TrialNet Pathway to Prevention of T1D

NCT ID: NCT00097292

Last Updated: 2025-07-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

75000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2004-02-01

Study Completion Date

2030-07-31

Brief Summary

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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.

Detailed Description

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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 and mIAA). Additional autoantibodies ICA, IA-2A, and ZnT8A will also measured in individuals positive for mIAA. ICA, IA-2A, and ZnT8A will be measured in individuals positive for GAD. 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, participants will be invited to have additional testing to determine their average risk of developing diabetes over the next 5 years. Participants that are single autoantibody positive will be re-tested annually for the development of multiple autoantibodies. Multiple autoantibody positive participants will undergo an eligibility visit which will include an Oral Glucose Tolerance Test (OGTT), re-testing for biochemical and islet cell autoantibodies if needed, and measurement of HbA1c.

Multiple autoantibody positive individuals with a normal glucose tolerance and an HbA1c \< 6.0% will be asked to come for follow-up on annual basis; multiple autoantibody positive individuals with an abnormal glucose tolerance or an HbA1c ≥ 6.0%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.

Conditions

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Diabetes Mellitus, Type 1

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Annual Re-Testing/Annual Metabolic Monitoring

Participants will be monitored annually for risk of type 1 diabetes.

No interventions assigned to this group

Semi-Annual Metabolic Monitoring

Participants will be monitored every six months for risk of type 1 diabetes

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Individuals 2 to 45 years old who have an immediate family member with type 1 diabetes (such as a child, parent, or sibling)
* Individuals 2-20 years old who have an extended family member with type 1 diabetes (such as a cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)
* Those aged 2 years to 45 years who are not family members and are known to have 1 or more islet antibodies

Exclusion Criteria

To be eligible a person must not:

* Have diabetes already
* Have previous or current use of medications for the control of hyperglycemia/diabetes.
* Currently be using immunosuppressive or immunomodulatory agents (topical and inhaled agents are acceptable)
* Have known severe active diseases, and/or diseases which are likely to limit life expectancy or lead to the use of chronic immunosuppressive or immunomodulatory therapies during the course of the study
Minimum Eligible Age

2 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role collaborator

American Diabetes Association

OTHER

Sponsor Role collaborator

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

Sponsor Role collaborator

National Center for Research Resources (NCRR)

NIH

Sponsor Role collaborator

University of South Florida

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kevan Herold, M.D.

Role: STUDY_CHAIR

Yale University

Locations

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Childrens Hospital of Orange County

Orange, California, United States

Site Status RECRUITING

University of California San Francisco

San Francisco, California, United States

Site Status RECRUITING

Stanford University Medical Center

Stanford, California, United States

Site Status RECRUITING

Barbara Davis Center for Childhood Diabetes

Denver, Colorado, United States

Site Status RECRUITING

Yale University School of Medicine

New Haven, Connecticut, United States

Site Status RECRUITING

University of Florida

Gainesville, Florida, United States

Site Status RECRUITING

Emory Children's Center

Atlanta, Georgia, United States

Site Status RECRUITING

Riley Hospital for Children, Indiana University

Indianapolis, Indiana, United States

Site Status RECRUITING

Joslin Diabetes Center

Boston, Massachusetts, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

The Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status RECRUITING

Columbia University

New York, New York, United States

Site Status RECRUITING

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Vanderbilt University

Nashville, Tennessee, United States

Site Status RECRUITING

University of Texas Medical Center at Dallas

Dallas, Texas, United States

Site Status RECRUITING

Baylor College of Medicine

Houston, Texas, United States

Site Status RECRUITING

Benaroya Research Institute

Seattle, Washington, United States

Site Status RECRUITING

Walter and Eliza Hall Institute

Parkville, Victoria, Australia

Site Status RECRUITING

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

University of Turku

Turku, , Finland

Site Status SUSPENDED

Vita-Salute San Raffaele University

Milan, , Italy

Site Status SUSPENDED

University of Bristol

Bristol, , United Kingdom

Site Status SUSPENDED

Countries

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United States Australia Canada Finland Italy United Kingdom

Central Contacts

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TrialNet Central Information Center general info

Role: CONTACT

1-800-425-8361

Facility Contacts

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Marissa Erickson

Role: primary

714-509-8613

Karen Ko

Role: primary

844-813-8273

Trudy Esrey, RD

Role: primary

650-498-4450

Brenda Bradfield

Role: primary

303-724-8595

Lori Carria

Role: primary

203-737-3595

Jennifer Hosford

Role: primary

352-294-5759

Xiaomiao Lan-Pidhainy, RN

Role: primary

404-712-0051

Maria Spall

Role: primary

866-230-8486

Nora Bryant

Role: primary

617-309-4141

Beth Pappenfus

Role: primary

612-624-2922

Cassandra McClain

Role: primary

816-960-8877

Jamie R Hyatt

Role: primary

212-851-5425

Kelli DeLallo, RN

Role: primary

412-692-5210

Faith Brendle, RN

Role: primary

615-936-8638

Lindsay Harter

Role: primary

214-648-4725

Sandra Pena

Role: primary

832-822-3380

Katie Bennett

Role: primary

206-341-8945

Felicity Healy

Role: primary

61-3-9342 7063

Rebecca Stochinsky

Role: primary

(416) 813-7654 ext. 202634

References

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Diabetes Prevention Trial--Type 1 Diabetes Study Group. Effects of insulin in relatives of patients with type 1 diabetes mellitus. N Engl J Med. 2002 May 30;346(22):1685-91. doi: 10.1056/NEJMoa012350.

