Trial record 2 of 640 for:    "Cystic Fibrosis"

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Children's Hospital of Philadelphia
Sponsor:
Information provided by (Responsible Party):
Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT02039986
First received: January 16, 2014
Last updated: January 17, 2014
Last verified: January 2014
  Purpose

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.


Condition
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 ] [ 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.


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 ] [ 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.


Estimated Enrollment: 12
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)
Groups/Cohorts
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.

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patients with a confirmed diagnosis of cystic fibrosis.

Criteria

Inclusion Criteria:

  • 6 yrs or older with cystic fibrosis
  • at least one G551D CFTR mutation or one non-G551D gating mutation for which ivacaftor is to be initiated
  • plans to start ivacaftor treatment for FDA approved indications by clinical care team or as part of an ongoing study of ivacaftor for other CFTR gating 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 illness or changes in therapy (including antibiotics) 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 ClinicalTrials.gov identifier: NCT02039986

Contacts
Contact: Devaney M Camburn, MS, CCRC 267-425-0148 camburnd@email.chop.edu
Contact: Andrea Kelly, MD, MSCE 215-590-1663 kellya@email.chop.edu

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

No publications provided

Responsible Party: Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT02039986     History of Changes
Other Study ID Numbers: 13-010465, KELLY13A0
Study First Received: January 16, 2014
Last Updated: January 17, 2014
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

Keywords provided by Children's Hospital of Philadelphia:
cystic fibrosis
CF
cystic fibrosis related diabetes
CFRD
kalydeco
ivacaftor

Additional relevant MeSH terms:
Cystic Fibrosis
Diabetes Mellitus
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on July 24, 2014