential who are not willing to follow the contraception requirements

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02392234


Locations
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United States, Alabama
Birmingham, Alabama, United States
United States, Arizona
Phoenix, Arizona, United States
Tucson, Arizona, United States
United States, California
Long Beach, California, United States
Oakland, California, United States
Palo Alto, California, United States
Sacramento, California, United States
United States, Colorado
Aurora, Colorado, United States
Denver, Colorado, United States
United States, Florida
Gainesville, Florida, United States
Miami, Florida, United States
Orlando, Florida, United States
Pensacola, Florida, United States
Tampa, Florida, United States
United States, Georgia
Atlanta, Georgia, United States
United States, Illinois
Chicago, Illinois, United States
Park Ridge, Illinois, United States
United States, Iowa
Iowa City, Iowa, United States
United States, Maryland
Baltimore, Maryland, United States
United States, Massachusetts
Boston, Massachusetts, United States
United States, Michigan
Ann Arbor, Michigan, United States
Grand Rapids, Michigan, United States
United States, Minnesota
Minneapolis, Minnesota, United States
United States, Missouri
Saint Louis, Missouri, United States
United States, New Hampshire
Lebanon, New Hampshire, United States
United States, New York
New York, New York, United States
Syracuse, New York, United States
Valhalla, New York, United States
United States, North Carolina
Chapel Hill, North Carolina, United States
United States, Ohio
Akron, Ohio, United States
Cincinnati, Ohio, United States
Cleveland, Ohio, United States
Toledo, Ohio, United States
United States, Oregon
Portland, Oregon, United States
United States, Pennsylvania
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
United States, South Carolina
Charleston, South Carolina, United States
United States, South Dakota
Sioux Falls, South Dakota, United States
United States, Tennessee
Memphis, Tennessee, United States
United States, Texas
Austin, Texas, United States
Dallas, Texas, United States
Tyler, Texas, United States
United States, Utah
Salt Lake City, Utah, United States
United States, Virginia
Norfolk, Virginia, United States
United States, Washington
Seattle, Washington, United States
Spokane, Washington, United States
United States, Wisconsin
Milwaukee, Wisconsin, United States
Australia
Adelaide, Australia
Melbourne, Australia
South Brisbane, Australia
Westmead, Australia
Belgium
Gent, Belgium
Canada
Montreal, Canada
Quebec City, Canada
Toronto, Canada
Vancouver, Canada
France
Marseille cedex 20, Bouches-du-Rhone, France
Montpellier cedex 5, Herault, France
Rennes cedex 09, Ille Et Vilaine, France
Lille cedex, Nord, France
Paris cedex 14, Paris, France
Paris cedex 15, Paris, France
Bron, Rhone, France
Bordeaux Cedex, France
Germany
Muenchen, Bayern, Germany
Munchen, Bayern, Germany
Hannover, Niedersachsen, Germany
Essen, Nordrhein Westfalen, Germany
Jena, Thueringen, Germany
Berlin, Germany
Israel
Haifa, Israel
Jerusalem, Israel
Petach-Tikva, Israel
Ramat-Gan, Israel
Italy
Ancona, Italy
Milano, Italy
Orbassano, Italy
Potenza, Italy
Roma, Italy
Verona, Italy
Netherlands
Amsterdam, Netherlands
Den Haag, Netherlands
Rotterdam, Netherlands
Utrecht, Netherlands
Switzerland
Bern, Switzerland
St Gallen, Switzerland
Zuerich, Switzerland
United Kingdom
Exeter, Devon, United Kingdom
London, Greater London, United Kingdom
Manchester, Greater Manchester, United Kingdom
Southampton, Hampshire, United Kingdom
Liverpool, Lancashire, United Kingdom
Glasgow, Strathclyde, United Kingdom
Leeds, West Yorkshire, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] June 10, 2016
Statistical Analysis Plan  [PDF] February 24, 2017

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02392234    
Other Study ID Numbers: VX14-661-108
2014-004788-18 ( EudraCT Number )
First Posted: March 18, 2015    Key Record Dates
Results First Posted: June 12, 2018
Last Update Posted: June 12, 2018
Last Verified: May 2018
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action