Precision Dosing of Alemtuzumab
Precision Dosing of Alemtuzumab for Allogeneic Hematopoietic Cell Transplantation in Non-Malignant Diseases
研究概览
详细说明
The hypothesis is that the model-informed dosing regimen will prospectively allow the precision dosing of alemtuzumab to target Day 0 levels to fall between 0.15-0.6ug/mL in greater than 80% of patients. The investigators have chosen a conservative pilot study approach, and the aim is to achieve Day 0 alemtuzumab levels between 0.15-0.6ug/mL in greater than 60% of 20 patients enrolled in this pilot study based on a Simon two-stage design as detailed below.
A Simon two-stage design (Simon, 1989) is being used. The null hypothesis that 30% of patients will achieve a Day 0 alemtuzumab level between 0.15-0.6ug/mL will be tested against a one-sided alternative that the alemtuzumab dose modification will result in 60% of patients achieving a Day 0 alemtuzumab level between 0.15-0.6ug/mL. In the first stage, 7 patients will be accrued. If there are 2 or fewer patients that achieve Day 0 alemtuzumab levels within the range of 0.15-0.6ug/mL, the study will be stopped. Otherwise, 13 additional patients will be accrued for a total of 20. The null hypothesis will be rejected if 11 or more patients achieve a Day 0 level of 0.15-0.6ug/mL within the total 20 patients. This design yields a type I error rate of 0.04 and power of 0.82 when the true response rate is 0.60. The investigators will enroll up to 30 patients in order allow for subject withdrawal, but will stop enrollment once 20 patients reach Day 0.
研究类型
注册 (实际的)
阶段
- 不适用
联系人和位置
学习地点
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Ohio
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Cincinnati、Ohio、美国、45229
- Cincinnati Children's Hospital Medical Center
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- Patients who are undergoing RIC HCT with alemtuzumab, fludarabine, and melphalan at CCHMC for treatment of a non-malignant disease.
- Age ≥ 6 weeks to ≤ 30 years (at time of enrollment).
- For the first 7 patients, patients must have a 10/10 HLA matched related or unrelated stem cell donor, or be receiving a CD34+ selected stem cell product. After the first 7 patients, any donor match may be allowed after data review by the PI and medical monitor.
Exclusion Criteria:
- Patients with a history of anaphylaxis to alemtuzumab.
- Patients who have previously received alemtuzumab and have not cleared alemtuzumab prior to the start of the preparative regimen.
- Life expectancy less than 2 weeks.
- Patients receiving dialysis.
- Failure to sign informed consent and/or assent, or inability to undergo informed consent process.
- It is not medically advisable to obtain the specimens necessary for this study.
- Not able to tolerate subcutaneous dosing (patients with severe skin conditions such as epidermolysis bullosa).
学习计划
研究是如何设计的？
设计细节
- 主要用途：其他
- 分配：不适用
- 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 |
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其他：Alemtuzumab
Patients less than 15kg will be given 0.6mg/kg alemtuzumab divided over days -14, -13, and -12 (0.2mg/kg/dose). Patients greater than 15kg will be given a 3mg "test" dose on day -14 in order to limit the first dose to no more than 3 mg per the manufacturer's recommendation. This will be followed by 0.23mg/kg/dose on days -13 and -12 (to equal a total dose of approximately 0.5-0.6mg/kg). Alemtuzumab will be drawn into a sterile syringe and given to patients subcutaneously. |
Alemtuzumab (Campath®) is a recombinant DNA-derived humanized monoclonal antibody directed against CD52.
Alemtuzumab is produced in mammalian cell (Chinese hamster ovary) suspension culture in a medium containing neomycin.
Neomycin is not detectable in the final product.
Alemtuzumab is a sterile, clear, colorless, isotonic pH 6.8-7.4 solution for injection.
Alemtuzumab is supplied in single-use clear glass ampules containing 30 mg of Alemtuzumab in 3 mL of solution.
Single use vial of alemtuzumab contains 30 mg alemtuzumab, 8 mg sodium chloride, 1.44 mg dibasic sodium phosphate, 0.2 mg potassium chloride, 0.2 mg monobasic potassium phosphate, 0.1 mg polysorbate 80, and 0.0187 mg disodium edetate dihydrate.
其他名称：
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研究衡量的是什么？
主要结果指标
结果测量 |
大体时间 |
|---|---|
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Percentage of patients who have alemtuzumab levels in the optimal therapeutic range on Day 0.
大体时间：100 Days
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100 Days
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合作者和调查者
调查人员
- 首席研究员：Rebecca Marsh, MD、Children's Hospital Medical Center, Cincinnati
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
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.
Alemtuzumab的临床试验
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Fondazione IRCCS Istituto Nazionale dei Tumori,...完全的
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Milton S. Hershey Medical Center终止
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National Institute of Allergy and Infectious Diseases...招聘中
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Paul Szabolcs招聘中先天性骨髓衰竭综合征 | 炎症条件 | 原发性免疫缺陷 (PID) | 遗传性代谢紊乱 (IMD) | 遗传性贫血 | 系统性幼年特发性关节炎 (sJIA) | 青少年类风湿关节炎（JRA）美国
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Johns Hopkins UniversityPatient-Centered Outcomes Research Institute; National Multiple Sclerosis Society主动，不招人