Description of Patients With Type 1 Diabetes Treated With Teplizumab (TEPLI-REAL)

A Real-World Observational Study Characterizing Patients With Type 1 Diabetes Treated With Teplizumab

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 Overview

• Status: Completed
• Conditions: Type 1 Diabetes
• Intervention / Treatment: Drug: Teplizumab

• Study Type: Observational
• Enrollment (Actual): 110

Contacts and Locations

Study Locations

• Israel
  • Petach-Tikva, Israel, 4920235
    • Schneider Children's Medical Center
  • Ramat-Gan, Israel, 52621
    • Sheba Medical Center
  • Tel Aviv, Israel, 6423906
    • Tel Aviv Sourasky Medical Center
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital Research Institute
  • California
    • San Francisco, California, United States, 94158
      • University of California, San Francisco
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Barbara Davis Center for Childhood Diabetes
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Children's National Medical Center
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami Medical Center
    • Tampa, Florida, United States, 33612
      • USF Diabetes Center
    • Vero Beach, Florida, United States, 32960
      • Doctor's Clinic
  • Georgia
    • Atlanta, Georgia, United States, 30318
      • Atlanta Diabetes Associates
    • Atlanta, Georgia, United States, 30329
      • Childrens Healthcare of Atlanta
  • Indiana
    • Carmel, Indiana, United States, 46032
      • Riley Hospital for Children
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • University of Louisville
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital Division of Endocrinology
  • New Jersey
    • Morristown, New Jersey, United States, 07960
      • Atlantic Health
  • New York
    • New York, New York, United States, 10016
      • Hassenfeld Children's Hospital at NYU Langone
    • New York, New York, United States, 10032
      • Morgan Stanley Children's Hospital
    • Staten Island, New York, United States, 10306
      • Ten's Medical PC
    • Syracuse, New York, United States, 13210
      • SUNY Upstate Medical University PARENT
  • North Dakota
    • Fargo, North Dakota, United States, 58122
      • Sanford Research/USD
  • Tennessee
    • Bartlett, Tennessee, United States, 38133
      • AM Diabetes & Endocrinology Center
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern Medical Center
    • El Paso, Texas, United States, 79902-4646
      • El Paso Medical Research Institute
  • Utah
    • Salt Lake City, Utah, United States, 84108
      • University of Utah Hospitals & Clinics
    • Sandy, Utah, United States, 84093
      • Diabetes and Endocrine Treatment Specialists

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Teplizumab treated participants; Participants who received teplizumab as part of their routine clinical care	Drug: Teplizumab; This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	At Day 1 (first dose of teplizumab)
Participants' family history of T1D and autoimmune diseases	First- and second-degree relatives with T1D	At Day 1 (first dose of teplizumab)
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	Date of stage 1 confirmation, date of dysglycemia confirmation	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		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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Development of stages of T1D	Patient monitoring for T1D progression from early to late stages	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		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)"
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	Anti-GAD65, IAA, Anti-IA-2, ICA, Anti-ZnT8	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		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)	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

Collaborators and Investigators

• Sponsor: Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): February 11, 2025
• Primary Completion (Actual): August 5, 2025
• Study Completion (Actual): August 5, 2025

Study Registration Dates

• First Submitted: January 24, 2025
• First Submitted That Met QC Criteria: March 18, 2025
• First Posted (Actual): March 24, 2025

Study Record Updates

• Last Update Posted (Estimated): August 15, 2025
• Last Update Submitted That Met QC Criteria: August 14, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: Type 1 Diabetes; Disease progression; Patient monitoring; Teplizumab; Real-world study; Treatment patterns; Stage 2 Type 1 Diabetes; Stage 3 Type 1 Diabetes
• Additional Relevant MeSH Terms: Endocrine System Diseases; Metabolic Diseases; Autoimmune Diseases; Immune System Diseases; Glucose Metabolism Disorders; Diabetes Mellitus; Diabetes Mellitus, Type 1
• Other Study ID Numbers: PDE0109; U1111-1315-4417 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No