ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis Who Have an 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: NCT02565914
Recruitment Status : Enrolling by invitation
First Posted : October 1, 2015
Last Update Posted : September 13, 2017
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This is a Phase 3, multicenter, open-label, 2-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in Studies NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111, and NCT02508207. The study is designed to evaluate the safety and efficacy of long term treatment of VX-661 in combination with ivacaftor.

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

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 1116 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Study Start Date : August 2015
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : September 2019

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: Part A Treatment Cohort: VX-661/ivacaftor
VX-661 100 mg/ ivacaftor 150 mg fixed dose combination (FDC) tablet daily (qd) in the morning and ivacaftor 150 mg tablet qd in the evening
Drug: VX-661/ivacaftor
Drug: ivacaftor
No Intervention: Part A Observational Cohort
Long-term Follow-up
Experimental: Part B Treatment: VX-661/ivacaftor
VX-661 100 mg/ ivacaftor 150 mg fixed dose combination (FDC) tablet daily (qd) in the morning and ivacaftor 150 mg tablet qd in the evening
Drug: VX-661/ivacaftor
Drug: ivacaftor



Primary Outcome Measures :
  1. Part A: Safety and tolerability of long-term treatment of VX-661 in combination with ivacaftor based on adverse events (AEs), ophthalmologic exams, clinical laboratory values, standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry [ Time Frame: from baseline through Study Completion (up to 3 years) ]

Secondary Outcome Measures :
  1. Parts A and B: Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline through Week 96 ]
  2. Part A: Relative change from baseline in ppFEV1 [ Time Frame: from baseline through Week 96 ]
  3. Parts A and B: Number of pulmonary exacerbations [ Time Frame: from baseline through Week 96 ]
  4. Parts A and B: Absolute change from baseline in body mass index (BMI) [ Time Frame: from baseline through Week 96 ]
  5. Parts A and B: Absolute change from baseline in BMI z-score for subjects aged <20 years [ Time Frame: from baseline through Week 96 ]
  6. Part A: Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through Week 96 ]
  7. Part A: Absolute change from baseline in body weight [ Time Frame: from baseline through Week 96 ]
  8. Part A: Absolute change from baseline in body weight z-score for subjects aged <20 years [ Time Frame: from baseline through Week 96 ]
  9. Part A: Absolute change from baseline in height z-score for subjects aged <20 years [ Time Frame: from baseline through Week 96 ]
  10. Part A: Time-to-first pulmonary exacerbation [ Time Frame: from baseline through Week 96 ]
  11. Part A: Pharmacokinetic (PK) parameters: trough concentrations of VX-661 , a VX-661 metabolite (M1-661), ivacaftor, ivacaftor metabolite (M1-ivacaftor) [ Time Frame: from baseline through Week 96 ]
  12. Part A: Observational Cohort: Safety, as determined by related serious adverse events (SAEs) [ Time Frame: from baseline through study Completion (up to 3 years) ]
  13. Part B: Safety and tolerability assessments including number of subjects with adverse events (AEs) and serious adverse events [ Time Frame: from baseline through safety follow-up visit ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

Inclusion Criteria:

Part A:

  • Subjects entering the Treatment Cohort must meet all of the following criteria:
  • Elect to enroll in the Treatment Cohort
  • Completed study drug Treatment Period in a parent study (NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111) or study drug treatment and the Safety Follow up Visit for subjects from NCT02508207.
  • Willing to remain on a stable CF regimen through the Safety Follow-up Visit.
  • Subjects re-enrolling in the Part A Treatment Cohort must meet all of the following criteria:

    • Previously received at least 4 weeks of study drug before discontinuing in Part A of Study NCT02565914 to participate in another qualified Vertex study.
    • Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part A Study NCT02565914.
  • Subjects entering the Part A Observational Cohort must meet the following criteria:

    • <18 years of age (age on the date of informed consent/assent in the parent study)
    • Completed study drug Treatment Period in a parent study or study drug treatment and the Safety Follow up Visit for subjects from NCT02508207, but do not elect to enroll in the NCT02565914 Treatment Cohort; or
    • Received at least 4 weeks of study drug treatment and completed visits up to the last scheduled visit of the Treatment Period of a parent study (and the Safety Follow up Visit for subjects from NCT02508207), but do not meet eligibility criteria for enrollment into the Treatment Cohort

Part B:

Part B Subjects who meet all of the following inclusion criteria will be eligible for Part B.

  • Did not withdraw consent from the parent study or Part A of Study NCT02565914.
  • Completed study drug treatment during the Treatment Period in Part A of NCT02565914, Studies NCT02730208 or NCT03150719, or other eligible Vertex studies.
  • Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit of Study NCT02565914.

Subjects re enrolling in Part B must meet all of the following criteria:

  • Previously received at least 4 weeks of study drug before discontinuing Study NCT02565914 to participate in another qualified Vertex study, which is defined as a Vertex study of investigational CFTR modulators that allows participation of subjects in Study NCT02565914.
  • Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part B.
  • Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit in Part B.

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 to the subject.
  • Pregnant and nursing females.
  • Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
  • History of drug intolerance in the parent study that would pose an additional risk to the subject.
  • Participation in an investigational drug trial (including studies investigating VX-661/ivacaftor or lumacaftor/ivacaftor) other than the parent studies of NCT02565914 or other eligible Vertex studies investigating VX-661 in combination with ivacaftor, or use of a commercially available CFTR modulator.

Other protocol defined Inclusion/Exclusion criteria may apply.


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): NCT02565914


  Show 158 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02565914     History of Changes
Other Study ID Numbers: VX14-661-110
2014-004827-29 ( EudraCT Number )
First Posted: October 1, 2015    Key Record Dates
Last Update Posted: September 13, 2017
Last Verified: August 2017

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