Clinical Trials

Current research studies

COPD/Chronic Bronchitis/Emphysema

Mucus Dehydration and The Evolution of COPD Lung Disease

Sponsor: National Institute of Health

Principal Investigator: Richard Boucher MD

This project, which is composed of three separate clinical trials, will test the hypothesis that a significant component of the chronic bronchitis phenotype reflects the relative dehydration of airway mucus. This in turn produces mucus adhesion to airway surfaces, infection of mucus with bacterial communities, inflammation, and airflow obstruction.

Clinical Trial 1 will look at whether or not mucus clearance is impaired in chronic bronchitis (CB) patients due to mucus dehydration. Clinical Trial 2 will analyze whether or not COPD acute exacerbations are associated with failures in mucus clearance, and if so, why? Finally, Clinical trial 3 will answer the question will airway surface hydration with hypertonic saline (HS) restore mucociliary clearance and/or cough clearance in the basal COPD state and during a COPD acute exacerbation.

We are currently recruiting for all three clinical trials. The number of study visits and compensation varies from 4 visits (up to $650 compensation) to 13 visits (up to $1400 compensation).The length of each study visit varies from 2-4 hours.

For more information please contact: Fred Fuller, RN

Cystic Fibrosis

Evaluating Regulators of Mucus Hydration Changes in Adult Lung Diseases - IRB 07-1778

Sponsor: National Institute of Health
Principal Investigator: Scott Donaldson, MD

One hypothesis for the progression of CF lung disease is that the airway surface liquid in the lungs becomes dehydrated and allows mucus adhesion to airway surfaces and causes airway obstruction. The aim of this study is to determine how hydration is regulated and how closely changes in mucus hydration and CF disease progression are associated. Patients with CF who are at least 18 years old, have an FEV1 between 30 and 60%, and have a chronic CF infection (i.e. Pseudomonas, etc) are eligible to participate in this study.

Contact: Nadia Benahmane or 919-966-9198

Evaluating Regulators of Mucus Clearance in Childhood Lung Diseases - GCRC 2706

Sponsor: National Institute of Health
Principal Investigator: Scott Donaldson, MD

The goal of this study is to investigate the extent that CF airway surface secretions become progressively dehydrated as disease severity increases as well as to measure regulators of hydration status in CF airways. Results from this study will be compared with those from adults to provide insight into the changes that occur within the lungs as CF lung disease progresses. Patients with CF who are under 4 years old and between 5-12 years old are eligible to participate.

Contact: Scott Donaldson or 919-966-9198

A Double Blind, Cross-Over Study Comparing Aerosolized Lucinactant and Vehicle on Mucociliary Clearance for CF Lung Disease - GCRC 2710

Sponsor: CF Foundation; Discovery Laboratories, Inc.; PARI GmbH
Principal Investigator: Scott Donaldson, MD

Surfactant (lucinactant) is a compound that is able to normalize the surface tension in airway lining fluids (a property that is dysregulated in CF). It has detergent properties that reduce mucus adhesiveness and enhance mucus clearance in the lungs. The compound has been used in other forms to treat respiratory distress in infants, and the goal of this study is to test its effectiveness as a CF therapy in aerosolized form. Patients with CF who are at least 14 years old and have an FEV1 of at least 40% are eligible.

Contact: Nadia Bendahmane

TIGER-1: A Multi-Center, Double-Blind, Placebo-Controlled Randomized, Efficacy and Safety Study of Denufosol Tetrasodium (INS37217) Inhalation Solution in Patients with Mild Cystic Fibrosis Lung Disease - GCRC 2509

Sponsor: Inspire Pharmaceuticals, Inc.
Principal Investigator: George Retsch-Bogart, MD

Denufosol is an investigational product that may improve the body’s ability to clear secretions out of the lungs. The aim of this study is to determine the safety and effectiveness of denufosol in patients with mild CF. Patients with CF who are at least 5 years old are eligible to participate.

Contact: Carol Barlow

Infant Study of Inhaled Saline - IRB #08-0778

Sponsor: CF Foundation
Principal Investigator: Stephanie Davis, MD

Though evidence supports the importance of early intervention in CF during infancy and early childhood, there is no evidence base to guide the use of pulmonary therapies in young children. The purpose of this study is to explore the safety and effectiveness of hypertonic saline as a therapy to enhance mucus clearance in infants and young children. The study requires 6 visits over 48 weeks and CF patients between 4 months and 5 years of age are eligible to participate.

Contact: Carol Barlow

Cholecalciferol Supplementation in Cystic Fibrosis - CTRC-2731

Sponsor: Cystic Fibrosis Foundation
Principal Investigator: Robert Aris, MD

The purpose of this study is to determine the type and dose of Vitamin D that works best to raise the level of Vitamin D in patients with Cystic Fibrosis. This study also investigates how higher levels of Vitamin D help the bone health of patients with CF.

Contact: Cheryl Boone

PTC 124: A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis IRB# 09-2030

Sponsor: PTC Therapeutics

PI: Dr. George Retsch-Bogart

Non-sense, or “stop” mutations account for approximately 10% of cystic fibrosis cases worldwide. This study tests an orally administered investigational agent to restore CFTR activity and improve clinical outcomes. Children and adults with cystic fibrosis and qualifying mutations that are over the age of 6 years, and who have an FEV₁ > 40% and <90% of predicted may qualify. The study requires 12 visits over 48 weeks.

Contact: Caroline LaFave, or 919-843-7121

“In vivo measurement of small airway mucociliary clearance” IRB # 09-2260

Sponsor: Cystic Fibrosis Foundation

PI: Dr. Scott Donaldson

Cystic fibrosis lung disease is thought to begin in the small airways, and involves impairment of mucus clearance. An assay that accurately reflects this defect, and which detects improvements in response to an effective therapy, is needed to aid drug development. This study compares existing and new mucociliary clearance protocols designed to improve our ability to characterize small airway clearance. Adults with cystic fibrosis who have an FEV₁ > 40% of predicted may qualify. The study requires 9 visits over 4-12 weeks.

Contact: Nadia Bendahmane, or 919-966-9198

“Sustained Impact of Hypertonic Saline on Mucociliary Clearance in Young Children with Cystic Fibrosis”

IRB# 09-1258

Sponsor: National Institute of Health

PI: Dr. Scott Donaldson

Previous work demonstrated that inhaled hypertonic saline (HS) reduces exacerbation frequency and improves lung function in patients with clinically apparent lung disease. It is unclear, however, whether HS will benefit young patients with mild lung disease. In this study, we will measure the effects of HS on mucociliary clearance, lung function and symptoms in patients with cystic fibrosis who are between 5-12 years of age and have an FEV₁ of at least 60% of predicted. The study requires 8 visits over 5-6 weeks.

Contact: Caroline LaFave, or 919-843-7121