Clinical Trials

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Bronchiectasis

No current studies at this time.

COPD/Chronic Bronchitis/Emphysemaclinstud1.jpg

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

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

INVESTIGATOR INITIATED STUDIES:

F19-MRI Study of CF vs. Healthy Lungs
This study is designed to develop a technique to allow visualization of ventilation (ventilation mapping) in the lung using inhaled perfluorpropane as an inhaled MRI contrast agent. Healthy and CF subjects will be enrolled to begin to describe CF specific changes

Sponsor: NIH
PI: Dr. Jennifer Goralski
Lead Research Coordinator: Kelsey Haywood
Contact Info: Kelsey_haywood@med.unc.edu
Inclusion Criteria:
- Male or Female >18 years of age;
- Healthy volunteers and CF adults with FEV1 >30% of predicted
- Time Commitment: 1-3 Visits over 1-15 days.

F19-MRI Study: Effect of CF Exacerbation Treatment
This study will use F19-MRI to visualize ventilation in CF patients experiencing a pulmonary exacerbation, and again following treatment for that exacerbation.  Correlation to traditional markers of lung disease (e.g. spirometry) will be made.

Sponsor: NIH
PI: Dr. Jennifer Goralski
Lead Research Coordinator: Kelsey Haywood
Contact Info: Kelsey_haywood@med.unc.edu
Inclusion Criteria:
- Male or Female >18 years of age;
- CF patients experiencing a pulmonary exacerbation
- FEV1 >30%.
- Time Commitment: 1-3 visits for a total of 7 hours over a 3 week period.  

Biomarkers of Inhaled Mucolytics
This study will use N-acetylcysteine as a test mucolytic to identify biomarkers that reflect reduction of mucin molecules.  The goal is to identify biomarkers that will aid the development of novel mucolytics currently in development.

Sponsor: NIH
PI: Dr. Scott Donaldson
Lead Research Coordinator: Amy Brightwood
Contact Info: amy.brightwood@med.unc.edu
Inclusion Criteria:
- Male of Female ≥18 years of age.
- Diagnosis of CF or history of chronic bronchitis or COPD.
- Time Commitment: 1 visit, lasting approximately 3.5 hours.

Durability of Hypertonic Saline -
Inhaled hypertonic saline has been shown to have prolonged effects on MCC in CF adults after repeated dosing, and leads to improved lung function and reduced exacerbations.  Our previous studies demonstrated that HS has a very short duration of action in healthy volunteers and CF children, where clinical efficacy has not been demonstrated.  This study is designed to determine whether a single dose of HS has a prolonged duration of action in CF adults, thus explaining its clinical efficacy.  These results will help us determine whether development of short acting hydrators are adequate for CF drug development.

Sponsor: CFF
PI: Dr. Scott Donaldson
Study Progress: Closed
Inclusion Criteria:
- Male or Females >18 years of age.
- FEV1 between 50-90%.

Home Sputum Collection Study
Mucus hydrators are a key component of CF therapies.  Sputum partial osmotic pressure measurements reflect the concentration of mucin biomolecules and their degree of hydration.  Use of this measurement could be useful in studies of novel hydrator therapies.  This study is designed to assess the short and medium-term repeatability of osmotic pressure measurements in CF sputum, as well as its ability to reflect the effects of hypertonic saline.

Sponsor: NC TraCS
PI: Dr. Scott Donaldson
Lead Research Coordinator: Jianmin Han
Contact Info: jianmhan@email.unc.edu
Inclusion Criteria:
- Male of Female ≥18 years of age.
- 2 CFTR Mutations.
- FEV1 ≥40%.
- Time Commitment: A total of 2 visits over a 4 week time period.

Intestinal Current Measurement
Rectal biopsy specimens are obtained to study CFTR function in Ussing chambers and specialized cell cultures.  The effects of CFTR modulators (ex vivo and in vivo) are the focus of these studies.

Sponsor: NC TraCS
PI: Dr. Scott Donaldson
Lead Research Coordinator: Nadia Shive
Contact Info: nadia_bendahmane@med.unc.edu
Inclusion Criteria:
- Male or Female ≥ 18 years of age.
- Mutation: Any
- FEV: >30% of predicted
- Time Commitment: Approximately 2 hours.

