Type 1 diabetes mellitus (T1D) is a chronic autoimmune disease caused by the destruction of pancreatic β cells. T1D pathogenesis progresses through several stages: Stage 1 T1D includes the presence of β cell autoimmunity and thus presence of islet autoantibodies, without the presence of dysglycemia and symptoms. Stage 2 T1D includes the presence of islet autoantibodies and dysglycemia, also with no symptoms. Stage 3 T1D includes presence of islet autoantibodies, overt hyperglycemia, and symptoms; most patients with Stage 3 T1D meet standard diagnostic criteria for diabetes and require insulin treatment. Teplizumab has been shown to delay progression to Stage 3 in participants at Stage 2 in a Phase 2 clinical trial, leading to subsequent approval in the United States of America (USA). Patients outside of the USA are able to receive the treatment through Pre-Registration Import Licenses and Managed Access Programs. The current study will collect data on the use of teplizumab in routine care, to better understand which patients received teplizumab and how these patients were managed after they received the treatment.
Study Type
OBSERVATIONAL
Enrollment
110
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, United States
University of California, San Francisco
San Francisco, California, United States
Barbara Davis Center For Childhood Diabetes
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Miami Medical Center
Miami, Florida, United States
Participant demographic characteristics at teplizumab initiation
Age, sex at birth, race (for US participants only), ethnicity (for US participants only), height, weight, Body mass index (BMI)
Time frame: At Day 1 (first dose of teplizumab)
Participants' family history of T1D and autoimmune diseases
First- and second-degree relatives with T1D
Time frame: At Day 1 (first dose of teplizumab)
Presence of T1D susceptibility genes: Genetic risk score
Time frame: At Day 1 (first dose of teplizumab)
Presence of T1D susceptibility genes: Human Leukocyte Antigen (HLA)-haplotype
Time frame: At Day 1 (first dose of teplizumab)
Participants' medical history
Date of stage 1 confirmation, date of dysglycemia confirmation
Time frame: From 6 months prior to the first dose of teplizumab (teplizumab initiation) (or the earliest date of all data contributing to Stage 2 T1D diagnosis, whichever is earlier) up to the medical records abstraction date, approximately 3-4 years
Assessment of blood glucose test results: CGM
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Development of stages of T1D
Patient monitoring for T1D progression from early to late stages
Time frame: From the date of first assessment of dysglycemia or positive autoantibody test up to date of first dose of teplizumab (teplizumab initiation), approximately 6 months to 1 year
Assessment of blood glucose test results: HbA1c
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Assessment of blood glucose test results: Fasting Plasma Glucose (FPG)
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Assessment of blood glucose: Oral glucose tolerance test (OGTT)
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Assessment of blood glucose test results: Random Plasma Glucose (PG)
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Assessment of bloog glucose test results: Continuous glucose monitoring (CGM)
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Assessment of blood glucose test results: Post-prandial glucose (PPG)
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Assessment of autoantibody tests results
Anti-GAD65, IAA, Anti-IA-2, ICA, Anti-ZnT8
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Assessment of C-peptide test results
Time frame: At screening (6 months before teplizumab initiation), within 6 weeks prior to teplizumab initiation, during teplizumab infusion (2 weeks), following completion of teplizumab treatment (through study completion, up to 30 months)"
Treatments participants received: teplizumab treatment and other treatments
Teplizumab treatment and other treatment variables (insulin, other glucose lowering agents)
Time frame: From earliest of 6 months or the earliest date of all data contributing to Stage 2 T1D diagnosis (i.e. first record of dysglycemia and/or positive autoantibody test) and after teplizumab treatment until end of follow-up, approximately 3-4 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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