Clinical trial NCT04477928: General Population Level Estimation for Type 1 Diabetes Risk in Children During Routine Care Delivery
Official title: Sanford Population Level Estimation of Type 1 Diabetes Risk GEnes in Children
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Type 1 Diabetes
Celiac Disease
Keywords: Population screening
Type 1 Diabetes
Celiac Disease
Autoantibodies
Prevention
Diabetic Ketoacidosis (DKA)
Genetic Risk Score
Differential Gene Expression
Economic Modeling
Quality of Life
Feasibility
Pragmatic
T1D
Lead sponsor: Sanford Health
Sponsor class: OTHER
Collaborators: name: The Leona M. and Harry B. Helmsley Charitable Trust
name: Pacific Northwest Research Institute
name: University of Exeter
First posted: 2020-07-20
Enrollment: 33000
Sex: ALL
Minimum age (years): 0
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: yes
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children born at and/or followed at Sanford Health System facilities:

1. . 0-5 years of age;
2. . 9-16 years of age;
3. . Siblings of children known to have T1D-relevant antibodies, ages 6 to 17 years old.

Brief summary: In partnership with Helmsley Charitable Trust, the Sanford PLEDGE Study is a large-scale, observational, feasibility study of general population screening for T1D and celiac autoantibodies. Screening is incorporated into routine health care visits within an integrated health system.

Detailed description: Most children with type 1 diabetes (T1D) do not have a family member with diabetes and often are not diagnosed until the child is very sick. Research suggests that screening and identifying children at risk for T1D autoantibodies can prevent serious illness at the time of diagnosis and improve long-term health outcomes.

The investigators will screen children, ages 0-5.99 or 9-16 years for blood markers related to T1D and celiac disease during routine healthcare delivery at birth, 1, 2 and 5 years, or once between 9 and 16 years of age. Children with confirmed autoantibodies will be offered participation in other monitoring or prevention trials (T1D), or referred to clinical care (celiac).

Eligibility criteria: Inclusion Criteria:

* Newborn Entry: Viable, term infants, defined as 36 weeks gestation by either dates or ultrasound who are born to pregnant women, 18 years or older, who are willing and able to provide informed consent (IC) prior to the onset of active labor. Who are born at a Sanford Health Hospital and plan to have routine well-child care at a Sanford Clinic
* Pediatric Entry: Children less than 6 years of age who receive their routine care at a Sanford facility and whose parents are able to provide IC.
* Adolescent Entry: Children, ages 9-16 years old, who receive their routine care at a Sanford facility and whose parents are able to provide IC.
* Siblings of children known to have T1D-relevant antibodies; ages 6 to 17 years old who receive care at a Sanford clinic
* Have an active MyChart account (with proxy access).

Exclusion Criteria:

* Subject is in the opinion of the investigator, unable to comply with the requirements of the study protocol.
* Children known to have T1D

Primary outcomes:
measure: Demonstrated feasibility of large-scale population screening, as evidenced by:
timeFrame: By year 10 of the study
Secondary outcomes:
measure: Seroconversion rates for T1D-relevant and celiac autoantibodies
timeFrame: By year 10 of study
measure: Percentage of T1D seropositive subjects who enroll in another T1D monitoring or prevention study.
timeFrame: By year 10 of study
measure: Percentage of celiac seropositive subjects referred on to GI or primary care
timeFrame: By year 10 of study
measure: The percentage of celiac seropositive subjects who were evaluated in clinical setting
timeFrame: By year 10 of study
measure: The rate of development of overt hyperglycemia consistent with T1D (Stage 3).
timeFrame: By year 10 of study
measure: Proportion of participants developing overt hyperglycemia consistent with T1D (Stage 3), who present in diabetic ketoacidosis (DKA)
timeFrame: By year 10 of study
measure: Number and type of procedure-related adverse events
timeFrame: By year 10 of study
measure: Assessment of costs associated with implementation of study compared to potential impacts on cost and quality of life.
timeFrame: By year 10 of study
Other outcomes:
Locations:
city: Bemidji
country: United States
facility: Sanford Bemidji Region Clinics
state: Minnesota
city: Bismarck
country: United States
facility: Sanford Bismarck Region Clinics
state: North Dakota
city: Fargo
country: United States
facility: Sanford Fargo Region Clinics
state: North Dakota
city: Sioux Falls
country: United States
facility: Sanford Sioux Falls Region Clinics
state: South Dakota
Study officials:
affiliation: Sanford Research
name: Kurt Griffin, PhD, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sims EK, Besser REJ, Dayan C, Geno Rasmussen C, Greenbaum C, Griffin KJ, Hagopian W, Knip M, Long AE, Martin F, Mathieu C, Rewers M, Steck AK, Wentworth JM, Rich SS, Kordonouri O, Ziegler AG, Herold KC; NIDDK Type 1 Diabetes TrialNet Study Group. Screening for Type 1 Diabetes in the General Population: A Status Report and Perspective. Diabetes. 2022 Apr 1;71(4):610-623. doi: 10.2337/dbi20-0054.