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Pathway to Prevention Study

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ClinicalTrials.gov Identifier: NCT00097292
Recruitment Status : Recruiting
First Posted : November 22, 2004
Last Update Posted : January 8, 2018
Information provided by (Responsible Party):

Study Description
Brief Summary:


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.


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.

Condition or disease
Diabetes Mellitus, Type 1

Detailed Description:

The Pathway to Prevention Study is conducted in two parts:

  • Screening
  • Monitoring (annual and semi-annual depending on risk)

In Screening , a simple blood test is done to screen for the presence of diabetes-related biochemical autoantibodies (GAD, IA-2A, mIAA). Islet cell autoantibodies (ICA) and ZnT8A are also measured in individuals positive for one or more biochemical autoantibodies. Participants can go to a TrialNet Clinical Center, Affiliate, or request a screening kit to have their blood drawn by a local physician or laboratory. Participants will be provided with their screening results within 4-6 weeks.

If autoantibodies are present initially and are confirmed by repeat testing, participants will be invited to have additional testing in baseline monitoring visit to determine their average risk of developing diabetes over the next 5 years. The baseline monitoring visit will include an Oral Glucose Tolerance Test (OGTT), re-testing for biochemical and islet cell autoantibodies if needed, measurement of HbA1c, and HLA (genetic) typing.

Individuals with less than 3% average risk will be asked to come for follow-up on annual basis; individuals with greater than average 32% risk will be asked to come for follow-up visits on semi-annual basis.

Participants will be monitored for possible progression towards type 1 diabetes and may be offered the opportunity to enter into a prevention study such (e.g., Oral Insulin prevention study) or an early treatment study if they are diagnosed with type 1 diabetes while participating in the Natural History Study.

Study Design

Study Type : Observational
Estimated Enrollment : 75000 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History Study of the Development of Type 1 Diabetes
Study Start Date : February 2004
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : July 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1
U.S. FDA Resources

Groups and Cohorts

Annual Monitoring
Participants will be monitored annually for risk of type 1 diabetes.
Semi-annual Monitoring
Participants will be monitored every six months for risk of type 1 diabetes

Outcome Measures

Primary Outcome Measures :
  1. Development of type 1 diabetes [ Time Frame: Monitoring is provided once or twice annually depending on risk level ]
    The primary outcome is the development of diabetes as defined by the American Diabetes Association (ADA) based on glucose testing, or the presence of symptoms and unequivocal hyperglycemia.

Secondary Outcome Measures :
  1. Metabolic and Autoantibody Assessments
    Oral Glucose Tolerance Test (OGTT) HbA1c Autoantibodies: ICA, IA-2A, GAD65A, mIAA, ZnT8A

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 45 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
Study Population
First and second/third degree relatives of individuals with type 1 diabetes.

