Overview

This trial has been completed.

Condition cystic fibrosis
Treatments vx-809, placebo, vx-770
Phase phase 3
Sponsor Vertex Pharmaceuticals Incorporated
Start date July 2015
End date September 2016
Trial size 206 participants
Trial identifier NCT02514473, VX14-809-109

Summary

To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
vx-809 lumacaftor
vx-770 ivacaftor
(Placebo Comparator)
Matching placebo q12h
placebo

Primary Outcomes

Measure
Absolute change in lung clearance index (LCI)
time frame: From Baseline through Week 24

Secondary Outcomes

Measure
Average absolute change in sweat chloride
time frame: From Baseline at Day 15 and at Week 4
Absolute change in body mass index (BMI)
time frame: From Baseline at Week 24
Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score
time frame: From Baseline through Week 24
Absolute change in LCI5.0
time frame: From Baseline through Week 24
Absolute change in sweat chloride
time frame: From Baseline at Week 24
Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)
time frame: From Baseline through Week 24
Relative change in ppFEV1
time frame: From Baseline through Week 24
Absolute change in BMI-for-age z-score
time frame: From Baseline at Week 24
Absolute change in weight
time frame: From Baseline at Week 24
Absolute change in weight-for-age z-score
time frame: From Baseline at Week 24
Absolute change in height
time frame: From Baseline at Week 24
Absolute change in height-for-age z-score
time frame: From Baseline at Week 24
Absolute change in Treatment Satisfaction Questionnaire for Medication (TSQM) domains
time frame: From Baseline Through Week 24
Time-to-first pulmonary exacerbation
time frame: through Week 24
Event of having at least 1 pulmonary exacerbation
time frame: through Week 24
Number of pulmonary exacerbations
time frame: through Week 24
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
time frame: through Week 24
Pharmacokinetic parameters: estimated peak concentrations and trough concentrations (C3-6h and Ctrough) of lumacaftor, M28-lumacaftor, ivacaftor, M1 ivacaftor and M6 ivacaftor
time frame: through Week 24

Eligibility Criteria

Male or female participants from 6 years up to 11 years old.

Inclusion Criteria: - Subjects who weigh ≥15 kg without shoes a the Screening Visit - Subjects with confirmed diagnosis of CF at the Screening Visit. - Subjects who are homozygous for the F508del CFTR mutation - Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height - Subjects with a screening LCI2.5 result greater than or equal to 7.5 Exclusion Criteria: - History of any comorbidity reviewed at the Screening Visit 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. - Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject - Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit. - An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 - History of solid organ or hematological transplantation at the Screening Visit

Additional Information

Official title A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, 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.