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Ivacaftor (Kalydeco) and Insulin in Cystic Fibrosis (CF)

This study is currently recruiting participants.
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Verified January 2017 by Children's Hospital of Philadelphia
Information provided by (Responsible Party):
Children's Hospital of Philadelphia Identifier:
First received: January 16, 2014
Last updated: January 19, 2017
Last verified: January 2017
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 Cystic Fibrosis

Study Type: Observational
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

Resource links provided by NLM:

Further study details as provided by Children's Hospital of Philadelphia:

Primary Outcome Measures:
  • Change from baseline in insulin secretion capacity at 16 weeks [ Time Frame: baseline and 16 weeks ]
    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.

Secondary Outcome Measures:
  • Composite change from baseline in relationships of insulin secretion and protein and interleukin levels at 16 weeks [ Time Frame: baseline and 16 weeks ]
    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.

Estimated Enrollment: 12
Actual Study Start Date: January 6, 2014
Estimated Study Completion Date: January 2019
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
all subjects
all subjects enrolled in same cohort

Detailed Description:
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.

Ages Eligible for Study:   6 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with a confirmed diagnosis of cystic fibrosis.

Inclusion Criteria:

  • 6 yrs or older with cystic fibrosis
  • at least one G551D CFTR mutation or other non-G551D gating mutation, or residual function CFTR mutation such as, but not limited to, R117H mutation, for which ivacaftor is to be initiated.
  • Plan to initiate ivacaftor treatment for FDA approved indications by clinical care team or as part of an ongoing study of ivacaftor for other CFTR mutations, including gating mutations, or residual function mutations.
  • not pregnant

Exclusion Criteria:

  • established diagnosis of non-CF related diabetes (ie., Type I diabetes)
  • history of clinically symptomatic pancreatitis in past year
  • prior lung or liver transplant
  • severe CF liver disease
  • fundoplication-related dumping syndrome
  • medical co-morbidities that are not CF-related or are unstable per the Investigator opinion
  • acute CF pulmonary exacerbation within 4 weeks prior to study procedures
  • treatment with oral or intravenous corticosteroids within 4 weeks of study
  • hemoglobin <10g/dL within 90 days of GPA test or at Screening
  • abnormal renal function within 90 days of GPA test or at Screening
  • long-standing CFRD with fasting hyperglycemia, elevated HbA1C (>8) beyond time surrounding diagnosis of CFRD, significant basal insulin requirement
  • inability to perform study specific procedures (MMTT, GPA).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02039986

Contact: Devaney M Camburn, MS, CCRC 267-425-0148
Contact: Andrea Kelly, MD, MSCE 215-590-1663

United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Devaney M Camburn, MS, CCRC    267-425-0148   
Contact: Andrea Kelly, MD, MSCE    215-590-1663   
Principal Investigator: Andrea Kelly, MD, MSCE         
Sub-Investigator: Ronald Rubenstein, MD, PhD         
Sponsors and Collaborators
Children's Hospital of Philadelphia
Principal Investigator: Andrea Kelly, MD, MSCE Children's Hospital of Philadelphia
  More Information

Responsible Party: Children's Hospital of Philadelphia Identifier: NCT02039986     History of Changes
Other Study ID Numbers: 13-010465
KELLY13A0 ( Other Grant/Funding Number: Cystic Fibrosis Foundation Therapeutics, Inc. )
Study First Received: January 16, 2014
Last Updated: January 19, 2017

Keywords provided by Children's Hospital of Philadelphia:
cystic fibrosis
cystic fibrosis related diabetes

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases processed this record on August 16, 2017