Real-World Study of Patients With Type 1 Diabetes Treated With Teplizumab as Part of Managed Access Programs (MAPs)

Official title

A Real-World Retrospective Observational Study Characterizing Patients With Stage 2 Type 1 Diabetes Treated With Teplizumab as Part of Managed Access Programs (MAPs)

Overview

This study is a multi-country, multi-center retrospective observational cohort study based on secondary data collected via chart review, with the aim of describing patient characteristics (including relevant comorbidities), monitoring and treatment practices related to Type 1 diabetes mellitus (T1D) progression, and time to T1D progression in participants who received teplizumab as part of Managed Access Programs (MAPs). This study design was chosen in order to gain rapid insight into the current use of teplizumab in clinical practice and the characteristics of patients who received the treatment.

Interventions

• Drug Teplizumab
  This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.

Primary outcome measures

• Participant demographics at teplizumab initiation [Time frame: At Day 1 (first dose of teplizumab)]
• Participants' family history of T1D and autoimmune diseases [Time frame: At Day 1 (first dose of teplizumab)]
• Presence of T1D susceptibility genes [Time frame: At Day 1 (first dose of teplizumab)]
• Presence of T1D susceptibility genes [Time frame: At Day 1 (first dose of teplizumab)]
• Participants' medical history [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 medical records abstraction date, approximately 3-4 years]

Secondary outcome measures (12)

• Time from Stage 1 T1D confirmation to Stage 2 confirmation [Time frame: From the date of Stage 1 T1D confirmation to date of first assessment of dysglycemia or positive autoantibody test up to date of first dose of teplizumab (teplizumab initiation), approximately 6 months to a year]
• Time from Stage 2 T1D confirmation to teplizumab initiation [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 a year]
• Assessment of blood glucose test results: Glycated hemoglobin (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 test results: 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 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 blood 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 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)]
• Assessment of autoantibody 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)]
• Proportion of participants who complete teplizumab treatment course [Time frame: At Day 1 (first dose of teplizumab) until end of follow-up, up to 24 months]
• Proportion of participants using insulin during the study period [Time frame: At Day 1 (first dose of teplizumab) until end of follow-up, up to 24 months]

Eligibility criteria

Inclusion criteria

• Patient written or electronic 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 as part of MAPs.

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.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

France · 3 centers

• Investigational Site Number: 2500001 — Bron
• Investigational Site Number: 2500003 — Paris
• Investigational Site Number: 2500002 — Paris

Spain · 3 centers

• Investigational Site Number: 7240001 — Madrid
• Investigational Site Number: 7240002 — Madrid
• Investigational Site Number: 7240003 — Zaragoza

Italy · 2 centers

• Investigational Site Number: 3800001 — Ancona
• Investigational Site Number: 3800003 — Palermo

United Kingdom · 2 centers

• Investigational Site Number: 8260001 — Birmingham
• Investigational Site Number: 8260002 — London

Belgium · 1 center

• Investigational Site Number: 0560001 — Leuven

Identifiers

NCT: NCT07457580 · OBS20675 · U111-1322-8196

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