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Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

2026年8月28日 更新者：St. Jude Children's Research Hospital

The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy.

Primary objective:

- To characterize the safety of Filgrastim plus plerixafor in participants with bone marrow failure syndromes as determined by the incidence of adverse events (AEs).

Secondary Objectives:

  • To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts.
  • To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak peripheral blood CD34+ counts.
  • To estimate efficacy of Filgrastim plus plerixafor for HSPC mobilization and apheresis collection in participants as determined by the yield of CD34+ cells (CD34+ cells/kg).

研究概览

地位

尚未招聘

条件

干预/治疗

详细说明

This is a phase I, open-label, single-center study to evaluate the safety of Filgrastim plus plerixafor stem cell mobilization and apheresis in patients with BMFS. This study will include a screening period with labs, physical examination, and bone marrow evaluation at least 6 months prior to mobilization and apheresis, an intervention period that includes mobilization and apheresis of patient HSPCs, and outpatient follow-up within 7-10 days after intervention. Study staff will follow up with the participant via telephone approximately 30 days after mobilization and apheresis. A bone marrow evaluation will be done within 6 months post-intervention.

研究类型

介入性

注册 (估计的)

12

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

学习地点

  • 美国
    • Tennessee
      • Memphis、Tennessee、美国、38105-2794
        • Saint Jude Children's Research Hospital
        • 接触：

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

  • 成人

接受健康志愿者

不

描述

Inclusion Criteria:

  • Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy
  • Age ≥ 18 years - 25 years

  • The following hematological parameters need to be met (regardless of transfusion or growth factor support)

    • Hb > 8 g/dL
    • ANC > 500/mm3
    • Platelet > 30,000/mm3
  • Bone marrow evaluation within the preceding 6 months prior to mobilization and apheresis
  • Participants should either have a central venous catheter (CVC) in place, be able to undergo apheresis without requiring a CVC, or agree to having a temporary apheresis catheter placed
  • Karnofsky score >80
  • Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II
  • Female participants of childbearing age should have a negative serum pregnancy test within one week of beginning Filgrastim and plerixafor administration

Exclusion Criteria:

  • Participant with sickle cell disease
  • Participant who has had a prior autologous or allogeneic HSCT
  • Active viral, bacterial, fungal, or parasitic infection
  • Total bilirubin >2.5x ULN or transaminases >5x ULN
  • Moderate or severe renal failure defined as serum/plasma creatinine >1.5 mg/dL and an estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 based on the CKD-Epi equation or the St. Jude equation
  • Diagnosis of MDS or other hematologic malignancy
  • History of malignancy
  • Known allergy to or contraindication for Filgrastim or plerixafor administration, or medications routinely administered during apheresis
  • Splenomegaly (size greater than upper limit of normal on examination)
  • Any disease or concomitant process that is not compatible with the study as per investigator opinion
  • Concomitant treatment with alternative investigational agent or participation in another clinical trial with an investigational drug within 5 half-lives of the investigational agent
  • Unwillingness to use a highly effective method of contraception for 1 month after plerixafor or GCSF
  • Pregnancy
  • Inability or unwillingness of research participant to give written informed consent.

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：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
实验性的：BDSTEM Treatment

Participants in this study will receive a twice daily dose of Filgrastim (GCSF) (5 mcg/kg BID) SQ starting on day 1 for 5 days followed by a single dose of SQ plerixafor (0.24 mg/kg) on day 5 followed by collection of CD34+ HSPCs via apheresis.

A portion of cells collected from the participant will be stored as backup to be used toward future gene therapy endeavors. The remaining cells will be donated for research studies
药品：Filgrastim
Administered twice daily dose starting on day 1 for 5 days.
药品：Plerixafor
Administered on day 5 via IV.
程序：Leukapheresis
Peripheral venous access or through a central venous catheter approximately 4-5 hours after the dose of plerixafor is given.

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
Incidence of treatment-emergent adverse events following filgrastim plus plerixafor administration
大体时间：From initiation of drug administration through Day +7 to +10 follow-up
Safety will be assessed by the incidence, type, and severity of adverse events occurring after administration of filgrastim plus plerixafor in participants with bone marrow failure syndromes.
From initiation of drug administration through Day +7 to +10 follow-up

次要结果测量

结果测量
措施说明
大体时间
Number of participants achieving peripheral blood CD34+ counts ≥5 cells/µL
大体时间：From initiation of plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Feasibility of hematopoietic stem and progenitor cell mobilization will be assessed by peripheral blood CD34+ cell counts measured after plerixafor administration and prior to or during apheresis.
From initiation of plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Peripheral blood CD34+ kinetics following filgrastim plus plerixafor administration
大体时间：After plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Peripheral blood CD34+ cell counts will be measured after plerixafor administration and prior to or during apheresis.
After plerixafor administration through completion of apheresis, or 6 hours after drug administration if apheresis is not performed
Observed CD34+ cell yield after 1 blood volume apheresis
大体时间：At completion of 1 blood volume apheresis on Day 5
CD34+ cell yield (CD34+ cells/kg) collected after processing 1 blood volume during apheresis following filgrastim plus plerixafor administration.
At completion of 1 blood volume apheresis on Day 5
Estimated total CD34+ cell yield from projected full-volume apheresis
大体时间：At completion of 1 blood volume apheresis on Day 5
Estimated total CD34+ cell yield (CD34+ cells/kg) projected from the observed yield after processing 1 blood volume during apheresis.
At completion of 1 blood volume apheresis on Day 5

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

St. Jude Children's Research Hospital

调查人员

  • 首席研究员：Alexis Leonard, MD、St. Jude Children's Research Hospital

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。

有用的网址

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11月1日

初级完成 (估计的)

2028年12月1日

研究完成 (估计的)

2029年7月1日

研究注册日期

首次提交

2026年4月16日

首先提交符合 QC 标准的

2026年5月8日

首次发布 (实际的)

2026年5月13日

研究记录更新

最后更新发布 (实际的)

2026年8月31日

上次提交的符合 QC 标准的更新

2026年8月28日

最后验证

2026年8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

  • BDSTEM

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

IPD 共享时间框架

Data will be made available at the time of article publication.

IPD 共享访问标准

Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.

IPD 共享支持信息类型

  • 研究方案
  • 树液
  • 国际碳纤维联合会

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

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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