Clinical trial NCT06892002: Description of Patients With Type 1 Diabetes Treated With Teplizumab
Official title: A Real-World Observational Study Characterizing Patients With Type 1 Diabetes Treated With Teplizumab
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Type 1 Diabetes
Keywords: Teplizumab
Type 1 Diabetes
Real-world study
Stage 2 Type 1 Diabetes
Stage 3 Type 1 Diabetes
Disease progression
Treatment patterns
Patient monitoring
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2025-03-24
Enrollment: 100
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients whose index date (teplizumab initiation date) was ≥ 6 weeks before site activation and who meet the eligibility criteria outlined below will be eligible for enrollment into the study.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Participant demographic characteristics at teplizumab initiation
timeFrame: At Day 1 (first dose of teplizumab)
measure: Participants' family history of T1D and autoimmune diseases
timeFrame: At Day 1 (first dose of teplizumab)
measure: Presence of T1D susceptibility genes: Genetic risk score
timeFrame: At Day 1 (first dose of teplizumab)
measure: Presence of T1D susceptibility genes: Human Leukocyte Antigen (HLA)-haplotype
timeFrame: At Day 1 (first dose of teplizumab)
measure: Participants' medical history
timeFrame: 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
measure: Assessment of blood glucose test results: CGM
timeFrame: 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 outcomes:
measure: Development of stages of T1D
timeFrame: 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
measure: Assessment of blood glucose test results: HbA1c
timeFrame: 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)"
measure: Assessment of blood glucose test results: Fasting Plasma Glucose (FPG)
timeFrame: 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)"
measure: Assessment of blood glucose: Oral glucose tolerance test (OGTT)
timeFrame: 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)"
measure: Assessment of blood glucose test results: Random Plasma Glucose (PG)
timeFrame: 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)"
measure: Assessment of bloog glucose test results: Continuous glucose monitoring (CGM)
timeFrame: 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)"
measure: Assessment of blood glucose test results: Post-prandial glucose (PPG)
timeFrame: 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)"
measure: Assessment of autoantibody tests results
timeFrame: 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)"
measure: Assessment of C-peptide test results
timeFrame: 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)"
measure: Treatments participants received: teplizumab treatment and other treatments
timeFrame: 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
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Atlanta Diabetes Associates
state: Georgia
city: Staten Island
country: United States
facility: Ten's Medical PC
state: New York
city: Bartlett
country: United States
facility: AM Diabetes & Endocrinology Center
state: Tennessee
city: El Paso
country: United States
facility: El Paso Medical Research Institute
state: Texas
city: Sandy
country: United States
facility: Diabetes and Endocrine Treatment Specialists
state: Utah
Study officials:
References: