Clinical Trials

Cystic FibrosisLung Transplantation | Asthma | Lung Cancer | PCD | Critical Care | Airway Biology | COPD/Chronic Bronchitis/Emphysema | Pulmonary Infections | Pulmonary HypertensionSarcoidosis | Bronchiectasis

Bronchiectasis

No current studies at this time.

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Idiopathic Bronchiectasis and Dural Ectasia: Is there a correlation? IRB Study #11-1997

Sponsor: UNC NCTRACS

PI: Katherine R. Birchard, M.D.
Co-Investigators: Mike Knowles, M.D., Leigh Anne Daniels, M.D

To determine if there is a connection between idiopathic bronchiectasis and connective tissue disorders, this study will look to see if dural ectasia is more common in patients with idiopathic bronchiectasis, as compared to patients of bronchiectasis of known causes and patients without bronchiectasis.

Contact: Katherine R. Birchard, MD

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

Gene Modifiers in Cystic Fibrosis IRB Study #00-1420

Sponsor: NIH
PI: Mike Knowles, MD

This study has been designed to compare the overall genetic make up of CF patients, in regard to gene modifiers, who are considered to have mild disease versus more severe disease. Blood samples from volunteers will be studied, along with pulmonary function tests and other medical information, in hopes that a connection can be made between genetic make-up and disease severity of CF lung disease.

Contact: cfmod@med.unc.edu

Genetic Modifiers of Inherited Liver Disease IRB Study #01-1421

Sponsor: CFF
PI: Mike Knowles, MD

This study has been designed to compare the overall genetic make up of CF patients, in regard to gene modifiers, who are considered to have mild disease versus more severe disease. Blood samples from volunteers will be studied, along with pulmonary function tests and other medical information, in hopes that a connection can be made between genetic make-up and development of CF liver disease.

Contact: cfmod@med.unc.edu

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

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

Lung Transplantation

No current studies at this time.

Asthma

Ongoing studies are conducted in the UNC Center for Environmental Medicine, Asthma and Lung Biology.

Lung Cancer

No current studies at this time.

PCD

Research Genetic Testing for Primary Ciliary Dyskinesia Using a Panel of Genes (5905), IRB Study #14-1225

Sponsor: NIH
PI: Mike Knowles, MD

Primary ciliary dyskinesia (PCD) is a Mendelian recessive, genetically heterogeneous disorder with defective mucociliary clearance (MCC), chronic oto-sino-pulmonary disease with bronchiectasis, and organ laterality defects in ~50% of patients (Kartagener Syndrome). Despite the need for early diagnosis and expert clinical care in PCD, establishing a diagnosis remains a major challenge, based on the traditional approaches of using electron microscopy and/or ciliary waveform analysis to define abnormalities of ciliary ultrastructure and/or function. The goal for this study is to determine whether a multi-gene (n=30) genetic test panel to confirm a diagnosis of PCD will identify as many as 70% of PCD patients. If this genetic test panel is successful, it will revolutionize the diagnostic approach in PCD, and lead to early identification and initiation of clinical monitoring and treatment.

Contact:

Longitudinal Study of Primary Ciliary Dyskinesia: Participants 5-18 Years of Age (5901), IRB Study # 05-2997

Sponsor: NIH
PI: Margaret Leigh, MD

Mucociliary clearance (MCC) is the primary defense mechanism for the lung. Inhaled particles (including microbial pathogens) are entrapped in mucus on the airway surface then cleared by the coordinated action of cilia. The volume and composition of airway surface liquid (ASL) influence the efficiency of ciliary function and MCC. Genetic diseases of MCC include disorders in ciliary function (primary ciliary dyskinesia, PCD), and ion transport (cystic fibrosis). The purpose of this longitudinal study protocol is to define age-related prevalence of phenotypic characteristics and the progression of key features of lung disease in subjects with Primary Ciliary Dyskinesia (PCD).

