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Measurement of Beta Cell Death in Individuals With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT03713437
Recruitment Status : Recruiting
First Posted : October 19, 2018
Last Update Posted : July 26, 2019
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Bracha Goldsweig, University of Nebraska

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.

Condition or disease Intervention/treatment
Cystic Fibrosis-related Diabetes Diagnostic Test: Measurement of differentially methylated insulin DNA

Detailed 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.

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Study Type : Observational
Estimated Enrollment : 40 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Measurement of Beta Cell Death in Individuals With Cystic Fibrosis
Actual Study Start Date : April 4, 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Insulin

Group/Cohort Intervention/treatment
Cystic Fibrosis
Serum sample will be drawn once
Diagnostic Test: Measurement of differentially methylated insulin DNA
A serum sample will be drawn to measure differentially methylated insulin DNA.

Healthy, age-matched controls
Serum sample will be drawn once
Diagnostic Test: Measurement of differentially methylated insulin DNA
A serum sample will be drawn to measure differentially methylated insulin DNA.




Primary Outcome Measures :
  1. Levels of differentially methylated insulin DNA from infancy to early adulthood in people with cystic fibrosis [ Time Frame: Level to be drawn once, usually within 3 months of recruitment into study. ]
    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.


Secondary Outcome Measures :
  1. Correlation between level of differentially methylated insulin DNA and oral glucose tolerance status in people with CF. [ Time Frame: Level to be drawn once, usually within 3 months of recruitment into study. ]
    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.

  2. Correlation between level of differentially methylated insulin DNA and use of CFTR modulator therapy. [ Time Frame: Level to be drawn once, usually within 3 months of recruitment into study. ]
    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.


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.


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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.

Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03713437


Contacts
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Contact: Bracha Goldsweig, MD 402-955-3871 bgoldsweig@childrensomaha.org
Contact: Jill Fahner 402-559-6275 jill.fahner@unmc.edu

Locations
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United States, Nebraska
Children's Hospital and Medical Center Recruiting
Omaha, Nebraska, United States, 68114
Contact: Bracha Goldsweig, MD    402-955-3871    bgoldsweig@childrensomaha.org   
Contact: Jill Fahner    402-599-6275    jill.fahner@unmc.edu   
Sponsors and Collaborators
University of Nebraska
Cystic Fibrosis Foundation
Investigators
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Principal Investigator: Bracha Goldsweig, MD University of Nebraska

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Responsible Party: Bracha Goldsweig, Assistant Professor, University of Nebraska
ClinicalTrials.gov Identifier: NCT03713437     History of Changes
Other Study ID Numbers: 611-18-EP
First Posted: October 19, 2018    Key Record Dates
Last Update Posted: July 26, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Insulin
Hypoglycemic Agents
Physiological Effects of Drugs