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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

Tracking Information
First Submitted Date October 15, 2018
First Posted Date October 19, 2018
Last Update Posted Date July 26, 2019
Actual Study Start Date April 4, 2019
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 19, 2018)
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.
Original Primary Outcome Measures
 (submitted: October 17, 2018)
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 rectruiment 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.
Change History
Current Secondary Outcome Measures
 (submitted: October 19, 2018)
  • 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.
  • 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.
Original Secondary Outcome Measures
 (submitted: October 17, 2018)
  • 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 rectruiment 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.
  • 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 rectruiment 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Measurement of Beta Cell Death in Individuals With Cystic Fibrosis
Official Title Measurement of Beta Cell Death in Individuals With Cystic Fibrosis
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.
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.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
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.
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.

Condition Cystic Fibrosis-related Diabetes
Intervention Diagnostic Test: Measurement of differentially methylated insulin DNA
A serum sample will be drawn to measure differentially methylated insulin DNA.
Study Groups/Cohorts
  • Cystic Fibrosis
    Serum sample will be drawn once
    Intervention: Diagnostic Test: Measurement of differentially methylated insulin DNA
  • Healthy, age-matched controls
    Serum sample will be drawn once
    Intervention: Diagnostic Test: Measurement of differentially methylated insulin DNA
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 17, 2018)
40
Original Estimated Enrollment Same as current
Estimated Study Completion Date June 2020
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers Not Provided
Contacts
Contact: Bracha Goldsweig, MD 402-955-3871 bgoldsweig@childrensomaha.org
Contact: Jill Fahner 402-559-6275 jill.fahner@unmc.edu
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03713437
Other Study ID Numbers 611-18-EP
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Bracha Goldsweig, University of Nebraska
Study Sponsor University of Nebraska
Collaborators Cystic Fibrosis Foundation
Investigators
Principal Investigator: Bracha Goldsweig, MD University of Nebraska
PRS Account University of Nebraska
Verification Date July 2019