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
Contacts and Locations

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): NCT00097292

Contact: TrialNet Central Information Center general info 1-800-425-8361

  Hide Study Locations
United States, California
University of California San Francisco Recruiting
San Francisco, California, United States, 94143-0434
Contact: Tracy Rodriguez    415-476-5020    tracy.rodriguez@ucsf.edu   
Principal Investigator: Stephen Gitelman, MD         
Stanford University Medical Center Recruiting
Stanford, California, United States, 94305-5208
Contact: Trudy Esrey, RD    650-498-4450    tesrey@stanford.edu   
Principal Investigator: Darrell Wilson, MD         
United States, Colorado
Barbara Davis Center for Childhood Diabetes Recruiting
Denver, Colorado, United States, 80262
Contact: Carrie Brill, RN    303-724-8272    carrie.brill@ucdenver.edu   
Principal Investigator: Andrea Steck, MD         
United States, Connecticut
Yale University School of Medicine Recruiting
New Haven, Connecticut, United States, 06519
Contact: Laurie Feldman    203-737-2760    laurie.feldman@yale.edu   
Contact    (203) 737-2511      
Principal Investigator: Kevan Herold, MD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32601-0296
Contact: Annie Abraham    877-343-2377    abraa@peds.ufl.edu   
Principal Investigator: Desmond A Schatz, MD         
University of Miami School of Medicine Recruiting
Miami, Florida, United States, 33101
Contact: Della Matheson, RN,CDE    305-243-3781    dmatheso@med.miami.edu   
Principal Investigator: Jennifer B Marks, MD         
University of South Florida Diabetes Center Recruiting
Tampa, Florida, United States, 33612
Contact: Danielle Henson, RN    813-396-9574    dhenson@health.usf.edu   
Principal Investigator: Henry Rodriguez, MD         
United States, Georgia
Emory Children's Center Recruiting
Atlanta, Georgia, United States, 30322
Contact: Karen Lindsley    404-727-1098    klindsl@emory.edu   
Principal Investigator: Andrew Muir, MD         
United States, Illinois
University of Chicago Recruiting
Chicago, Illinois, United States, 60637
Contact: Gail Gannon    773-702-3853    ggannon@medicine.bsd.uchicago.edu   
Principal Investigator: Louis Philipson, MD         
United States, Indiana
Riley Hospital for Children, Indiana University Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Maria Spall    866-230-8486    malnicho@iu.edu   
Principal Investigator: Linda DeMeglio, MD         
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 58944
Contact: Janice Leschyshyn, RN    612-626-8467    lesch004@umn.edu   
Principal Investigator: Antoinette Moran, MD         
United States, Missouri
The Children's Mercy Hospital Recruiting
Kansas City, Missouri, United States, 64111
Contact: Dani Wolfe    816-960-8942    dmwolfe@cmh.edu   
Principal Investigator: Wayne Moore, Md, PhD         
United States, New York
Columbia University Recruiting
New York, New York, United States, 10032
Contact: Sarah Pollak    212-851-5465    sjp2174@columbia.edu   
Principal Investigator: Robin S Goland, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Kelli DeLallo, RN    412-692-5210    kelli.delallo@chp.edu   
Principal Investigator: Dorothy Becker, MD         
United States, Tennessee
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: Faith Brendle, RN    615-936-8638    faith.brendle@vanderbilt.edu   
Principal Investigator: William Russell, MD         
United States, Texas
University of Texas Medical Center at Dallas Recruiting
Dallas, Texas, United States, 75390-8858
Contact: Lourdes Pruneda, RN    214-648-4717    Maria.Pruneda@utsouthwestern.edu   
Principal Investigator: Philip Raskin, MD         
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: David Gomez    832-825-1049    david.gomez@bcm.edu   
Principal Investigator: Maria Redondo, MD         
United States, Washington
Benaroya Research Institute Recruiting
Seattle, Washington, United States, 98101-2795
Contact: Marli McCulloch-Olson    206-342-6943    Marli@benaroyaresearch.org   
Principal Investigator: Carla Greenbaum, MD         
Australia, Victoria
Walter and Eliza Hall Institute Recruiting
Parkville, Victoria, Australia, 3050
Contact: Felicity Healy    61-3-9342 7063    felicity.healy@mh.org.au   
Principal Investigator: Peter C Colman, MD         
Canada, Ontario
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G-1x8
Contact: Lesley A Eisel, RN    416-813-7654 ext 1798    lesley.eisel@sickkids.ca   
Principal Investigator: Diane Wherrett, MD         
University of Turku Recruiting
Turku, Finland, FIN-20520
Contact: Annika Adamsson    +358405644674    annika.adamsson@utu.fi   
Principal Investigator: Jorma Toppari, MD         
Vita-Salute San Raffaele University Recruiting
Milan, Italy, +39-02-2643 2818
Contact: Pauline Grogan, RN    +39-02-2643 2818    grogan.pauline@hsr.it   
Principal Investigator: Emanuele Bossi, MD         
United Kingdom
University of Bristol Recruiting
Bristol, United Kingdom, BS10 5NB UK
Contact: Harriet Castleden    44 117 323 8737    harriet.castleden@bristol.ac.uk   
Principal Investigator: Polly Bingley, MD         
Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institute of Allergy and Infectious Diseases (NIAID)
National Center for Research Resources (NCRR)
Juvenile Diabetes Research Foundation
American Diabetes Association
Study Chair: Carla J Greenbaum, M.D. Benaroya Research Institute
More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier: NCT00097292     History of Changes
Other Study ID Numbers: NHStudy (IND)
UC4DK117009 ( U.S. NIH Grant/Contract )
UC4DK106993 ( U.S. NIH Grant/Contract )
First Posted: November 22, 2004    Key Record Dates
Last Update Posted: January 8, 2018
Last Verified: December 2017

Keywords provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
"at risk" for developing type 1 diabetes
juvenile diabetes
Type 1 Diabetes TrialNet

Additional relevant MeSH terms:
Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases