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)
试验速览
- 阶段
- 4 期
- 状态
- 进行中（未招募）
- 发起方
- Sanofi
- 入组人数
- 20
- 试验地点
- 10
- 主要终点
- Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 7 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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)
排除标准
- •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.
研究组 & 干预措施
teplizumab injection
teplizumab injection, sterile solution for intravenous use
干预措施: teplizumab (Biological)
结局指标
主要结局
Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs)
时间窗: Through 104 Weeks
Safety and tolerability endpoint
次要结局
- Anti-drug antibody (ADA) titers and presence of neutralizing antibodies (Nab)(Through 104 weeks)
- Serum concentrations of teplizumab(Sparse PK samples between Days 1 and 28)
- CD3 receptor occupancy(Days 1 and 9)