Measurement of Beta Cell Death in Individuals With Cystic Fibrosis

NCT03713437 | Completed

• 1 other identifier: 0611-18-EP
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

This study evaluates the feasibility of using differentially methylated insulin DNA, a biomarker of beta cell death, in determining the time course of beta cell death and development of diabetes in people with cystic fibrosis. Study participants with cystic fibrosis and healthy control participants will have a blood sample drawn in order to measure the levels of differentially methylated insulin DNA.

Conditions

Cystic Fibrosis-related Diabetes

Outcome Measures

Primary Outcomes (1)

• Levels of differentially methylated insulin DNA from infancy to early adulthood in people with cystic fibrosis

  Levels of differentially methylated insulin DNA in people with CF from infancy to young adulthood will be measured and compared to levels in healthy, age-matched controls.

  Level to be drawn once, usually within 3 months of recruitment into study.

Secondary Outcomes (2)

• Correlation between level of differentially methylated insulin DNA and oral glucose tolerance status in people with CF.

  Level to be drawn once, usually within 3 months of recruitment into study.

• Correlation between level of differentially methylated insulin DNA and use of CFTR modulator therapy.

  Level to be drawn once, usually within 3 months of recruitment into study.

Study Arms (2)

Cystic Fibrosis

Serum sample will be drawn once

Diagnostic Test: Measurement of differentially methylated insulin DNA

Healthy, age-matched controls

Serum sample will be drawn once

Diagnostic Test: Measurement of differentially methylated insulin DNA

Interventions

Measurement of differentially methylated insulin DNA DIAGNOSTIC_TEST

A serum sample will be drawn to measure differentially methylated insulin DNA.

Cystic Fibrosis; Healthy, age-matched controls

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Subjects with CF between the age of 0 and 21 years who are at their baseline health and have not started CFTR modulator therapy within the past 6 months. Healthy, age-matched controls between 0 and 21 years of age who do not have pancreatic endocrine or exocrine dysfunction.

You may qualify if:

• Age 0 - 21 years
• Diagnosis of CF by two CF-causing mutations or elevated sweat chloride test
• Normal glucose tolerance, impaired glucose tolerance, indeterminate glucose tolerance or CFRD
• Pancreatic insufficiency

You may not qualify if:

• Age \> 21 years
• Diagnosis of type 1 or type 2 diabetes
• Pregnancy
• Oral or IV steroid use in the past 2 weeks
• Pulmonary exacerbation requiring hospital admission in the past 2 weeks.
• Initiation of CFTR corrector or potentiator medication within 6 months
• Age 0 - 21 years
• Age \> 21 years
• Diagnosis of type 1 or type 2 diabetes or pre-diabetes
• Disorders impacting pancreatic exocrine function
• Pregnancy
• Oral or IV steroid use in the past 2 weeks

Sponsors & Collaborators

• University of Nebraska: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (1)

Children's Hospital and Medical Center

Omaha, Nebraska, 68114, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Participants will provide serum sample to determine levels of fructosamine, interleukin 6, glutamic acid decarboxylase, insulin antibody, zinc transporter, IA-2 antibody, and differentially methylated insulin DNA.

Study Officials

• Ashley R Deschamp, MD

  University of Nebraska

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: October 15, 2018
• First Posted: October 19, 2018
• Study Start: April 4, 2019
• Primary Completion: June 30, 2021
• Study Completion: June 30, 2021
• Last Updated: September 5, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)