Teplizumab in Pediatric Stage 2 Type 1 Diabetes

Study Overview
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Brief Summary
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The purpose of this study is to assess the safety and PK of teplizumab in participants with Stage 2 type 1 diabetes who are <8 years of age.

Detailed Description
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This is a single-arm, non-randomized, open-label, multicenter study in the U.S. to assess the safety and pharmacokinetics of teplizumab in pediatric participants with Stage 2 type 1 diabetes (T1D) (two T1D-related autoantibodies and dysglycemia) who are 0 to <8 years of age. Anti-drug antibodies/neutralizing antibodies (ADA/NAb) will also be assessed and serologic response to vaccines and exploratory T1D clinical features will be explored. Approximately 20 participants will be enrolled The regimen consists an intravenous infusion of teplizumab once daily for 14 consecutive days. The study duration for each individual may last up to approximately 26 months The safety, tolerability, PK, ADA, and T1D clinical feature data will be summarized using descriptive statistics.

Official Title
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Single Arm, Open-label Study to Assess the Safety and Pharmacokinetics of a 14-day Regimen of Teplizumab in Pediatric Stage 2 Type 1 Diabetes (Participants <8 Years of Age With at Least Two Autoantibodies and Dysglycemia)

Conditions
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Type 1 Diabetes

Intervention / Treatment
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* Biological: teplizumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant is a male or female 0 to < 8 years of age, inclusive, at Day 1 Participant has Stage 2 T1D (two T1D-related autoantibodies and dysglycemia) Exclusion Criteria: Participant has any autoimmune disease other than T1D with the exception of stable thyroid or celiac disease Has an active infection and/or fever Has a history of or serologic evidence at screening of current or past infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV). Has any condition that, in the opinion of the Investigator, would interfere with the study conduct or the safety of the participant.

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 7 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Single arm, non-randomized, open-label, multi-center study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: teplizumab injection<br>teplizumab injection, sterile solution for intravenous use | Biological: teplizumab<br>* CD3-directed humanized monoclonal antibody<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of TEAEs, AESIs, TEAEs leading to withdrawal, SAEs | Safety Endpoint | 104 Weeks | 
| Serum concentrations of teplizumab | Pharmacokinetics endpoint | 104 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Serum concentration of ADA anti-drug antibodies (anti-teplizumab antibodies in ng/mL) | Immunogenicity endpoint | 104 weeks | 
| Serum concentration of NAb anti-drug antibodies (anti-teplizumab neutralizing antibodies in ng/mL) | Immunogenticity endpoint | 104 weeks | 
| Serum concentration of CD3 receptor occupancy on T cells | Pharmacodynamic endpoint | 104 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Type 1 Diabetes, Stage 2 T1D, Pediatric T1D