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A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone

2017年5月15日 更新者：Genzyme, a Sanofi Company

A Phase 1/2 Combined Dose Ranging and Randomized, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilization of Haematopoietic Stem Cells Into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilization Regimens Alone in Pediatric Patients, Aged 1 to <18 Years, With Solid Tumours Eligible for Autologous Transplants.

This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages:

  • Stage 1 is a dose-escalation study.
  • Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study.

All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative).

Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.

研究概览

地位

完全的

条件

干预/治疗

研究类型

介入性

注册 (实际的)

46

阶段

  • 阶段2
  • 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。

学习地点

  • 丹麦
      • København Ø、丹麦、2100
        • Investigational Site Number 61
  • 以色列
      • Petach Tikva、以色列、4920235
        • Investigational Site Number 92
      • Tel-Aviv、以色列、64239
        • Investigational Site Number 91
  • 匈牙利
      • Budapest、匈牙利、1097
        • Investigational Site Number 83
  • 德国
      • Frankfurt Am Main、德国、60590
        • Investigational Site Number 33
      • Freiburg、德国、79106
        • Investigational Site Number 34
      • Hamburg、德国、20246
        • Investigational Site Number 35
      • Hannover、德国、30625
        • Investigational Site Number 31
      • München、德国、80337
        • Investigational Site Number 36
  • 意大利
      • Genova、意大利、16100
        • Investigational Site Number 21
      • Milano、意大利、20133
        • Investigational Site Number 24
      • Padova、意大利、35128
        • Investigational Site Number 23
      • Roma、意大利、00165
        • Investigational Site Number 22
      • Torino、意大利、10126
        • Investigational Site Number 26
  • 捷克语
      • Brno、捷克语、62500
        • Investigational Site Number 81
      • Praha 5 - Motol、捷克语、15006
        • Investigational Site Number 82
  • 比利时
      • Gent、比利时、9000
        • Investigational Site Number 51
  • 法国
      • Lyon、法国、69373
        • Investigational Site Number 42
      • Paris Cedex 05、法国、75248
        • Investigational Site Number 43
  • 波兰
      • Krakow、波兰、30-663
        • Investigational Site Number 85
      • Wroclaw、波兰、50-368
        • Investigational Site Number 84
  • 英国
      • Birmingham、英国、B4 6NH
        • Investigational Site Number 11
      • Glasgow、英国、G51 4TF
        • Investigational Site Number 13
  • 荷兰
      • Amsterdam、荷兰、1105 AZ
        • Investigational Site Number 72
      • Rotterdam、荷兰、3015 GJ
        • Investigational Site Number 71
  • 西班牙
      • Barcelona、西班牙、08035
        • Investigational Site Number 94
      • Madrid、西班牙、28009
        • Investigational Site Number 93

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

1年 至 18年 (孩子、成人)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria:

  • Age 2 to < 18 years during stage 1 and 1 to < 18 years during stage 2
  • Ewing's sarcoma, soft tissue sarcoma, lymphoma, neuroblastoma, brain tumors or other malignancy (excluding any form of leukemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy
  • Eligible for autologous transplantation
  • Recovered from all acute significant toxic effects of prior chemotherapy
  • Adequate performance status (for patients ≥16 years of age, defined as Karnofsky score >60 and for patients <16 years of age, defined as Lansky score >60)
  • Absolute neutrophil count >0.75 × 10^9/L
  • Platelet count >50 × 10^9/L
  • Calculated creatinine clearance (using the Schwartz method): during study Stage 1, >80 mL/min/1.73m^2 and during study Stage 2, >60 mL/min/1.73m^2
  • Aspartate aminotransferase(AST)/serum glutamic oxaloacetic transaminase(SGOT), alanine aminotransferase(ALT)/serum glutamic pyruvic transaminase (SGPT) and total bilirubin <3 × upper limit of normal
  • The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
  • Patients who are sexually active must be willing to abstain from sexual intercourse or agree to use an approved form of contraception while receiving plerixafor and/or standard mobilization treatment and for at least 3 months following any plerixafor treatment

Exclusion Criteria:

