Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 12
- 试验地点
- 1
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 25 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
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
干预措施: Filgrastim (Drug)
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
干预措施: Plerixafor (Drug)
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
干预措施: Leukapheresis (Procedure)