Overview

This trial has been completed.

Conditions cystic fibrosis, advanced lung disease
Treatments lumacaftor, ivacaftor
Phase phase 3
Sponsor Vertex Pharmaceuticals Incorporated
Start date March 2015
End date October 2016
Trial size 46 participants
Trial identifier NCT02390219, VX14-809-106

Summary

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks.
lumacaftor VX-809
ivacaftor VX-770

Primary Outcomes

Measure
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 Outcomes

Measure
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

Eligibility Criteria

Male or female participants at least 12 years old.

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.

Additional Information

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
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Vertex Pharmaceuticals Incorporated.