Stationary Digital Chest Tomosynthesis
This imaging modality is being developed at UNC and is being evaluated, in comparison to CXR and CT scans, to assess its abiltiyy to detect bronchiectasis in CF and bronchiectasis subjects.
Sponsor: NC TraCS

PI: Drs. Jennifer Goralski and Yueh Lee
Lead Research Coordinator: Michele Vickers          
Contact Info: Michele_vickers@med.unc.edu

Inclusion Criteria:
-     Adult Population:
      - Male or Female 18≥ years of age.
      - Diagnosis of mild to moderate CF as defined by a FEV1 >60% and 30-60%,
        respectively (CXR) or a diagnosis of airways disease (CT). 
      - BMI <35
      - Undergone a CXR (or CT) in the past 7 days.
      - Time Commitment: 1-3 Visits over a 3-6 month time period.
-     Pediatric Population:
      - Male or Female 5-17 years of age.
      - Diagnosis of mild to moderate CF as defined by a FEV1 >60% and 30-60%,
        respectively (CXR) or a diagnosis of airways disease (CT).
      - BMI <35
      - Undergone a CXR (or CT) in the past 7 days.
      - Time Commitment: 1-3 Visits over a 3-6 month time period.

 

MULTICENTER CLINICAL TRIALS

Vertex 661 and Ivacaftor (VX-661-108) in people with cystic fibrosis ages 12 and older with one copy of the F508del-CFTR mutation and a second CFTR mutation predicted to have residual function
This trial will look at the safety and effectiveness of the drug VX-661 in combination with ivacaftor.

Study Drugs:
VX-661/Ivacaftor
Type of Therapy: CFTR Modulation
Sponsor
: Vertex Pharmaceuticals
PI: Dr. Scott Donaldson
Lead Research Coordinator: Rose Cunnion
Contact Info: rcunnion@email.unc.edu
Inclusion Criteria:
- Males and Females ≥12 years of age.
- 1 F508del-CFTR mutation and 1 CFTR mutation predicted to have residual function.
- FEV1 ≥ 40% and ≤ 90%.
- Time Commitment: A total of 11 study visits over a 32 week time period.

Vertex 661 and ivacaftor (VX-661-111) in adults with cystic fibrosis and two copies of the delta F508 mutation
This trial will look at the effectiveness of the drug VX-661 in combination with ivacaftor based on several health outcome measures.

Study Drugs:
VX-661/VX-770
Type of Therapy: CFTR Modulation
Sponsor
:Vertex Pharmaceuticals
PI: Dr. Scott Donaldson (National PI)
Lead Research Coordinator: Nadia Shive
Contact Info: nadia_bendahmane@med.unc.edu
Inclusion Criteria:
Subjects who meet all of the following inclusion criteria will be eligible.
- Male and female ≥18 years of age.
- 2 F508del-CFTR mutations.
- FEV1 ≥40% and ≤90%.
- Time Commitment: A total of 10 contacts including clinic visits, phone calls, and study follow-
  up over a 3 month time period.

VX-661-110
This is a long-term, open label extension study of VX661 and ivacaftor in patients enrolled in feeder studies (e.g. VX-661-111)

Sponsor
:Vertex Pharmaceuticals
PI: Dr. Scott Donaldson
Lead Research Coordinator: Amy Brightwood
Contact Info: amy.brightwood@med.unc.edu 
Inclusion Criteria:
- Male or Female ≥12 years of age.
- Heterozygous or Homozygous for F508 CFTR Mutation.
- Completed study drug treatment during the Treatment Period in a parent study.
- Not pregnant or nursing
- Willing to remain on a stable CF medication regimen.
- Time Commitment: 12-13 visits over a 2 year time period.

GOAL-e2: G551D Observational Study of Ivacaftor
This trial examines the open label, post-approval effects of ivacaftor on multiple biomarkers that may reflect CFTR activity in patients with gating mutations.

Sponsor
: CFF
PI: Dr. Scott Donaldson
Lead Research Coordinator: Nadia Shive
Contact Info: nadia_bendahmane@med.unc.edu
Inclusion Criteria:
- Male or female ≥ 6 years of age.
- R117H mutation or Non-G551D mutation.
- Time Commitment: Up to approximately 6 years.   

Phase 2 safety study of N91115 in adults with CF with two copies of the F508del-CFTR gene mutation who are currently taking ivacaftor/lumicaftor (Orkambi)
This trial will test the safety and effectiveness of a new type of CFTR modulator drug called N91115 in adults with cystic fibrosis who have two copies of the F508del-CFTR gene mutation and are currently taking Orkambi.

Study Drugs:
N91115
Type of Therapy: CFTR Modulation
Sponsor
: Nivalis Pharmacueticals
PI: Dr. Scott Donaldson
Lead Research Coordinator: Nadia Shive
Contact Info: nadia_bendahmane@med.unc.edu
Study Progress: Closed
Inclusion Criteria:
- Male or Female ≥18 years of age.
- Diagnosis of mild-to-moderate cystic fibrosis or bronchiectasis.

PROSPECT Study
This is a prospective, open label, post-approval study of Orkambi in patients with two copies of F508del mutations.  Multiple outcome measures will be evaluated, including mucociliary clearance, exhaled NO, LCI (lung clearance index via multiple breath N2 washout).

