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Pathway to Prevention Study

  • Study HIC#:2000021339
  • Last Updated:07/15/2021

RATIONALE

The accrual of data from the laboratory and from epidemiologic and prevention trials has improved the understanding of the etiology and pathogenesis of type 1 diabetes mellitus (T1DM). Genetic and immunologic factors play a key role in the development of T1DM, and characterization of the early metabolic abnormalities in T1DM is steadily increasing. However, information regarding the natural history of T1DM remains incomplete. The TrialNet Natural History Study of the Development of T1DM (Pathway to Prevention Study) has been designed to clarify this picture, and in so doing, will contribute to the development and implementation of studies aimed at prevention of and early treatment in T1DM.

Purpose:

TrialNet is an international network dedicated to the study, prevention, and early treatment of type 1 diabetes. TrialNet sites are located throughout the United States, Canada, Finland, United Kingdom, Italy, Germany, Sweden, Australia, and New Zealand. TrialNet is dedicated to testing new approaches to the prevention of and early intervention for type 1 diabetes.

The goal of the TrialNet Natural History Study of the Development of Type 1 Diabetes is to enhance our understanding of the demographic, immunologic, and metabolic characteristics of individuals at risk for developing type 1 diabetes.

The Natural History Study will screen relatives of people with type 1 diabetes to identify those at risk for developing the disease. Relatives of people with type 1 diabetes have about a 5% percent chance of being positive for the antibodies associated with diabetes. TrialNet will identify adults and children at risk for developing diabetes by testing for the presence of these antibodies in the blood. A positive antibody test is an early indication that damage to insulin-secreting cells may have begun. If this test is positive, additional testing will be offered to determine the likelihood that a person may develop diabetes. Individuals with antibodies will be offered the opportunity for further testing to determine their risk of developing diabetes over the next 5 years and to receive close monitoring for the development of diabetes.

  • Age1 year - 45 years
  • GenderBoth
  • Start Date09/16/2006
  • End Date07/31/2025

Trial Purpose and Description

The overall objective of this study is to perform baseline and repeat assessments over time of the metabolic and immunologic status of individuals at risk for T1D in order:

a) To characterize their risk for developing T1D and identify subjects for prevention trials,
b) To describe the pathogenic evolution of T1D, and
c) To increase the understanding of the pathogenic factors involved in the development of T1D.
Within this overall objective, the specific objectives are:
1. To determine the risk for the occurrence of T1D according to OGTTs, C-peptide levels, islet autoantibodies, HbA1c levels, markers of cell-mediated immunity, and genetic markers associated with T1D.
2. To examine the accuracy of TrialNet measures in predicting future T1D.
3. To characterize the progression of immunologic abnormalities in the development of T1D by serially studying islet autoantibodies and immune mechanistic studies.
4. To characterize the progression of metabolic decompensation in the development of T1D by serially studying insulin, C-peptide, other islet hormones, HbA1c and glucose levels, and to identify immunologic and other factors associated with this decompensation.
5. To determine the incidence of severe acute metabolic decompensation as the initial clinical presentation in individuals who have been identified as being at increased risk for T1D.
6. To identify individuals who qualify for TrialNet T1D prevention trials.
7. To accrue additional information about immunologic and metabolic factors related to the pathogenesis of T1D and validate new methods or tests that mark disease progression or response to therapy.
8. To accrue additional information about genomic markers associated with risk for the development of T1D.
9. For those participants who participated in the DPT-1 study, to examine associations of characteristics (e.g. demographics, immunologic, metabolic, etc.) assessed during the DPT-1 study with characteristics and outcomes assessed in TrialNet.

Eligibility Criteria

Inclusion Criteria:

  • Individuals 1 to 45 years old who have an immediate family member with type 1 diabetes (such as a child, parent, or sibling)
  • Individuals 1-20 years old who have an extended family member with type 1 diabetes (such as a cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)

Exclusion Criteria:

To be eligible a person must not:

  • Have diabetes already
  • Have a previous history of being treated with insulin or oral diabetes medications.
  • Currently be using systemic immunosuppressive agents (topical and inhaled agents are acceptable)
  • Have any known serious diseases

For more information about this study, contact the Help us Discover team on: