Overview

This trial is active, not recruiting.

Condition cystic fibrosis
Treatments vx-770, placebo
Phase phase 2
Sponsor Vertex Pharmaceuticals Incorporated
Collaborator Cystic Fibrosis Foundation
Start date October 2010
End date June 2013
Trial size 16 participants
Trial identifier NCT01161537, VX10-770-107

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.

Hyperpolarized noble gas magnetic resonance imaging (HG-MRI) is a promising new means of assessing lung function by direct imaging of certain non-radioactive isotopes of an inert noble gas, such as helium or xenon. Through this technique, high-resolution 3-dimensional images of lung ventilation can be obtained in both pediatric and adult patients during a single short breath-hold following inhalation of the gas.

This is a 2-part study to evaluate the effect of VX-770 on hyperpolarized 3He-MRI, and to evaluate the safety and efficacy of VX-770 in subjects aged 12 years and older with CF who have the G551D-CFTR mutation. Part A is a single-blind, placebo-controlled study that includes 4 weeks of VX-770 treatment and 4 weeks of placebo treatment. Part B is an open-label, 48 week study of long-term effect of VX 770 on hyperpolarized 3He-MRI.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking single blind (subject)
Primary purpose treatment

Primary Outcomes

Measure
Part A: Change in total ventilation defect defined by 3He-MRI
time frame: 8 weeks
Part B: Change in total ventilation defect defined by 3He-MRI
time frame: Baseline through 48 weeks

Secondary Outcomes

Measure
Part A: Safety as determined by adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), and vital signs
time frame: 8 weeks
Part A: Efficacy as measured by change in in percent predicted FEV1, Sweat Chloride, and CF Questionnaire-Revised (CFQ-R) score
time frame: 8 weeks
Part B: Safety as determined by adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), and vital signs
time frame: Baseline through 48 weeks
Part B: Efficacy as measured by change in in percent predicted FEV1, Sweat Chloride, and CF Questionnaire-Revised (CFQ-R) score
time frame: Baseline through 48 weeks

Eligibility Criteria

Male or female participants at least 12 years old.

Inclusion Criteria: - Male or female with Cystic Fibrosis - Must have the G551D-CFTR mutation on at least 1 allele - FEV1 ≥40% of predicted normal for age, gender, and height at Screening - 12 years of age or older - Must be able to swallow tablets Exclusion Criteria: - History of solid organ or hematological transplantation - Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening - Use of inhaled hypertonic saline treatment within 14 days prior to the Screening Visit - Extensive body tattoos or other physical features that will confound MRI

Additional Information

Official title A Phase 2, Single-Blind, Placebo-Controlled Study to Evaluate the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis, the G551D Mutation, and FEV1 ≥40% Predicted
Principal investigator Talissa Altes, MD
Trial information was received from ClinicalTrials.gov and was last updated in June 2013.
Information provided to ClinicalTrials.gov by Vertex Pharmaceuticals Incorporated.