Topics

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

2015-06-03 23:57:19 | BioPortfolio

Summary

This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with CF who are homozygous for the F508del CFTR mutation.

Description

This is a Phase 3, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in people with CF who are homozygous for the F508del-CFTR mutation. This study is designed to evaluate the efficacy and safety of VX-661 in combination with ivacaftor. The active treatment regimen will be comprised of a morning dose of a fixed-dose combination tablet of 100 mg VX-661/150 mg ivacaftor once daily (qd) and an evening dose of ivacaftor 150 mg to be taken approximately 12 hours after the morning dose. The placebo regimen will be visually matched tablets to be taken with the same schedule as the active treatment.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Conditions

Cystic Fibrosis

Intervention

VX-661/ ivacaftor, ivacaftor, VX-661/ ivacaftor placebo, ivacaftor placebo

Location

Little Rock
Alaska
United States

Status

Recruiting

Source

Vertex Pharmaceuticals Incorporated

Results (where available)

View Results

Links

Published on BioPortfolio: 2015-06-03T23:57:19-0400

Clinical Trials [6956 Associated Clinical Trials listed on BioPortfolio]

Gut Imaging for Function & Transit in Cystic Fibrosis Study 2

People with Cystic Fibrosis (CF) have problems digesting their food properly. More than 8 in 10 people with CF must take medication to assist their digestion. In spite of this, complicatio...

A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

The primary purpose of study is to evaluate the treatment effect of VX-661 in combination with ivacaftor (VX-661/ivacaftor) on chest imaging endpoints using low-dose computed tomography (L...

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are

A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 in combination with ivacaftor (IVA) (VX-661/IVA) in subjects with cystic fibrosis (CF) who are ho...

Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation

PubMed Articles [2604 Associated PubMed Articles listed on BioPortfolio]

Elexacaftor/Ivacaftor/Tezacaftor: First Approval.

A fixed-dose combination tablet of the cystic fibrosis transmembrane conductance regulator (CFTR) corrector tezacaftor and the CFTR potentiator ivacaftor with the next-generation CFTR corrector elexac...

Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.

Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del ...

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved with the combination of a CF...

Tezacaftor and ivacaftor for the treatment of cystic fibrosis.

: Cystic fibrosis (CF) is a complex, multi-system, genetic disease affecting over 70,000 people worldwide. The underlying defect is a mutation in the CFTR gene. Dysfunctional CFTR protein results in a...

Elexacaftor/tezacaftor/ivacaftor (Trikafta) for cystic fibrosis.

Medical and Biotech [MESH] Definitions

An autosomal recessive genetic disease of the EXOCRINE GLANDS. It is caused by mutations in the gene encoding the CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR expressed in several organs including the LUNG, the PANCREAS, the BILIARY SYSTEM, and the SWEAT GLANDS. Cystic fibrosis is characterized by epithelial secretory dysfunction associated with ductal obstruction resulting in AIRWAY OBSTRUCTION; chronic RESPIRATORY INFECTIONS; PANCREATIC INSUFFICIENCY; maldigestion; salt depletion; and HEAT PROSTRATION.

A chloride channel that regulates secretion in many exocrine tissues. Abnormalities in the CFTR gene have been shown to cause cystic fibrosis. (Hum Genet 1994;93(4):364-8)

Misunderstanding among individuals, frequently research subjects, of scientific methods such as randomization and placebo controls.

A strain of mice widely studied as a model for cystic fibrosis. These mice are generated from embryonic stem cells in which the CFTR (cystic fibrosis transmembrane conductance regulator) gene is inactivated by gene targeting. As a result, all mice have one copy of this altered gene in all their tissues. Mice homozygous for the disrupted gene exhibit many features common to young cystic fibrosis patients, including failure to thrive, meconium ileus, and alteration of mucous and serous glands.

An effect usually, but not necessarily, beneficial that is attributable to an expectation that the regimen will have an effect, i.e., the effect is due to the power of suggestion.

More From BioPortfolio on "A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor"

Quick Search

Relevant Topics

Cystic Fibrosis
Affecting over 8,500 people in the UK, Cystic Fibrosis (CF) is one of the UK's most common life-threatening inherited diseases. Around half of the CF population can expect to live over 38 years, although improvements in treatments mean a baby born ...

BioPortfolio - life science, medical devices and pharmaceutical conference
BioPortfolio is a leading news, information and knowledge resource covering the global life science industries impacted on by biotechnology. The site aims to provide the lay person, the researcher and the management executive with a single location to so...


Searches Linking to this Trial