Multilevel Models of Therapeutic Response in the Lungs

• Status: Active, not recruiting
• Trial end date: 2022-06-01
• Target enrollment: 0
• Participant gender: All

Summary

When developing new medications for lung diseases like Cystic Fibrosis (CF), scientists perform lab experiments using cells from the airways, physiology studies of how the lungs change when a drug is given, and clinical studies to determine how drugs affect overall health. The investigators of this study are seeking to develop computer models that will predict how patients will respond to drugs by just doing lab studies on cell samples from their noses. Such models would allow for medications to be developed more rapidly for all patients and allow treatments to be personalized as well. In order to develop these computer models a series of tests will be performed on patients who have CF. Tests will include sampling cells from the nose and measuring lung physiology using a combination of different imaging, breathing, and other studies performed both before and after participants take a therapy. Similar tests will be performed on people who do not have CF, and on the parents of the CF participants who carry a single CF gene because this will provide information on how specific genes might affect CF lung disease.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Tim Corcoran; University of Pittsburgh
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)
• Treatments: Edetic Acid; Pentetic Acid; Technetium Tc 99m Sulfur Colloid

Criteria

Cystic Fibrosis Subjects: Inclusion Criteria

• Ages 12 or older

• Diagnosis of cystic fibrosis as determined by sweat test or genotype

• Clinically stable as determined by a physician co-investigator

Cystic Fibrosis Subjects: Exclusion Criteria

• Smokers or users of electronic cigarettes

• FEV1%p <30% of predicted

• Nursing, pregnant or unwilling to test for pregnancy

• Intolerant to hypertonic saline

• Unable or unwilling to discontinue hypertonic saline, Pulmozyme, and long acting bronchodilators for 24 hrs before testing and short acting bronchodilators on testing days.

CF parents: Inclusion Criteria

• Ages 18 and older

• Biological parent of a CF patient who is also enrolled in the study

CF parents: Exclusion Criteria

• Smokers or users of electronic cigarettes

• FEV1%p <30% of predicted

• Nursing, pregnant or unwilling to test for pregnancy

• Unwilling to discontinue long acting bronchodilators for 24 hrs before testing and short acting bronchodilators on testing days.

• Unwilling to perform CFTR genotyping.

Healthy controls: Inclusion Criteria

• Ages 18 and older

• No history of lung disease

Healthy Controls: Exclusion Criteria

• Smokers or users of electronic cigarettes

• FEV1%p <70% of predicted

• Nursing, pregnant or unwilling to test for pregnancy

• Carriers of known disease causing CFTR mutations

• Unwilling to perform CFTR genotyping.