Contact:

Rare Genetic Disorders of the Airways: Cross-sectional Comparison of Clinical Features, and Development of Novel Screening and Genetic Tests (5902), IRB Study # 05-2979

Sponsor: NIH
PI: Mike Knowles, MD

Mucociliary clearance, in which mucus secretions are cleared from the breathing airways, is the primary defense mechanism for the lungs. Inhaled particles, including microbes that can cause infections, are normally entrapped in mucus on the airway surfaces and then cleared out by the coordinated action of tiny hair-like structures called cilia. Individuals with primary ciliary dyskinesia, variant cystic fibrosis, and pseudohypoaldosteronism have defective mucociliary clearance. The purpose of this study is to collect clinical and genetic information about these three airway diseases to improve current diagnostic procedures.

Contact:

CTRC-2589 Early Onset and Progression of Primary Ciliary Dyskinesia Lung Disease Prior to 10 Years of Age (5903), IRB Study # 08-0764

Sponsor: NIH
PI: Margaret Leigh, MD

Primary ciliary dyskinesia (PCD), also known as Kartagener syndrome, is a genetic disorder of the cilia, which are microscopic hair-like cells. Cilia work to keep the respiratory system clean by moving mucus that contains debris to the large airways, where it can be coughed out. People with PCD have cilia that do not move properly and therefore are not effective in cleaning the respiratory system. This study will determine when PCD starts and how it changes over time, specifically in terms of how well the lungs work, what germs grow in lung secretions, and how the lungs look on computed tomography (CT) scans.

Contact: Beth Godwin

Cross-Sectional Characterization of Idiopathic Bronchiectasis (5904), IRB Study #10-1523

Sponsor: NIH
PI: Mike Knowles, MD

Bronchiectasis is a type of lung condition in which the lungs' airways are abnormally stretched and widened. This stretching and widening makes it difficult for mucus and other substances to move out of the lungs, encouraging the growth of bacteria and leading to breathing problems or infection. Bronchiectasis can be caused by genetic disorders or diseases such as tuberculosis or rheumatoid arthritis. Researchers are interested in developing better ways to diagnose and treat a lung problem called idiopathic or unexplained bronchiectasis. The goal of this study is to better describe the physical characteristics, radiographic patterns, and airway microbiology of unexplained bronchiectasis and to look for possible genetic links or risk factors.

Contact:

Critical Care

Statins for Acutely Injured Lungs from Sepsis (SAILS)

Sponsor: National Institutes of Health, ARDS Network
Site Investigators: Shannon Carson, MD & Lydia Chang, MD

The SAILS study is a multicenter, double-blind, randomized, placebo-controlled clinical trial of rosuvastatin versus placebo comparator for the treatment of patients with ALI or ARDS from suspected sepsis. The hypothesis of this study is that rosuvastatin therapy will improve mortality in patients with sepsis-induced ALI.
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Modifying the Incidence of Neurologic Dysfunction (MIND-USA)

Sponsor: National Institutes of Health
Site Investigators: Shannon Carson, MD & Lydia Chang, MD

The MIND-USA study is a multi-center, double-blind, randomized placebo-controlled trial investigating the effects of haldoperidol and ziprasidone on delirium in at-risk critically ill patients. The hypothesis is that the administration of these commonly used medications will improve short- and long-term clinical outcomes, including days alive without acute brain dysfunction.
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Identifying Effective Strategies to Disclose Prognosis: Physician-Family Communication in Patients with Acute Lung Injury (EC-ALI)

Sponsor: National Institutes of Health
Site Investigators: Shannon Carson, MD & Lydia Chang, MD

The EC-ALI Study is a multi-center, prospective cohort study using both quantitative and qualitative methods to identify effective, acceptable strategies to communicate information about patient care and outcomes to surrogate decision makers. The research plans to determine which communication strategies help accurate understanding and which generate misconceptions about clinical care and outcomes for patients who are critically ill.
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Mortality Prediction Model for Prolonged Mechanical Ventilation (PRO-VENT)

