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A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02508207
Recruitment Status : Completed
First Posted : July 24, 2015
Results First Posted : July 27, 2018
Last Update Posted : July 19, 2021
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Tezacaftor/Ivacaftor Drug: Ivacaftor Drug: Tezacaftor/Ivacaftor matching placebo Drug: Ivacaftor matching placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 34 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-Blind, Placebo Controlled, Parallel-Group, Exploratory Study to Evaluate Effects of VX-661 in Combination With Ivacaftor on Lung and Extrapulmonary Systems in Subjects Aged 18 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Study Start Date : February 2016
Actual Primary Completion Date : June 2017
Actual Study Completion Date : June 2017

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Placebo Comparator: Placebo
Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days.
Drug: Tezacaftor/Ivacaftor matching placebo
Drug: Ivacaftor matching placebo
Experimental: TEZ/IVA
Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days.
Drug: Tezacaftor/Ivacaftor
Tezacaftor/Ivacaftor FDC

Drug: Ivacaftor



Primary Outcome Measures :
  1. Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28 [ Time Frame: Baseline, Day 28 ]
    MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28.


Secondary Outcome Measures :
  1. Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 [ Time Frame: Baseline, Day 28 ]
    Percent predicted FEV1 is the ratio of FEV1 to the predicted FEV1, expressed as a percentage. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  2. Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29 [ Time Frame: Baseline, Day 29 ]
    Absolute change from Baseline in small bowel AUC over 1-minute mean pH increments through 30 minutes at Day 29 was assessed.

  3. Absolute Change From Baseline in Sweat Chloride at Day 29 [ Time Frame: Baseline, Day 29 ]
  4. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Day 57 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female participants, homozygous for the F508del CFTR mutation
  • Confirmed diagnosis of CF by sweat chloride testing
  • Forced Expiratory Volume in 1 Second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height at Screening Visit
  • 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 subject.
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
  • History or evidence of clinically significant findings on ophthalmologic examination during the Screening Period.
  • History of solid organ or hematological transplantation
  • Pregnant or nursing females
  • Participants who have had radiation exposure within 1 year before the first mucociliary clearance (MCC) procedure that would cause them to exceed federal regulations by participating in this study
  • In the opinion of the investigator, unable to adequately perform inhalation maneuvers during the MCC procedures

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): NCT02508207


Locations
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United States, Alabama
Birmingham, Alabama, United States
United States, Indiana
Indianapolis, Indiana, United States
United States, Maryland
Baltimore, Maryland, United States
United States, North Carolina
Chapel Hill, North Carolina, United States
United States, Ohio
Cincinnati, Ohio, United States
Toledo, Ohio, United States
United States, Pennsylvania
Pittsburgh, Pennsylvania, United States
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] September 19, 2016
Statistical Analysis Plan  [PDF] July 27, 2017

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02508207    
Other Study ID Numbers: VX14-661-111
First Posted: July 24, 2015    Key Record Dates
Results First Posted: July 27, 2018
Last Update Posted: July 19, 2021
Last Verified: June 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