Clinical trial NCT03423862: Development of Predictive Biomarkers
Official title: Development of Predictive Biomarkers for the Rate of C-peptide Decline in Persons With Recent Onset Type 1 Diabetes
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Type1diabetes
Lead sponsor: Medical College of Wisconsin
Sponsor class: OTHER
Collaborators: name: Juvenile Diabetes Research Foundation
name: Benaroya Research Institute
First posted: 2018-02-06
Enrollment: 50
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Males and females between the ages of 6 and 17 years who were diagnosed with type 1 diabetes within the 3 months prior to their first study visit, who are being treated at Children's Hospital of Wisconsin.

Brief summary: Investigators aim to further the understanding of the various factors that govern the progression of beta-cell death in individuals recently diagnosed with Type 1 diabetes (T1D). Specifically, the investigators wish to examine the utility of plasma-induced signatures and other measures as predictive biomarkers for the rate of C-peptide decline in individuals with recent onset T1D. Persistent C-peptide in individuals with T1D reflects some degree of β-cell function and is clinically associated with a reduction in both severe hypoglycemic events and microvascular complications such as diabetic nephropathy and retinopathy. There is significant heterogeneity in the rate of C-peptide decline in individuals with T1D, reflective of the complex disease process. For example, \~10% of individuals have no discernable fall in stimulated C-peptide after two years from clinical diagnosis as compared to other individuals with very rapid C-peptide decline. It is currently impossible to predict how long, and to what extent, someone will have residual C-peptide production. This complicates clinical management but also the design and interpretation of T1D β-cell preservation trials. The "gold standard" outcome measure of any T1D β-cell preservation trial is the stimulated C-peptide to a mixed meal tolerance test (MMTT). Given the variability in this measure, intervention studies must include more subjects over a longer period of time. This slows the rate of scientific discovery and increases cost. This study aims to define the governing mechanisms of post-onset T1D disease trajectory. Understanding the trajectory of the disease may lead to the development of biomarkers to predict disease progression and therapies that could reverse or prevent the development of Type 1 diabetes.


Eligibility criteria: Inclusion Criteria:

1. Males and females 5-17 years of age with a clinical diagnosis of T1D
2. T1D diagnosis date between 1-3 months at the time of study visit 2 (baseline visit)
3. Treatment naïve of any immunomodulatory agent
4. Receiving routine out-patient diabetes care at the CHW Diabetes Clinic

Exclusion Criteria:

1. Presence of severe, active disease that requires the use of chronic medication, with the exception of well-controlled autoimmune thyroiditis/hypothyroidism or celiac disease that is well-controlled on a gluten free diet.
2. Diabetes other than T1D
3. Chronic illness known to affect glucose metabolism
4. Psychiatric impairment, with the exception of well-controlled depression or anxiety, that will affect the ability to participate in the study
5. Female participants of child-bearing age with reproductive potential must not be knowingly pregnant
6. Any condition that, in the investigator's opinion, may compromise study participation or may confound the interpretation of the study results

Primary outcomes:
measure: Predicting the honeymoon period
timeFrame: 3 years (the duration of the study)
Secondary outcomes:
measure: Establishing the relationship between baseline inflammation and other measures
timeFrame: 3 years (the duration of the study)
Other outcomes:
Locations:
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: Medical College of Wisconsin
name: Susanne Cabrera, MD
role: PRINCIPAL_INVESTIGATOR
References: