Clinical Studies
The ENACT Study
Enabling Access to Optimal Therapy in CF
This clinical trial is examining the action and effects of several new drugs for the treatment of cystic fibrosis in children. The hope is that the ability to predict those individuals who will or will not respond to existing therapies – prior to treatment – will avoid needless risk of side effects and the high cost of a potentially ineffective treatment regimen.
Learn More About the ENACT Study
The ELECTRA Pregnancy Study
Evaluating Maternal and Neonatal Pharmacokinetics of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators
When patients with cystic fibrosis become pregnant, physicians advise them to continue CFTR Modulator medication, even though there are not adequate studies demonstrating the optimal dosing in pregnancy, how it affects the developing fetus, or composition in breastmilk. This clinical trial is a multi-center collaboration that aims to define CFTR therapeutics during pregnancy.
Learn More About the ELECTRA Pregnancy Study
Other Studies Related to CF
To find out about more CF clinical trials, visit the Cystic Fibrosis Foundation webpage.
https://www.cff.org/research-and-clinical-trials
Pharmacokinetics/Pharmacodynamics (PK/PD)
The Pulmonary Pharmacology Group is researching how CFTR modulators accumulate in cells. Although the clinical efficacy of modulators depends on their concentrations in target tissues, the pharmacokinetic properties of these drugs in epithelia are not utilized to guide patient care. With the assistance of other labs, we developed assays to determine the concentrations of modulator compounds in cells and plasma from patients on modulator therapy. Our analysis showed that cellular ivacaftor concentrations differ from plasma concentrations measured concurrently, and evidence of in vivo ivacaftor accumulation. By studying these plasma concentrations overtime we hope to determine the rate at which the drug is metabolized and with a simple blood draw to determine when the patient took their medication.
Pharmacogenomics (PGx)
The Pulmonary Pharmacology Group is researching the effects of a patient’s genetics on their ability to metabolize medications. Patients with cystic fibrosis develop multiple other conditions over their lifetime. This means that patients are on multiple medications at a time. Medications are metabolized by many enzymes in the body. The effectiveness of these enzymes is dependent on variations in gene expression ranging from no metabolism to ultra-rapid metabolizers. Since cystic fibrosis patients live with many different conditions and take so many medications it is highly likely that these patients have at least one actionable genetic variation. By researching a patients history and genotype the group hopes to determine what medications are safe for patients.