Clinical Trial: Characterization of Apolipoprotein A-I Pathways in Idiopathic Pulmonary Fibrosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Characterization of Apolipoprotein A-I Pathways in Idiopathic Pulmonary Fibrosis

Brief Summary:

Background:

- Idiopathic pulmonary fibrosis (IPF) is a chronic lung disease that becomes worse over time. There is currently no effective treatment for it. Researchers want to study the disease and learn new ways to treat it.

Objectives:

- To discover new pathways that are involved in pulmonary fibrosis. To develop new drugs that may be used to treat pulmonary fibrosis.

Eligibility:

• People at least 18 years old with IPF.
• Healthy volunteers at least 18 years old.

Design:

• Participants will be screened with medical history, questionnaire, and physical exam. They will have blood, lung, and walking tests and chest scans.
• All participants will have 1 study visit, including:
• Medical history and physical exam.
• Questions about their breathing.
• Blood tests.
• Breathing tests.
• Six-minute walk test.
• Pregnancy test.
• Chest x-ray (healthy volunteers) or chest CT scan (people with pulmonary fibrosis ).
• Small area of skin may be removed.
• Genetic tests of blood and skin samples. Participants will probably not be informed of any findings. Samples may be used to make stem cells for use in research. Participants may be contacted in the future to give co
  

  Detailed Summary: Idiopathic Pulmonary Fibrosis (IPF) is a chronic progressive disease that occurs primarily in older individuals, 55 to 75 years of age, with a median survival of approximately 3 years from time of diagnosis. At present, there are no effective treatments for patients with IPF. Levels of apolipoprotein A-I (apoA-I) have been found to be reduced in the lungs of patients with IPF, while administration of human apoA-I has been shown to reduce bleomycin-induced collagen deposition in a murine model. Here, we would like to assess whether apoA-I pathways modify lung cell biology in patients with IPF. This is a specimen procurement, clinical phenotyping and genotyping protocol that will assess whether holo-apoA-I and apolipoprotein A-I mimetic peptides, can attenuate key pathogenic manifestations of IPF, such as proliferation and extracellular matrix generation by pulmonary fibroblasts, which may serve as evidence to support future human clinical trials of apoA-I for the treatment of IPF. Furthermore, the identification of new apoA-I responsive genes and pathways that mediate fibroblast proliferation in IPF may provide insights into disease pathogenesis and identify new therapeutic targets. Lastly, if induced pluripotent stem (iPS) cells can be successfully shown to model responsiveness of lung cells to apoA-I therapy, then this approach may be expanded with the goal of providing a personalized medicine analysis that could in the future guide selection of the most effective therapy for individual patients.
  Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
  

  Current Primary Outcome: Specimen procurement, clinical phenotyping and genotyping that will assess whether holo-apoA-I and apolipoprotein A-I mimetic peptides, can attenuate key pathogenic manifestations of IPF [ Time Frame: 5 years ]
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome:
  
  

  Original Secondary Outcome:
  
  Information By: National Institutes of Health Clinical Center (CC)
  

  Dates:
  Date Received: December 11, 2014
  Date Started: November 12, 2014
  Date Completion: December 31, 2019
  Last Updated: May 17, 2017
  Last Verified: May 16, 2017