Diabetes Mellitus, Type 1
Conditions
Keywords
Type 1 diabetes, Stage 2, Pediatric type 1 diabetes, T1D, At-risk
Brief summary
The purpose of this study is to assess the safety and pharmacokinetics (PK) of teplizumab in participants with Stage 2 type 1 diabetes who are \<8 years of age.
Detailed description
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. Approximately 20 participants will be enrolled. The regimen consists of 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.
Interventions
BIOLOGICALteplizumab
CD3-directed humanized monoclonal antibody
Sponsors
Sanofi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single arm, non-randomized, open-label, multi-center study
Eligibility
Sex/Gender
ALL
Age
0 Years to 7 Years
Healthy volunteers
No
Inclusion criteria
1. Participant is a male or female 0 to \< 8 years of age, inclusive, at Day 1 2. Participant has Stage 2 T1D (two T1D-related autoantibodies and dysglycemia)
Exclusion criteria
1. Participant has any autoimmune disease other than T1D with the exception of stable thyroid or celiac disease 2. Has an active infection and/or fever 3. 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). 4. Has any condition that, in the opinion of the Investigator, would interfere with the study conduct or the safety of the participant.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs) | Through 104 Weeks | Safety and tolerability endpoint |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Serum concentrations of teplizumab | Sparse PK samples between Days 1 and 28 | Pharmacokinetic (PK) endpoint |
| Anti-drug antibody (ADA) titers and presence of neutralizing antibodies (Nab) | Through 104 weeks | Immunogenicity endpoint |
| CD3 receptor occupancy | Days 1 and 9 | To assess the effects of teplizumab on cluster of differentiation 3 (CD3) receptor occupancy |
Countries
United States
Contacts
STUDY_DIRECTORClinical Sciences & Operations
Sanofi
Outcome results
None listed