Type 1 Diabetes
Conditions
Keywords
Teplizumab, Type 1 Diabetes, Real-world study, Stage 2 Type 1 Diabetes, Stage 3 Type 1 Diabetes, Disease progression, Treatment patterns, Patient monitoring
Brief summary
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.
Interventions
DRUGTeplizumab
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Sponsors
Sanofi
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patient informed consent or assent (for patients \< 18 years old) according to local regulations or appropriate informed consent waivers prior to any study related activity. * Patient received ≥ 1 day of teplizumab treatment.
Exclusion criteria
* Participation in an interventional clinical study on the index date. Participation in an interventional clinical study is defined as initiating the product/procedure or control under investigation. An interventional clinical study is a study that requires deviation from standard clinical practice by following a study protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Participant demographic characteristics at teplizumab initiation | At Day 1 (first dose of teplizumab) | Age, sex at birth, race (for US participants only), ethnicity (for US participants only), height, weight, Body mass index (BMI) |
| Participants' family history of T1D and autoimmune diseases | At Day 1 (first dose of teplizumab) | First- and second-degree relatives with T1D |
| Presence of T1D susceptibility genes: Genetic risk score | At Day 1 (first dose of teplizumab) | — |
| Presence of T1D susceptibility genes: Human Leukocyte Antigen (HLA)-haplotype | At Day 1 (first dose of teplizumab) | — |
| Participants' medical history | 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 | Date of stage 1 confirmation, date of dysglycemia confirmation |
| Assessment of blood glucose test results: CGM | 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) | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of blood glucose test results: Post-prandial glucose (PPG) | 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 | 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) | Anti-GAD65, IAA, Anti-IA-2, ICA, Anti-ZnT8 |
| Development of stages of T1D | 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 | Patient monitoring for T1D progression from early to late stages |
| Treatments participants received: teplizumab treatment and other treatments | 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 | Teplizumab treatment and other treatment variables (insulin, other glucose lowering agents) |
| Assessment of C-peptide test results | 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: HbA1c | 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) | 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) | 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) | 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) | 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) | — |
Countries
Israel, United States
Outcome results
None listed