26DT052; Scaling Busulfan Dose to Body Surface Area in Children
2026年8月25日 更新者：Children's Hospital of Philadelphia
Pilot Study of Scaling Busulfan Dose to Body Surface Area in Children Undergoing Hematopoietic Stem Cell Transplantation
The goal of this pilot study is to determine if scaling the first busulfan dose to Body Surface Area (BSA) in children with a BSA ≥0.5 m2 and using a BSA-banded dosing table for infants (BSA <0.5 m2) increases the fraction of patients achieving a therapeutic drug exposure after the first dose.
研究概览
地位
尚未招聘
干预/治疗
详细说明
Busulfan is a drug used in conditioning regimens for bone marrow transplantation.
Busulfan levels outside the desired range can cause excessive side effects or failure of bone marrow engraftment.
The initial dose of busulfan is currently scaled to body weight; on subsequent days the dose may be adjusted to achieve busulfan levels in a therapeutic range.
However, only half of children receiving a busulfan dose scaled to body weight achieve therapeutic blood levels after the first dose.
We performed computer simulations of alternative dosing methods for infants and children, and identified that dosing based on body surface area (BSA) could significantly increase the number of children achieving therapeutic blood levels after the first dose.
This study will test whether this new dosing method results in a higher percentage of patients achieving the desired busulfan blood levels after the first dose.
The study will enroll up to 38 children receiving high doses of busulfan as part of their standard conditioning regimen prior to bone marrow transplant.
We will use the busulfan blood levels that are routinely measured after the first dose to determine the effectiveness of our new dosing method.
研究类型
介入性
注册 (估计的)
38
阶段
- 阶段1
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Meghan Donnelly, MPH
- 电话号码：267-426-9343
- 邮箱：donnellymt@chop.edu
研究联系人备份
- 姓名：Ruchi Singh, PhD
- 邮箱：singhr3@chop.edu
学习地点
-
-
Pennsylvania
-
Philadelphia、Pennsylvania、美国、19103
- Children's Hospital of Philadelphia
-
首席研究员：
- Frank Balis, MD
-
接触：
- Meghan Donnelly, MPH
- 电话号码：267-426-9343
- 邮箱：donnellymt@chop.edu
-
-
参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 孩子
- 成人
接受健康志愿者
不
描述
Inclusion Criteria:
- Subject age: ≤21 years
Body Surface Area (BSA):
- Group A: BSA ≥0.5 m2
- Group B: BSA <0.5 m2
- Planned for once-daily busulfan-containing conditioning regimen pre-bone marrow transplant
- Scheduled to have TDM after the first dose of busulfan
- Diagnosis: both benign and malignant conditions are eligible
Exclusion Criteria:
1. At the time of enrollment, patients may not receive medications that significantly alter busulfan clearance, as specified below.
a. If patients had received the drugs listed below prior to enrollment, the following washout periods, based on ≥ 6 times drug t½, are required.
Deferasirox: ≥7 days Metronidazole: ≥7 days Ketoconazole, voriconazole: ≥7 days Itraconazole, posaconazole: ≥14 days Phenytoin: ≥21 days
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：非随机化
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Group A
Patients receiving once daily busulfan in their pre-transplant conditioning regimen will have the first busulfan dose scaled to their BSA instead of their body weight.
On day 1, patients with BSA ≥0.5 m2 will receive 100 mg/m2 Busulfan.
Subsequent doses of busulfan on days 2-4 will be guided by standard care, including therapeutic drug monitoring (TDM) to achieve an AUC within the therapeutic range.
|
Busulfan is a cell cycle non-specific alkylating agent which is approved by the Food and Drug Administration (FDA) and is commercially available.
|
|
实验性的：Group B
Patients receiving once daily busulfan in their pre-transplant conditioning regimen will have the first busulfan dose scaled to their BSA instead of their body weight.
On day 1, patients with BSA <0.5 m2 the dose will be selected from a BSA-banded dosing table.
Subsequent doses of busulfan on days 2-4 will be guided by standard care, including therapeutic drug monitoring (TDM) to achieve an AUC within the therapeutic range.
|
Busulfan is a cell cycle non-specific alkylating agent which is approved by the Food and Drug Administration (FDA) and is commercially available.
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Number of participants who achieve a therapeutic drug exposure after the first dose of Busulfan
大体时间：1 day
|
Dosing for patients in Group A (BSA >=0.5m2) will be scaled to Body Surface Area, and patients in Group B (BSA <0.5m2) will be dosed based on the infant dosing table for Day 1 dose.
The value range for therapeutic drug exposure is 36,000 μM/min (Lower Bound) - 6,000 μM/min (Upper Bound)
|
1 day
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Glutathione sample concentration over the 4-day course of busulfan administration.
大体时间：4 days
|
Plasma glutathione samples will be obtained daily to determine glutathione concentration over the 4 days busulfan is administered.
A 4-day total of gluathione sample concentration will be reported.
|
4 days
|
|
Number of participants with sinusoidal obstruction
大体时间：100 days post transplant
|
Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
|
100 days post transplant
|
|
Number of participants with engraftment failure
大体时间：100 days post transplant
|
Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
|
100 days post transplant
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年9月1日
初级完成 (估计的)
2030年9月1日
研究完成 (估计的)
2031年9月1日
研究注册日期
首次提交
2026年8月25日
首先提交符合 QC 标准的
2026年8月25日
首次发布 (实际的)
2026年8月28日
研究记录更新
最后更新发布 (实际的)
2026年8月28日
上次提交的符合 QC 标准的更新
2026年8月25日
最后验证
2026年8月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.