Biomarkers of Injury and Destruction in the Cystic Fibrosis Lung

NCT01805713 | Completed DMC

• 2 other identifiers: 1210M22861K12HD068322
• Study Type: observational
• Target: 150
• Locations: 1 country
• Sites: 1

Brief Summary

Cystic fibrosis (CF) is the most common autosomal recessive genetic disease in Caucasians. It results in lung disease that affects quality of life and causes early death. Lung damage from CF starts in infancy and continues over time. Lung damage can negatively affect how the lung functions. It would be ideal to measure lung damage in CF patients in three instances: (1) During the first year of life after diagnosis by state newborn screening programs, (2) In children and adults over long periods of time (years), and (3) During times of illness (pulmonary exacerbation), to allow for better treatment and therapy to prevent loss of lung function. The lung is made of elastin, collagen and cartilage. When the lung is damaged by CF, these components break down into pieces that can be measured in urine, sputum and blood. These products may represent markers of lung injury. We believe that the levels of these markers will be increased over time in CF patients and even higher in patients who are sick with lung symptoms. The goal of my research is to measure the amount of lung breakdown products in urine, sputum and blood in infants, children and adults with CF during times when well and also during times of illness. I also hope to use new technologies involving the study of proteins and metabolites in samples like sputum, urine and blood to help provide new information regarding CF lung disease. These studies will help us to better treat CF lung disease.

Conditions

Cystic Fibrosis

Keywords

Biomarkers

Outcome Measures

Primary Outcomes (1)

• Biomarkes of Lung Injury

  Desmosine,Clara Cell Protein(CC10) and cathepsin B concentration in biological specimens.

  Two Years

Secondary Outcomes (2)

• Pulmonary Function

  Two Years

• Pulmonary Exacerbations

  Two years

Study Arms (2)

CF Infant Cohort

Infants with CF followed for the first two years of life.

CF Child/Adult Cohort

CF patients \> 8 years of age followed during hospitalization for pulmonary exacerbation and when well for two years.

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Infants with Cystic Fibrosis Diagnosed by Newborn Screen. Subjects with Cystic Fibrosis =\> 8 Years of Age.

You may qualify if:

• Age \< 6 months
• Cystic Fibrosis Diagnosed by Newborn Screen

You may not qualify if:

• Age over 6months
• FEV1 \< 35% predicted A
• Use of IV antibiotics for a pulmonary exacerbation or respiratory symptoms 12 months prior to enrollment.
• Hospitalization for a pulmonary exacerbation 12 months prior to enrollment.
• Use of oral antibiotic for respiratory symptoms 28 days prior to enrollment.
• Any changes in medical regimen for treatment of CF (e.g. no addition of or elimination of therapies such as hypertonic saline, inhaled corticosteroids or mucolytic therapy) within 28 days of enrollment visit.
• FEV1 \< 35% predicted A
• Use of IV antibiotics for a pulmonary exacerbation or respiratory symptoms 12 months prior to enrollment.
• Hospitalization for a pulmonary exacerbation 12 months prior to enrollment.
• Use of oral antibiotic for respiratory symptoms 28 days prior to enrollment.
• Any changes in medical regimen for treatment of CF (e.g. no addition of or elimination of therapies such as hypertonic saline, inhaled corticosteroids or mucolytic therapy) within 28 days of enrollment visit.

Sponsors & Collaborators

• University of Minnesota: lead
• National Institutes of Health (NIH): collaborator
• Minnesota Medical Foundation: collaborator
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): collaborator

Study Sites (1)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood, Urine, BALF and Sputum.

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Theresa A Laguna, MD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2013
• First Posted: March 6, 2013
• Study Start: December 1, 2012
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: January 12, 2018

Record last verified: 2018-01

Locations

US (1)