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Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02390219
First received: March 11, 2015
Last updated: October 26, 2016
Last verified: January 2016
  Purpose
The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation

Condition Intervention Phase
Cystic Fibrosis Advanced Lung Disease Drug: Lumacaftor Drug: Ivacaftor Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Cystic Fibrosis and Advanced Lung Disease, Homozygous for the F508del-CFTR Mutation

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Safety and tolerability assessments based on adverse events, clinical laboratory values (hematology and serum chemistry), and standard 12 lead electrocardiograms (ECGs) [ Time Frame: Baseline up to 28 days after the last dose (up to Week 28) ]

Secondary Outcome Measures:
  • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) at each visit up to Week 24 [ Time Frame: From Baseline, up to Week 24 ]
  • Absolute change from baseline in FEV1 (in Liters [L]) at each visit up to week 24 [ Time Frame: From Baseline, up to Week 24 ]
  • Total number of days participants received intravenous (IV) antibiotics for pulmonary exacerbations [ Time Frame: Baseline through Week 24 ]
  • Number of hospitalizations (all causes) participants had [ Time Frame: Baseline through Week 24 ]
  • Absolute change from baseline to average of Day 15 and Week 4 measurements in sweat chloride [ Time Frame: Baseline, Day 15 and Week 4 ]
  • Absolute change from baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) respiratory domain score through Week 24 [ Time Frame: Baseline, through Week 24 ]

Enrollment: 46
Study Start Date: March 2015
Study Completion Date: October 2016
Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Lumacaftor/Ivacaftor combination
Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks.
Drug: Lumacaftor
Other Name: VX-809
Drug: Ivacaftor
Other Name: VX-770

  Eligibility

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

Inclusion Criteria:

  • Homozygous for the F508del-CFTR mutation; historical genotype must be documented in the participant's source documents.
  • Percent predicted FEV1 <40 of adjusted for age, sex, and height at Screening

Exclusion Criteria:

  • Participant currently receiving invasive mechanical ventilation.
  • 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
  • Any clinically significant laboratory abnormalities at screening that would interfere with the study assessments or pose an undue risk for the subject
  • A 12-lead electrocardiograms (ECG) demonstrating QTcF >450 msec at Screening
  • History of solid organ or hematological transplantation
  • History of alcohol or drug abuse in the past year
  • Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening.
  • Use of strong inhibitors, moderate inducers, or strong inducers of CYP3A
  • Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at Screening and Day 1.
  • Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
  • Use of beta blockers or the equivalent at Screening.
  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: NCT02390219

Locations
United States, Colorado
Denver, Colorado, United States
United States, Florida
Tampa, Florida, United States
United States, Illinois
Chicago, Illinois, United States
United States, Missouri
St. Louis, Missouri, United States
United States, Pennsylvania
Pittsburgh, Pennsylvania, United States
United States, Texas
Houston, Texas, United States
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02390219     History of Changes
Other Study ID Numbers: VX14-809-106
Study First Received: March 11, 2015
Last Updated: October 26, 2016

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Lung Diseases
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on July 26, 2017