VX-770 for the Treatment of Chronic Bronchitis

An Open Label Study to Investigate the Role of Ivacaftor (VX-770) for the Treatment of Chronic Bronchitis With CFTR Dysfunction

This research study will test how well a new drug affects bronchiectasis or chronic bronchitis. The new drug, Ivacaftor (KALYDECO), is a drug that has recently been approved by the U.S. Food and Drug Administration (FDA) for patients with a lung disease called Cystic Fibrosis (CF). It has not been approved for use in patients with bronchiectasis or chronic bronchitis.

Study Overview

• Status: Withdrawn
• Conditions: Chronic Bronchitis
• Intervention / Treatment: Drug: Ivacaftor (VX-770)

Detailed Description

The study is an open label study of orally-administered ivacaftor in subjects with chronic bronchitis and/or bronchiectasis. Subjects will be administered the study drug ivacaftor 150 mg twice daily (BID). The study drug is commercially available and will be purchased by the participant.

• Study Type: Interventional
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male or Female age 18 years
• A Clinical diagnosis of Bronchiectasis and/or Chronic Bronchitis in the investigators opinion
• Exhibit symptoms of chronic bronchitis as defined by the Medical Research Council
• FEV1 percent predicted greater than 40 percent Post Bronchodilator
• Clinically stable in the last 4 weeks with no evidence of exacerbation
• Weight of 40 kg to120 kg
• Willingness to use at least one form of acceptable birth control including abstinence, condom with spermicide, or hormonal contraceptives
• Willing to monitor blood glucose if known history of diabetes mellitus requiring insulin or medical therapy
• Element of CFTR Dysfunction, as defined by Sweat Chloride

Exclusion

• Daytime use of Oxygen Therapy
• Documented history of drug abuse within the last year
• Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease within 28 days before receiving the first dose of study drug.
• Cirrhosis or elevated liver transaminases > 3X ULN
• GFR < 50 estimated by Cockroft-Gault
• Any illness or abnormal lab finding that, in the opinion of the investigator might confound the results of the study or pose an additional risk in administering study drug to the subject.
• Pregnant or Breastfeeding
• Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit juice. (Excluded medications and foods including the drugs and foods are provided in the appendix document)
• Uncontrolled Diabetes
• Clinically significant arrhythmias or conduction abnormalities that in the opinion of the investigator that affect patient safety

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Ivacaftor (VX770); Ivacaftor (VX-770) for the Treatment of Chronic Bronchitis with CFTR Dysfunction	Drug: Ivacaftor (VX-770); of Ivacaftor (VX-770) for the Treatment of Chronic Bronchitis with CFTR Dysfunction

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
change in lung function	1 year

Collaborators and Investigators

• Sponsor: University of Alabama at Birmingham

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 1, 2020
• Primary Completion (ACTUAL): February 1, 2022
• Study Completion (ACTUAL): February 1, 2022

Study Registration Dates

• First Submitted: August 14, 2017
• First Submitted That Met QC Criteria: August 14, 2017
• First Posted (ACTUAL): August 16, 2017

Study Record Updates

• Last Update Posted (ACTUAL): February 17, 2022
• Last Update Submitted That Met QC Criteria: February 1, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Lung Diseases; Disease Attributes; Bronchial Diseases; Lung Diseases, Obstructive; Pulmonary Disease, Chronic Obstructive; Acute Disease; Bronchitis; Bronchitis, Chronic; Molecular Mechanisms of Pharmacological Action; Membrane Transport Modulators; Chloride Channel Agonists; Ivacaftor
• Other Study ID Numbers: F160914011

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No