Measurement of Beta Cell Death in Individuals With Cystic Fibrosis

Cystic Fibrosis-related Diabetes Clinical Trial

Official title:

Measurement of Beta Cell Death in Individuals With Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03713437
• Other study ID #: 0611-18-EP
• Status: Completed
• Start date: April 4, 2019
• Est. completion date: June 30, 2021

Study information

• Verified date: September 2023
• Source: University of Nebraska
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

Cystic fibrosis related diabetes (CFRD) causes increased morbidity and mortality in people with cystic fibrosis (CF). The prevalence of CFRD increases with age. While CFRD is diagnosed in only 2 percent of children under 10 year sof age, it is present in 19 percent of adolescents and up to 50 percent of adults with CF. Although CFRD is uncommon in children, recent animal and human studies have shown that milder glycemic abnormalities are common in infants and young children with CF. One of the proposed mechanisms for early glucose dysregulation in CF is related to ongoing beta cell death that may start at a very early age. The assay to be used in this study measures differentially methylated insulin DNA, released exclusively by beta cells, to determine levels of beta cell death. This assay has been shown to detect beta cell death in individuals at risk of developing type 1 diabetes. If this assay successfully detects beta cell death in individuals with CF, the investigators can identify critical time points of beta cell loss in people with CF. Understanding how and when glycemic dysregulation occurs in CF will lead to better treatment of CFRD in the future.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: June 30, 2021
• Est. primary completion date: June 30, 2021
• Gender: All
• Age group: N/A to 21 Years

Eligibility

Inclusion Criteria for Cystic Fibrosis Subjects:

• 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

Exclusion Criteria for Cystic Fibrosis Subjects:

• 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

Inclusion Criteria for healthy, age-matched controls:

• Age 0 - 21 years

Exclusion Criteria for healthy, age-matched controls:

• 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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Cystic Fibrosis-related Diabetes
• Fibrosis

Intervention

Diagnostic Test:
• Measurement of differentially methylated insulin DNA
A serum sample will be drawn to measure differentially methylated insulin DNA.

Locations

Country	Name	City	State
United States	Children's Hospital and Medical Center	Omaha	Nebraska

Sponsors (2)

Lead Sponsor	Collaborator
University of Nebraska	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Correlation between level of differentially methylated insulin DNA and oral glucose tolerance status in people with CF.	Levels of differentially methylated insulin DNA in adolescents and young adults with CF will be correlated with oral glucose tolerance status such as impaired glucose tolerance, indeterminate glucose tolerance and CFRD.	Level to be drawn once, usually within 3 months of recruitment into study.
Secondary	Correlation between level of differentially methylated insulin DNA and use of CFTR modulator therapy.	Measure differences in levels of differentially methylated insulin DNA in people with CF on CFTR modulator drugs and people with CF not on modulator therapy.	Level to be drawn once, usually within 3 months of recruitment into study.