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Study of VX-770 in Cystic Fibrosis Subjects

2014-08-27 03:13:58 | BioPortfolio

Summary

Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The encoded protein, CFTR, is an epithelial chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. Although the disease affects multiple organs, the leading cause of mortality is the progressive loss of lung function. Obstruction of airways with thick mucous, chronic bacterial infection of the airways, and inflammatory response are all thought to play a role in causing lung damage. Through its function as a chloride channel, CFTR is believed to be integral in epithelial ion and water transport and hence, maintaining the normal hydration of lung secretions.

VX-770 is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR. Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, VX-770 has been selected for clinical development as a possible treatment for patients with CF.

The current study will enroll subjects with CF who have completed Study VX08-770-102 (Study 102) and Study VX08-770-103 (Study 103) to further evaluate the safety and efficacy of long term VX-770 treatment. Patients who were previously enrolled in Study 102 and Study 103; and have met certain criteria are eligible to enroll in this study. Study VX08-770-105 (Study 105) also offers an opportunity for subjects who received placebo in Study 102 and Study 103 to receive VX-770 treatment.

Description

This open-label, rollover study of orally administered VX-770 will be conducted in subjects with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Patients who were previously enrolled in Study 102 and Study 103; and have met certain criteria are eligible to enroll in this study. In Study 105, the treatment duration in the countries in which the study is conducted will be the sooner of approximately 96 weeks or until VX 770 is commercially available in each respective country.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Cystic Fibrosis

Intervention

VX-770

Location

Birmingham
Alabama
United States

Status

Enrolling by invitation

Source

Vertex Pharmaceuticals Incorporated

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:13:58-0400

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