Phase 2 N=93 Randomized Triple-blind Treatment

Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

Cystic Fibrosis

Bottom Line

View on ClinicalTrials.gov: NCT00865904 ↗

Enrolled (actual)

Serious AEs

11.2%

Results posted

Aug 2015

Primary outcome: Primary: Safety and Tolerability Based on Adverse Events (AEs) — 17; 16; 15; 16 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: VX-809 (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Vertex Pharmaceuticals Incorporated
Primary completion: Dec 2009

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety and Tolerability Based on Adverse Events (AEs)	17; 16; 15; 16; 18; 1	—
SECONDARY Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28	0.029; -0.049; -0.031; 0.015; -0.009	—
SECONDARY Change From Baseline in Percent Predicted FEV1 at Day 28	0.285; -1.637; -0.375; 0.168; -0.165	—
SECONDARY Change From Baseline in Forced Vital Capacity (FVC) at Day 28	0.085; -0.055; -0.020; -0.004; -0.023	—
SECONDARY Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28	-0.030; -0.011; -0.029; 0.045; -0.052	—
SECONDARY Change From Baseline in Sweat Chloride at Day 28	0.84; 0.93; -3.77; -5.29; -7.38	—
SECONDARY Change From Baseline in Nasal Potential Difference (NPD) of Zero Chloride Plus Isoproterenol Response at Day 28	-1.017; 0.832; 0.142; 1.382; 1.583	—
SECONDARY Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Domain Scores at Day 28	-1.341; -0.206; -1.626; 2.611; 0.058; 4.620	—
SECONDARY Maximum Plasma Concentration (Cmax) of VX-809	760; 1850; 2930; 6410; 1100; 2660	—
SECONDARY Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24) of VX-809	7190; 16600; 29000; 59500; 12900; 28800	—

Summary

The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Eligibility Criteria

Inclusion Criteria

Confirmed diagnosis of CF with ∆F508-CFTR mutation in both alleles
Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) of predicted normal for age, gender, and height
Weight >=40 kilograms (kg) and body mass index greater than or equal to 18.5 kilogram per square meter (kg/m^2)
Screening laboratory values, tests, and physical examination within acceptable ranges
Negative pregnancy test (for women of child-bearing potential)
Able and willing to follow contraceptive requirements
Willing to remain on a stable medication regimen for the duration of study participation

Exclusion Criteria

History of any illness, or any ongoing acute illness, that could impact the safety of the study participant or may confound results of study
Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug
Impaired hepatic or renal function
History of organ or hematological transplant

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00865904). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.