Sponsor: UNC
Principal Investigator: Shannon Carson, MD

Patients requiring prolonged mechanical ventilation (PMV) often survive the initial severe stages of their illness, but remain dependent on life support systems. Long-term prognosis is often confusing or uncertain for patients, families, and physicians. In order to help clarify prognosis for these complicated patients, a prognostic model that identifies PMV patients who have a high risk of mortality was developed and validated in a multi-center study. Currently, the study continues to identify variables for this model to predict prognosis earlier on in a patient’s stay, as well as predict functional outcomes using both qualitative and quantitative methodology.
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Informing Decisions in Chronic Critical Illness (The SIT Study)

Sponsor: National Institutes of Health
Principal Investigator: Shannon Carson, MD

This study is a multi-center, blinded, randomized controlled trial of protocol-driven meetings in which a Supportive Information Team, made up of an interdisciplinary group of palliative care clinicians, provide informational support to families of chronically critically ill patients in order to facilitate communication and decision-making with the ICU physician. The purpose is to learn more about how best to provide support and information to families and evaluate the impact on families of patients on prolonged mechanical ventilation.
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Decision Aid for Improving Decision-making for Patients with Prolonged Mechanical Ventilation. (DST)

Sponsor: National Institutes of Health
Site Investigator: Shannon Carson, MD

This study is a multi-center, randomized, controlled trial (RCT) with 6-month follow up to determine how useful the decision aid would be in improving the quality of the decision making process for patients who are critically ill, their families, and the ICU physicians and nurses. The study plans to measure the effect of the decision aid on physician-family concordance, quality of communication and medical comprehension. The study also aims to measure the decision aid’s effect on post-traumatic stress among family members, and patients’ health care utilization over 6 months.

Airway Biology

Ongoing studies are conducted in the UNC Center for Environmental Medicine, Asthma and Lung Biology.

Pulmonary Infections

No current studies at this time.

Pulmonary Hypertension

Ambrisentan in Patients with Porto-pulmonary Hypertension Multicenter Open Label Trial

Sponsor: Tufts Medical Center
Principal Investigator: Hubert James Ford III, MD

The primary objective of this study is to evaluate the efficacy of ambrisentan, an approved drug for pulmonary hypertension, on exercise capacity and hemodynamics in a population of subjects with porto-pulmonary hypertension (POPH).

Contact:

BEAT: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Assess the Efficacy and Safety of Oral BPS 314d-MR Added-On to Treprostinil, Inhaled (Tyvaso®) in Subjects with Pulmonary Arterial Hypertension

Sponsor: Lung Biotechnology
Principal Investigator: H. James Ford III, MD

The primary objective of this study is to compare the effect of BPS-314d-MR (beraprost) versus placebo added to treprostinil, inhaled (Tyvaso®).

Contact:

 OPUS: US-based, observational, drug registry of Opsumit® (macitentan) new users in clinical practice

Principal Investigator: H. James Ford III, MD

This study is a prospective observational drug registry developed to characterize the safety profile (including primarily potential serious hepatic risks) and to describe clinical characteristics and outcomes of patients newly treated with Opsumit in the post-marketing setting.

Contact:

A Randomized, Double-blind, Placebo-Controlled, Phase II Multicenter Trial of a Monoclonal Antibody to CD20 (Rituximab) for the Treatment of Systemic Sclerosis-Associated Pulmonary Arterial Hypertension (SSc-PAH)

 Principal Investigator: H. James Ford III, MD

This study will compare patients treated with rituximab to those on placebo for change in six minute walk distance (6MWD). The secondary objectives of this study are to compare treatment groups for other measures of clinical disease progression and to determine whether the effects on clinical disease progression are paralleled by changes in selected biomarkers. Additionally, the safety and tolerability of rituximab for the treatment of SSc-PAH in patients receiving stable background medical treatment with prostanoid, endothelin receptor antagonist, PDE-5 inhibitor, and/or guanylate cyclase stimulators therapy will be assessed.

Contact:

Sarcoidosis

No current studies at this time.