Pharmacokinetics of Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine) (BAT®) in Pediatric Patients With a Confirmed or Suspected Exposure to Botulinum Neurotoxin
试验速览
- 阶段
- 4 期
- 状态
- Enrolling By Invitation
- 入组人数
- 10
- 主要终点
- Margin of PK Equivalence for 90% Survival
研究概览
简要总结
The purpose of this study is to verify the pediatric dosing recommendations for BAT product in pediatric patients that are treated with BAT product due to a confirmed or suspected case of botulism. A minimum of one serum sample should be collected but whenever feasible additional serum samples (up to three per enrolled participant) may be collected from the participant or obtained from surplus standard of care samples, if available, within 32 hours after BAT product administration. Safety of the BAT product will also be evaluated. Emergent will follow-up with the physician by telephone after 30 days post-BAT product administration to collect AEs, SAEs, and unanticipated events.
详细描述
Primary Objective: To collect blood from pediatric participants to analyze the pharmacokinetics (PK) of BAT product to verify the current US FDA-approved pediatric dosing recommendations for BAT product.
Safety Objective: To evaluate the safety of BAT product in pediatric participants.
Protocol Design: This is a single arm, multi-site PK study in pediatric patients treated with BAT product.
Pharmacokinetic Parameters: The serum concentrations of BAT product obtained will be modeled using a population PK approach based on a previously developed model for BAT serotypes A through G in healthy adult human participants.
Safety Endpoints: The incidence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESI) that occur within 30 days after BAT product administration. In this study hypersensitivity/allergic reactions including serum sickness, febrile reactions, and hemodynamic instability and bradycardia, as well as reports of an infectious disease transmission will be included as AESI.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 1 Day 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Legally authorized representative is able and willing to voluntarily provide informed consent and patients to provide assent, if applicable.
- •Pediatric participants (age groups: birth to <two years, two to <six years, and six to <12 years).
- •Treatment with BAT product (initial dose only).
- •Blood sample can be collected (or standard of care sample scavenged) within 32 hours of completion of BAT product infusion.
排除标准
- •If the 5 mL blood sample volume is deemed, at the discretion of the investigator, to be unsafe based on patient weight or condition of health.
- •History of treatment with BabyBIG or other botulism antitoxin within the past 90 days.
研究组 & 干预措施
Blood sample collection
One to three 5 mL blood samples will be collected from pediatric participants treated with BAT product ideally 6-24 hours after administration, but within a maximum of 32 hours after administration.
干预措施: Blood sample collection (Biological)
结局指标
主要结局
Margin of PK Equivalence for 90% Survival
时间窗: ideally up to 32 hours post-BAT product administration
Margin of PK equivalence (MOE) for 90% survival relative to the healthy adult PK model, calculated to verify the appropriateness of administered pediatric dose. All collected samples of sufficient volume will be used provided both the BAT dosing time and sample collection time are known, even if outside the 32 hour collection window.
次要结局
- Intercompartmental Clearance [CLd](ideally up to 32 hours post-BAT product administration)
- Area Under Concentration Curve From Time 0 to Last Measurable Concentration [AUC0-t](ideally up to 32 hours post-BAT product administration)
- Area Under Concentration Curve From Time 0 to Infinity [AUC0-inf](ideally up to 32 hours post-BAT product administration)
- Maximum Serum Serotype A Concentration [Cmax](ideally up to 32 hours post-BAT product administration)
- Central Volume of Distribution [Vc](ideally up to 32 hours post-BAT product administration)
- Between Subject Variability [BSV](ideally up to 32 hours post-BAT product administration)
- Systemic Clearance [CL](ideally up to 32 hours post-BAT product administration)
- Peripheral Volume of Distribution [Vp](ideally up to 32 hours post-BAT product administration)