Sponsor
: CFF
PI: Dr. Scott Donaldson
Lead Research Coordinator: Nadia Shive
Contact Info: nadia_bendahmane@med.unc.edu
Inclusion Criteria:
- Male or female ≥ 12 years of age.
- Two F508del CFTR mutations.
- Time Commitment: 10 contacts including clinic visits, phone calls, emails, etc. up to a total of 
  12 months overall.

To search for additional Clinical Trials that you may be eligible for please go to the Cystic Fibrosis Foundation's Clinical Trials Finder.

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

BRAVE: Burden of Respiratory Viruses in HIV Infection
Sponsor: National Institutes of Health, Center for AIDS Research
Principal Investigator: William Fischer, MD

 Most infectious respiratory diseases in HIV-infected patients have been attributed to bacterial and fungal infections but little is known about the role respiratory viruses play in HIV infected patients. This study aims to collect excess respiratory samples and clinical information from HIV infected inpatients and outpatients with respiratory symptoms to better understand which viruses affect the lung health of HIV patients, whether HIV disease is a risk factor for severe outcomes in viral illness, and identify novel respiratory viral pathogens missed by current diagnostic standards.

 HIRE: The Effect of HIV on the Immune Response to Viral Infection in Human Nasal Epithelial Cells
Principal Investigator: William Fischer, MD

 Emerging evidence suggests that viral pathogens play a significant role in the lung health of HIV infected patients who have both increased susceptibility to and severity of disease despite treatment with anti-retroviral therapy. The goal of this study is to understand how HIV infection affects mucosal inflammation at the level of respiratory epithelial cells, which are the primary target of respiratory viruses such as influenza.  

 International work

EVD- 001 Evaluation of convalescent plasma in the treatment of Ebola virus disease in Liberia.
Sponsor:  Clinical RM
Funding:  Bill and Melinda Gates Foundation

This was the first clinical trial of a novel therapeutic agent in an Ebola outbreak.  This trial has ended as there is no active transmission of Ebola virus in West Africa currently.

EVD-002 Collection of Ebola Virus Disease Convalescent Plasma and Longitudinal Clinical and Serosurvey of EVD Survivors
Sponsor:  Clinical RM
Funding:  Bill and Melinda Gates Foundation

 The goal of this project is to establish a longitudinal observational cohort of EVD survivors to prequalify potential ECP donors for ECP donation, collect ECP by apheresis for clinical trials, compassionate use, immune globulin production or other EVD research, and better understand the clinical, viral, and immunologic sequelae of Ebola virus disease.

PREVAIL IV – Double-blind, Randomized, Two-phase, Placebo-controlled, Phase II Trial of GS-5734 to Assess the Antiviral Activity, Longer-term Clearance of Ebola virus, and Safety in Male Ebola Survivors with Evidence of Ebola virus persistence in semen.
Sponsor:  National Institutes of Health
Principal Investigators:  William Fischer, Elizabeth Higgs, and Dehkontee Dennis

Following the end of widespread active transmission at least 12 clusters of infections emerged due to the persistence of Ebola virus in survivors of Ebola virus disease.  This trial evaluates the efficacy of a novel compound, GS-5734, in reducing shedding of Ebola virus in the semen of EVD survivors.

Pulmonary Hypertension

A Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Subjects with Pulmonary Hypertension due to Parenchymal Lung Disease

Sponsor: United Therapeutics Corporation
Principal Investigator: H. James Ford III, MD

The primary of objective of the study is  To evaluate the safety and efficacy of inhaled treprostinil in subjects with PH associated with ILD including CPFE.

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:

LIBERTY: A Phase 2, Randomized, Double-BLInd, Placebo-Controlled Study of UBEnimex in Patients with Pulmonary ARTerial HYpertension (WHO Group 1)

Sponsor: Eiger Pharmaceuticals, Inc.
Principal Investigator: H. James Ford III, MD

The primary objectives of this study are to evaluate the efficacy, safety, and tolerability of ubenimex in patients with PAH (WHO Group 1).

Contact: &

Pulmonary Hypertension Association Registry

Sponsor: Pulmonary Hypertension Association
Principal Investigator

The goals of the PHAR include 1) measuring and improving quality of care (including assessing differences in adherence to evidence-based guidelines and establishing benchmarks for health outcomes), 2) determining the clinical effectiveness, comparative effectiveness, and cost effectiveness of treatment approaches, 3) understanding risk factors for outcomes and regional/center differences, and 4) facilitating funded clinical trials of new therapies and collaboration with the PAH community at large, including providers, patients, and their caregivers.

Contact: &

Sarcoidosis

No current studies at this time.