  • Any form of leukemia
  • A co-morbid condition which, in the view of the Investigator, renders the patient at high-risk from treatment complications
  • Previous stem cell transplantation
  • Persistent high percentage marrow involvement prior to mobilization will be prohibited.
  • On-going toxicities (excluding alopecia) Grade ≥2 resulting from prior chemotherapy
  • Acute infection
  • Fever (temperature >38.5°C) - if fever is between 37°C and 38.5°C, infection must be excluded as a cause
  • Known HIV seropositivity, AIDS, hepatitis C or active hepatitis B infections
  • Positive pregnancy test in post pubertal girls
  • History of clinically significant cardiac abnormality or arrhythmia
  • Use of an investigational drug which is not approved in any indication either in adults or pediatrics within 2 weeks prior to the first dose of G-CSF to be administered as part of the patient's planned standard mobilization regimen, and/or during the study up until engraftment of the transplant. If patients are on investigational drugs as part of their anti-cancer regimen, this should be discussed with the Sponsor before screening. Drugs approved for other indications that are being used in a manner considered standard of care for this transplant procedure are allowed
  • The patient (and/or their parent/legal guardian), in the opinion of the Investigator, is unable to adhere to the requirements of the study

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：随机化
  • 介入模型：并行分配
  • 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：Plerixafor 160 μg/kg
Patients will receive subcutaneous (SC) injection of 160 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
药品：plerixafor
160 μg/kg subcutaneous (SC) injection
药品：plerixafor
240 μg/kg subcutaneous (SC) injection
药品：plerixafor
320 μg/kg subcutaneous (SC) injection
实验性的：Plerixafor 240 μg/kg
Patients will receive subcutaneous (SC) injection of 240 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
药品：plerixafor
160 μg/kg subcutaneous (SC) injection
药品：plerixafor
240 μg/kg subcutaneous (SC) injection
药品：plerixafor
320 μg/kg subcutaneous (SC) injection
实验性的：Plerixafor 320 μg/kg
Patients will receive subcutaneous (SC) injection of 320 μg/kg plerixafor in addition to their standard mobilization regimen. Each dose of plerixafor will be administered in the evening 9 to 11 hours prior to apheresis (up to a maximum of 5 apheresis sessions).
药品：plerixafor
160 μg/kg subcutaneous (SC) injection
药品：plerixafor
240 μg/kg subcutaneous (SC) injection
药品：plerixafor
320 μg/kg subcutaneous (SC) injection

研究衡量的是什么？

主要结果指标

结果测量
大体时间
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
大体时间：Up to 5 days
Up to 5 days

次要结果测量

结果测量
措施说明
大体时间
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
大体时间：Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Yield of CD34+ cells for each apheresis
大体时间：Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Total CD34+ cell yield
大体时间：Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Percentage of patients proceeding to transplant
大体时间：Within 6 months of last apheresis
During Stage 1 and Stage 2
Within 6 months of last apheresis
Percentage of patients successfully engrafting
大体时间：3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Percentage of patients with durable engraftment
大体时间：3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Summary of adverse events (AEs)
大体时间：Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis)
During Stage 1 and Stage 2
Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis)
Duration of hospitalizations (planned or unplanned)
大体时间：Throughout the duration of the study
During Stage 1 and Stage 2
Throughout the duration of the study
Mobilization of tumor cells into peripheral blood
大体时间：Up to 5 days
During Stage 1 and Stage 2
Up to 5 days
Relapse rates
大体时间：3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Occurrence of secondary malignancies
大体时间：3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Incidence of primary and secondary graft failure
大体时间：3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months post-transplant
Time to secondary graft failure
大体时间：Up to 24 months post-transplant
During Stage 1 and Stage 2
Up to 24 months post-transplant
Survival rates
大体时间：3, 6, 12 and 24 months post-transplant
During Stage 1 and Stage 2
3, 6, 12 and 24 months 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。

赞助

Genzyme, a Sanofi Company

合作者

Sanofi

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

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

2014年3月3日

初级完成 (实际的)

2017年5月9日

研究完成 (实际的)

2017年5月9日

研究注册日期

首次提交

2011年1月28日

首先提交符合 QC 标准的

2011年2月1日

首次发布 (估计)

2011年2月2日

研究记录更新

最后更新发布 (实际的)

2017年5月16日

上次提交的符合 QC 标准的更新

2017年5月15日

最后验证

2017年5月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • DFI12860
  • 2010-019340-40 (EudraCT编号)
  • MOZ15609 (其他标识符：Genzyme other study code)

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.

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