COG ADVL0317: A PHASE I STUDY OF PS-341 (VELCADE, BORTEZOMIV) IN PED PATIENTS

COG ADVL0317：PS-341（VELCADE、BORTEZOMIV）在 PED 患者中的 I 期研究

• 批准号: 7379434
• 负责人: JULIE R PARK
• 金额: $ 0.17万
• 依托单位: UNIVERSITY OF WASHINGTON
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2007-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7379434
• 关键词: COG; ADVL0317; PHASE; STUDY; PS

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This phase I study tests the safety of a new experimental drug called PS-341 (Velcade or Bortezomib) in refractory/recurrent leukemia patients. (Leukemia is a type of cancer that affects a child's blood cells, recurrent means the disease came back after being treated, and refractory means that the disease is hard to treat.) Laboratory experiments performed with PS-341 showed that it kills cancer cells by stopping their growth. A safe dose of PS-341 has already been found in adults and in children with solid tumors, but a safe dose has not been found in children with leukemia. The goals of this study are to test the safety of PS-341, to see what effects (good and bad) they have on children and children's leukemia, and to find the highest dose of PS-341 that can be given to children with leukemia without causing severe side effects. Other goals of this study are to learn how the body handles the drug, and to better understand how cancer cells are different from healthy cells. The study is designed such that the children are separated into groups. The first group of children will be given PS-341 at the starting dose. If the side effects at the starting dose are not too serious, the next group of children will be given a higher dose of PS-341. This will continue until the maximum dose is found that does not result in unacceptable side effects. The dose of PS-341 given will not be increased in each individual child. Each course of therapy will last 21 days. During each course, the patient will be given PS-341 on days 1, 4, 8 and 11 of each course. PS-341 will be given through a vein. A sample overview of a treatment course is below: Monday Tuesday Wednesday Thursday Friday Saturday Sunday Week 1 PS-341 PS-341 Week 2 PS-341 PS-341 Week 3 Rest period; no PS-341 is given this week Day 18-22 Evaluation: retreat if disease is not worse and side effects are tolerable Children on this study will be given standard medical exams during therapy, such as physical exams, protein level tests and tests to examine liver and kidney function. A bone marrow aspirate will be done at the end of every course for the first three courses, and then at the end of every other course. In addition to these standard tests, patients will be the given the option to have blood samples taken on a few days during the first course of therapy for tests called pharmacokinetics. Pharmacokinetic tests find out how the drug is behaving in cells in the body. Patients will also be given the option to participate in other special research studies involving their bone marrow and blood samples.

该子项目是利用 NIH/NCRR 资助的中心拨款提供的资源的众多研究子项目之一。子项目和研究者 (PI) 可能已从另一个 NIH 来源获得主要资金，因此可以在其他 CRISP 条目中得到体现。列出的机构是中心的机构，不一定是研究者的机构。这项 I 期研究测试了一种名为 PS-341（Velcade 或 Bortezomib）的新实验药物在难治性/复发性白血病患者中的安全性。 （白血病是一种影响儿童血细胞的癌症，复发性意味着疾病在治疗后复发，难治性意味着该疾病难以治疗。）PS-341 进行的实验室实验表明，它可以通过阻止癌细胞生长来杀死癌细胞。已经在成人和患有实体瘤的儿童中发现了 PS-341 的安全剂量，但尚未在患有白血病的儿童中找到安全剂量。这项研究的目标是测试 PS-341 的安全性，看看它们对儿童和儿童白血病有什么影响（好和坏），并找到可以给予白血病儿童而不引起严重副作用的 PS-341 最高剂量。这项研究的其他目标是了解身体如何处理药物，并更好地了解癌细胞与健康细胞有何不同。 该研究的设计是将孩子们分成几组。第一组儿童将接受起始剂量的 PS-341。如果起始剂量的副作用不太严重，下一组儿童将接受更高剂量的 PS-341。这将持续下去，直到发现最大剂量不会导致不可接受的副作用。每个儿童的 PS-341 剂量不会增加。 每个疗程将持续21天。在每个疗程期间，患者将在每个疗程的第 1、4、8 和 11 天接受 PS-341。 PS-341 将通过静脉注射。治疗过程的示例概述如下： 周一周二周三周四周五周六周日第 1 周 PS-341 PS-341 第 2 周 PS-341 PS-341 第 3 周 休息期；本周不给予 PS-341 第 18-22 天 评估：如果疾病没有恶化并且副作用可以忍受，则撤退 参与本研究的儿童将在治疗期间接受标准医疗检查，例如体检、蛋白质水平测试以及肝肾功能检查。前三个疗程的每个疗程结束时将进行骨髓抽吸，然后在其他疗程结束时进行骨髓抽吸。 除了这些标准测试之外，患者还可以选择在第一个疗程期间的几天内采集血液样本，进行称为药代动力学的测试。药代动力学测试可了解药物在体内细胞中的行为方式。患者还可以选择参与涉及骨髓和血液样本的其他特殊研究。