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Trial record 1 of 1 for:    NCT00097292
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Pathway to Prevention Study

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2016 by 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
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Identifier:
First received: November 19, 2004
Last updated: September 4, 2016
Last verified: September 2016


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.

Diabetes Mellitus, Type 1

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History Study of the Development of Type 1 Diabetes

Resource links provided by NLM:

Further study details as provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):

Primary Outcome Measures:
  • Development of type 1 diabetes [ Time Frame: Monitoring is provided once or twice annually depending on risk level ] [ Designated as safety issue: No ]
    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:
  • Metabolic and Autoantibody Assessments [ Designated as safety issue: No ]
    Oral Glucose Tolerance Test (OGTT) HbA1c Autoantibodies: ICA, IA-2A, GAD65A, mIAA, ZnT8A

Estimated Enrollment: 75000
Study Start Date: February 2004
Estimated Study Completion Date: July 2019
Estimated Primary Completion Date: July 2019 (Final data collection date for primary outcome measure)
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

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.


Ages Eligible for Study:   1 Year to 45 Years   (Child, Adult)
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: 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   
Principal Investigator: Stephen Gitelman, MD         
Stanford University Medical Center Recruiting
Stanford, California, United States, 94305-5208
Contact: Trudy Esrey, RD    650-498-4450   
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   
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   
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   
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   
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   
Principal Investigator: Henry Rodriguez, MD         
United States, Georgia
Emory Children's Center Recruiting
Atlanta, Georgia, United States, 30322
Contact: Karen Lindsley    404-727-1098   
Principal Investigator: Andrew Muir, MD         
United States, Illinois
University of Chicago Recruiting
Chicago, Illinois, United States, 60637
Contact: Gail Gannon    773-702-3853   
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   
Principal Investigator: Linda DeMeglio, MD         
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 58944
Contact: Janice Leschyshyn, RN    612-626-8467   
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   
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   
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   
Principal Investigator: Dorothy Becker, MD         
United States, Tennessee
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: Faith Brendle, RN    615-936-8638   
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   
Principal Investigator: Philip Raskin, MD         
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: David Gomez    832-825-1049   
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   
Principal Investigator: Carla Greenbaum, MD         
Australia, Victoria
Walter and Eliza Hall Institute Recruiting
Parkville, Victoria, Australia, 3050
Contact: Felicity Healy    61-3-9342 7063   
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   
Principal Investigator: Diane Wherrett, MD         
University of Turku Recruiting
Turku, Finland, FIN-20520
Contact: Annika Adamsson    +358405644674   
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   
Principal Investigator: Emanuele Bossi, MD         
United Kingdom
University of Bristol Recruiting
Bristol, United Kingdom, BS10 5NB UK
Contact: Harriet Castleden    44 117 323 8737   
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 Identifier (NCT Number):
Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Identifier: NCT00097292     History of Changes
Other Study ID Numbers: NHStudy (IND) 
Study First Received: November 19, 2004
Last Updated: September 4, 2016
Health Authority: United States: Federal Government

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 processed this record on October 21, 2016