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

NCT02039986 | Completed

• 2 other identifiers: 13-010465KELLY13A0
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cystic Fibrosis Related Diabetes; Cystic Fibrosis

Keywords

cystic fibrosis; CF; cystic fibrosis related diabetes; CFRD; kalydeco; ivacaftor

Outcome Measures

Primary Outcomes (1)

• Change from baseline in insulin secretion capacity at 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.

  baseline and 16 weeks

Secondary Outcomes (1)

• Composite change from baseline in relationships of insulin secretion and protein and interleukin levels at 16 weeks

  baseline and 16 weeks

Study Arms (1)

all subjects

all subjects enrolled in same cohort

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with a confirmed diagnosis of cystic fibrosis.

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Andrea Kelly, MD, MSCE

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 16, 2014
• First Posted: January 20, 2014
• Study Start: January 6, 2014
• Primary Completion: October 11, 2016
• Study Completion: October 11, 2016
• Last Updated: December 14, 2018

Record last verified: 2018-12

Locations

US (1)