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Trial record 1 of 1 for:    VX15-371-101
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A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2016 by Vertex Pharmaceuticals Incorporated
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02709109
First received: February 29, 2016
Last updated: February 14, 2017
Last verified: December 2016
  Purpose
The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in saline compared to saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the F508del-CFTR mutation, and being treated with Orkambi

Condition Intervention Phase
Cystic Fibrosis
Drug: VX-371
Drug: Saline
Drug: Placebo
Drug: Orkambi
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Participant, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official Title: A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Safety & Tolerability as measured by number of subjects who experience AEs, and clinically significant changes in ECG parameters, vital signs, Laboratory tests, Spirometry, and ophthalmologic tests. [ Time Frame: from baseline up to 28 days post last administration of study drug, up to 16 Weeks ]
  • Safety & Tolerability as measured by percentage of subjects who experience AEs, and clinically significant changes in ECG parameters, vital signs, Laboratory tests, Spirometry, and ophthalmologic tests. [ Time Frame: from baseline up to 28 days post last administration of study drug, up to 16 Weeks ]
  • Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: from study baseline at Day 28 in each Treatment Period ]

Secondary Outcome Measures:
  • PK parameter estimation of VX-371 trough concentration (Ctrough) [ Time Frame: from study baseline at Day 28 in each Treatment Period ]

Estimated Enrollment: 150
Study Start Date: February 2016
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: VX-371 + Saline , then Saline
Participants receive VX-371 + Saline during Treatment Period 1 followed by Saline during Treatment Period 2. A washout period of 28 Days will be maintained between the two treatment periods. All subjects must be receiving stable treatment with Orkambi before the first dose of study drug and throughout the duration of the study.
Drug: VX-371 Drug: Saline Drug: Orkambi
Other Name: lumacaftor/ivacaftor
Experimental: Saline, then VX-371 + Saline
Participants receive Saline during Treatment Period 1 followed by VX-371 + Saline during Treatment Period 2. A washout period of 28 Days will be maintained between the two treatment periods. All subjects must be receiving stable treatment with Orkambi before the first dose of study drug and throughout the duration of the study.
Drug: VX-371 Drug: Saline Drug: Orkambi
Other Name: lumacaftor/ivacaftor
Experimental: VX-371 + Placebo, then Placebo
Participants receive VX-371 + placebo (saline) during Treatment Period 1 followed by placebo (saline) during Treatment Period 2. A washout period of 28 Days will be maintained between the two treatment periods. All subjects must be receiving stable treatment with Orkambi before the first dose of study drug and throughout the duration of the study.
Drug: VX-371 Drug: Placebo Drug: Orkambi
Other Name: lumacaftor/ivacaftor
Experimental: Placebo, then VX-371 + Placebo
Participants receive placebo (saline) during Treatment Period 1 followed by VX-371 + placebo (saline) during Treatment Period 2. A washout period of 28 Days will be maintained between the two treatment periods. All subjects must be receiving stable treatment with Orkambi before the first dose of study drug and throughout the duration of the study.
Drug: VX-371 Drug: Placebo Drug: Orkambi
Other Name: lumacaftor/ivacaftor

  Eligibility

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

Inclusion Criteria:

  • Willing and able to use the delivery device as directed by the study manual.
  • Confirmed diagnosis of CF, defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis.
  • Homozygous for the F508del CFTR mutation. If the CFTR screening genotype result is not received before randomization, a previous CFTR genotype lab report may be used to establish eligibility.
  • Percent predicted FEV1 of ≥40 to <90 percentage points adjusted for age, sex, and height according to the Global Lung Initiative (GLI) at the Screening Visit.
  • Willing to discontinue physician-prescribed saline use.
  • Female subjects of childbearing potential with a negative serum pregnancy test at the Screening Visit.

Exclusion Criteria:

  • History of any comorbidity, which 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.
  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug).
  • A 12 lead ECG demonstrating QTcF >450 msec at the Screening Visit.
  • History of solid organ or hematological transplantation.
  • Used diuretics or renin-angiotensin aldosterone system antihypertensive drugs in the 28 days prior to Screening or an anticipated need for any of these medications during the study.
  • Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit.
  • Inability to withhold short-acting, long-acting, or once-daily, long-acting bronchodilator use for 4, 12, or 24 hours prior to clinic visit, respectively.
  • History of significant intolerance to inhaled saline, or intolerance to the single dose of saline at Screening
  • Known hypersensitivity or history of intolerance to Orkambi.
  • Pregnant and nursing females.
  • Subjects who have participated in Parion Sciences Study NCT02343445.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02709109

Contacts
Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

  Show 40 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02709109     History of Changes
Other Study ID Numbers: VX15-371-101
2015-004841-13 ( EudraCT Number )
Study First Received: February 29, 2016
Last Updated: February 14, 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

ClinicalTrials.gov processed this record on May 23, 2017