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

Vertex Pharmaceuticals Incorporated93 enrolled

Overview

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.

This was a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Cystic Fibrosis

Interventions

VX-809DRUG

Capsules

PlaceboDRUG

Placebo matched to VX-809 capsules.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (25)

Unnamed facility

Birmingham, Alabama, United States

Unnamed facility

Palo Alto, California, United States

Outcomes

Primary Outcomes

Safety and Tolerability Based on Adverse Events (AEs)

AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.

Time frame: Up to 14 days after last dose (last dose = Day 28)

Secondary Outcomes

Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time frame: Baseline, Day 28

Change From Baseline in Percent Predicted FEV1 at Day 28

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method.

Time frame: Baseline, Day 28

Change From Baseline in Forced Vital Capacity (FVC) at Day 28

FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible.

Time frame: Baseline, Day 28

Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.