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A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor

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ClinicalTrials.gov Identifier: NCT02412111
Recruitment Status : Completed
First Posted : April 8, 2015
Results First Posted : January 8, 2019
Last Update Posted : January 8, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Ivacaftor Drug: Tezacaftor/Ivacaftor Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 156 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of 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 CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive
Actual Study Start Date : June 2015
Actual Primary Completion Date : September 2017
Actual Study Completion Date : September 2017

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: Ivacaftor (Run-in Period)
Ivacaftor 150 milligram (mg) tablet orally every 12 hours for 4 weeks.
Drug: Ivacaftor
Other Name: VX-770

Experimental: VX-661 + Ivacaftor (Active comparator period)
VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning and ivacaftor 150 mg tablet orally once daily in the evening for 8 weeks.
Drug: Ivacaftor
Other Name: VX-770

Drug: Tezacaftor/Ivacaftor
Other Name: VX-661/VX-770

Active Comparator: Ivacaftor monotherapy (Active comparator period)
Ivacaftor 150 mg tablet orally every 12 hours as monotherapy for 8 weeks.
Drug: Ivacaftor
Other Name: VX-770




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


Secondary Outcome Measures :
  1. Relative Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8 [ Time Frame: Baseline, Through Week 8 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  2. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline Through Week 8 [ Time Frame: Baseline, Through Week 8 ]
    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.

  3. Absolute Change From Baseline in Sweat Chloride Through Week 8 [ Time Frame: Baseline, Through Week 8 ]
    Sweat samples were collected using an approved collection device.

  4. Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Week 16 ]
  5. Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA) [ Time Frame: Predose on Week -2 for Run-in period; Pre-dose on Week 2 for Active comparator 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 mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
  • FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height during screening
  • 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.
  • Pregnant and nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Week -4 Visits).
  • Sexually active subjects 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): NCT02412111


  Show 68 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] April 18, 2017
Statistical Analysis Plan  [PDF] July 28, 2017


Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02412111     History of Changes
Other Study ID Numbers: VX14-661-109
First Posted: April 8, 2015    Key Record Dates
Results First Posted: January 8, 2019
Last Update Posted: January 8, 2019
Last Verified: January 2019

Additional relevant MeSH terms:
Fibrosis
Cystic 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