Ivacaftor (Kalydeco) and Insulin in Cystic Fibrosis (CF)
This study is aimed at better understanding the impact of ivacaftor upon insulin and incretin secretion and glucose tolerance in patients with Cystic Fibrosis with a glycine (G551D) mutation. Investigators hypothesize that treatment with ivacaftor improves insulin secretion in individuals with CF.
Cystic Fibrosis Related Diabetes
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Effects of Ivacaftor (Kalydeco) Treatment Upon Insulin and Incretin Secretion in Patients With Cystic Fibrosis|
- Change from baseline in insulin secretion capacity at 16 weeks [ Time Frame: baseline and 16 weeks ] [ Designated as safety issue: No ]To compare insulin secretion and maximal insulin secretory capacity prior to initiation of ivacaftor and after 16 weeks of ivacaftor treatment in subjects with CF and at least one G551D CFTR mutation, or other CFTR gating mutation, and to explore the impact of ivacaftor upon incretin secretion, incretin regulation of insulin secretion, and glucose excursion during a mixed meal tolerance test in CF.
- Composite change from baseline in relationships of insulin secretion and protein and interleukin levels at 16 weeks [ Time Frame: baseline and 16 weeks ] [ Designated as safety issue: No ]To explore the composite relationships of insulin secretion, maximal insulin secretory capacity, and incretin secretion with secreted frizzled protein-4 levels and interleukin 1β levels.
|Study Start Date:||January 2014|
|Estimated Study Completion Date:||January 2017|
|Estimated Primary Completion Date:||January 2016 (Final data collection date for primary outcome measure)|
all subjects enrolled in same cohort
Cystic Fibrosis Related Diabetes (CFRD) is associated with worse nutritional status, greater pulmonary function decline, and increased mortality, highlighting its relevance in Cystic Fibrosis (CF). CFRD arises primarily from compromised insulin secretion - traditionally considered a by-product of pancreatic exocrine tissue damage and fibrosis. Recent developments in the field of diabetes are propelling a re-examination of this basic explanation. The impact of the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, upon insulin secretion and glucose regulation has not been examined, but improved glucose tolerance has been appreciated anecdotally. This study aims to understand the impact of ivacaftor therapy upon blood glucose and insulin and incretin secretion.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02039986
|Contact: Devaney M Camburn, MS, CCRCfirstname.lastname@example.org|
|Contact: Andrea Kelly, MD, MSCEemail@example.com|
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia||Recruiting|
|Philadelphia, Pennsylvania, United States, 19104|
|Contact: Devaney M Camburn, MS, CCRC 267-425-0148 firstname.lastname@example.org|
|Contact: Andrea Kelly, MD, MSCE 215-590-1663 email@example.com|
|Principal Investigator: Andrea Kelly, MD, MSCE|
|Sub-Investigator: Ronald Rubenstein, MD, PhD|
|Principal Investigator:||Andrea Kelly, MD, MSCE||Children's Hospital of Philadelphia|