A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.
研究概览
地位
详细说明
Severe graft dysfunction, such as the degree of donor chimerism predominantly in the myeloid compartment is one of major problem in patients with Wiskott-Aldrich syndrome (WAS), especially after hematopoietic stem cell transplantation (HSCT) from alternative donor. It often leads to the development of severe thrombocytopenia or even transplants rejection. In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages due to lowing risk of mixed chimerism after HSCT. This effect is based on the fact that simultaneous use of plerixafor with G-CSF is efficient in inducing stem cell release and opening of bone marrow (BM) niches. Moreover, stem cell release probably leads to liberation of host stem cells from the anti-apoptotic effects of the BM stroma for the more powerful effect of chemotherapy.
In this study, the investigators use TCR alpha/beta grafts depletion of the grafts as basic technology for HSCT from haploidentical and unrelated donors approved in Institution.
Thus, the purpose of this study is to evaluate the safety and efficiency of myeloablative conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patients with Wiskott-Aldrich syndrome.
研究类型
注册 (预期的)
阶段
- 阶段2
联系人和位置
学习地点
-
-
-
Moscow、俄罗斯联邦、117997
- 招聘中
- Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
-
接触：
- Dmitry Balashov, MD, PhD
- 电话号码：6534 +7(495)287-6570
- 邮箱：bala8@yandex.ru
-
接触：
- Michael Maschan, Professor
- 电话号码：+7(926)287-6570
- 邮箱：mmaschan@yandex.ru
-
副研究员：
- Michael Maschan, Professor
-
副研究员：
- Alexandra Laberko, MD
-
副研究员：
- Svetlana Kozlovskaya, MD
-
副研究员：
- Elena Gutovskaya, MD
-
副研究员：
- Anna Shcherbina, Professor
-
-
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 aged ≥ 1 months and < 19 years
- Patients diagnosed with Wiskott-Aldrich syndrome eligible for an allogeneic transplantation and lacking a related HLA-matched donor
- Lansky/Karnofsky score > 40, WHO > 4
- Signed written informed consent
Exclusion Criteria:
- Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%)
- Serious concurrent uncontrolled medical disorder
- Lack of parents' informed consent.
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 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：Plerixafor/G-CSF for HSCT conditioning
Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.
|
Mobilization of hematopoietic stem (HSC) into circulation
Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.
|
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 |
|---|---|---|
|
Event free survival (EFS)
大体时间：24 months
|
The EFS probability compared with historical control.
We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia
|
24 months
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Overall survival (OS)
大体时间：24 months
|
The OS probability compared with historical control.
|
24 months
|
|
Percentage of patients with full/mixed donor chimerism
大体时间：12 months
|
Evaluation of the percentage of patients with the full/mixed donor chimerism (whole blood and CD3+ lineage).
In addition, patients will be divided in accordance with % of donors cells: >95%; 50%-95%; 10%-49%; <10%.
All data will be compared with historical control
|
12 months
|
|
Transplant related mortality (TRM)
大体时间：24 months
|
The TRM probability compared with historical control.
|
24 months
|
|
Severe thrombocytopenia (ST)
大体时间：24 months
|
The ST probability after HSCT compared with historical control
|
24 months
|
|
Autoimmune complications (AC)
大体时间：24 months
|
The AC probability after HSCT compared with historical control
|
24 months
|
|
Acute Graft Versus Host Diseases (aGVHD)
大体时间：12 months
|
Cumulative Incidence and severity of aGVHD
|
12 months
|
|
Chronic Graft Versus Host Diseases (cGVHD)
大体时间：24 months
|
Cumulative Incidence and severity of cGVHD
|
24 months
|
|
Plerixafor related complications (PRC)
大体时间：2 week
|
PRC: severity, features, incidence
|
2 week
|
合作者和调查者
调查人员
- 学习椅：Alexei Maschan, Professor、Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
- 首席研究员：Dmitry Balashov、Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
出版物和有用的链接
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 (预期的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
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.
G-CSF for Conditioning before HSCT.的临床试验
-
Stanford University终止淋巴瘤，非霍奇金 | 弥漫性大 B 细胞淋巴瘤 (DLBCL) | 淋巴瘤，B细胞 | 恶性淋巴瘤，非霍奇金美国
-
City of Hope Medical CenterNational Cancer Institute (NCI)主动，不招人
-
National Cancer Institute (NCI)完全的