Reference Type BACKGROUND
PMID: 12037147 (View on PubMed)

Gale EA, Bingley PJ, Emmett CL, Collier T; European Nicotinamide Diabetes Intervention Trial (ENDIT) Group. European Nicotinamide Diabetes Intervention Trial (ENDIT): a randomised controlled trial of intervention before the onset of type 1 diabetes. Lancet. 2004 Mar 20;363(9413):925-31. doi: 10.1016/S0140-6736(04)15786-3.

Reference Type BACKGROUND
PMID: 15043959 (View on PubMed)

Atkinson MA, Eisenbarth GS. Type 1 diabetes: new perspectives on disease pathogenesis and treatment. Lancet. 2001 Jul 21;358(9277):221-9. doi: 10.1016/S0140-6736(01)05415-0.

Reference Type BACKGROUND
PMID: 11476858 (View on PubMed)

Verge CF, Gianani R, Kawasaki E, Yu L, Pietropaolo M, Jackson RA, Chase HP, Eisenbarth GS. Prediction of type I diabetes in first-degree relatives using a combination of insulin, GAD, and ICA512bdc/IA-2 autoantibodies. Diabetes. 1996 Jul;45(7):926-33. doi: 10.2337/diab.45.7.926.

Reference Type BACKGROUND
PMID: 8666144 (View on PubMed)

Bingley PJ, Christie MR, Bonifacio E, Bonfanti R, Shattock M, Fonte MT, Bottazzo GF, Gale EA. Combined analysis of autoantibodies improves prediction of IDDM in islet cell antibody-positive relatives. Diabetes. 1994 Nov;43(11):1304-10. doi: 10.2337/diab.43.11.1304.

Reference Type BACKGROUND
PMID: 7926304 (View on PubMed)

Martinenghi S, Merolla A, Grogan P, Bianconi E, Senni E, Goncharova A, Massara F, Ragogna F, Bazzigaluppi E, Pastore MR, Bonfanti R, Bosi E. Prevention of diabetic ketoacidosis in relatives screened for islet autoantibodies and followed up in the TrialNet Pathway to Prevention study at a single institution in Italy. Diabetologia. 2025 Sep;68(9):1889-1898. doi: 10.1007/s00125-025-06461-z. Epub 2025 May 29.

Reference Type DERIVED
PMID: 40439773 (View on PubMed)

Triolo TM, Pyle L, Broncucia H, Armstrong T, Yu L, Gottlieb PA, Steck AK. Association of High-Affinity Autoantibodies With Type 1 Diabetes High-Risk HLA Haplotypes. J Clin Endocrinol Metab. 2022 Mar 24;107(4):e1510-e1517. doi: 10.1210/clinem/dgab853.

Reference Type DERIVED
PMID: 34850014 (View on PubMed)

Evans-Molina C, Sims EK, DiMeglio LA, Ismail HM, Steck AK, Palmer JP, Krischer JP, Geyer S, Xu P, Sosenko JM; Type 1 Diabetes TrialNet Study Group. beta Cell dysfunction exists more than 5 years before type 1 diabetes diagnosis. JCI Insight. 2018 Aug 9;3(15):e120877. doi: 10.1172/jci.insight.120877. eCollection 2018 Aug 9.

Reference Type DERIVED
PMID: 30089716 (View on PubMed)

Ferrara CT, Geyer SM, Evans-Molina C, Libman IM, Becker DJ, Wentworth JM, Moran A, Gitelman SE, Redondo MJ; Type 1 Diabetes TrialNet Study Group. The Role of Age and Excess Body Mass Index in Progression to Type 1 Diabetes in At-Risk Adults. J Clin Endocrinol Metab. 2017 Dec 1;102(12):4596-4603. doi: 10.1210/jc.2017-01490.

Reference Type DERIVED
PMID: 29092051 (View on PubMed)

Bosi E, Boulware DC, Becker DJ, Buckner JH, Geyer S, Gottlieb PA, Henderson C, Kinderman A, Sosenko JM, Steck AK, Bingley PJ; Type 1 Diabetes TrialNet Study Group. Impact of Age and Antibody Type on Progression From Single to Multiple Autoantibodies in Type 1 Diabetes Relatives. J Clin Endocrinol Metab. 2017 Aug 1;102(8):2881-2886. doi: 10.1210/jc.2017-00569.

Reference Type DERIVED
PMID: 28531305 (View on PubMed)

Herold KC, Usmani-Brown S, Ghazi T, Lebastchi J, Beam CA, Bellin MD, Ledizet M, Sosenko JM, Krischer JP, Palmer JP; Type 1 Diabetes TrialNet Study Group. beta cell death and dysfunction during type 1 diabetes development in at-risk individuals. J Clin Invest. 2015 Mar 2;125(3):1163-73. doi: 10.1172/JCI78142. Epub 2015 Feb 2.

Reference Type DERIVED
PMID: 25642774 (View on PubMed)

Related Links

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Other Identifiers

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UC4DK117009

Identifier Type: NIH

Identifier Source: secondary_id

View Link

UC4DK106993

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NHStudy (IND)

Identifier Type: -

Identifier Source: org_study_id

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