BMT Autologous MSCs for GvHD
关键词
抽象
描述
EPIC MSC2014-002 solution- Autologous Mesenchymal Stromal Cells expanded using pooled human platelet lysate,is made up of autologous marrow-derived mesenchymal stromal cells ex vivo expanded numerically for approximately 14 days using pooled human Platelet Lysate (phPL), harvested from culture on the day of infusion and suspended at a concentration of 4 million cells/ml in Plasmalyte A with 0.5% human serum albumin. This is a phase I dose-escalation, open label, non-randomized, non-placebo controlled, single group assignment study to evaluate the safety and tolerability of EPIC MSC2014-002. The product will be infused intravenously and will be administered at one of three dose levels: (Dose level 1): Single Cell infusion 2 x 10^6 cells/kg, (Dose Level 2): Two weekly Cell infusions 2 x 10^6 cells/kg , (Dose level 3): Four weekly Cell infusion 2 x 10^6 cells/kg. This Phase I clinical trial will enroll 12-24 subjects with acute or chronic GVHD. The duration of this study for each patient is 1 year. The investigators anticipate that this study will be completed within 3 years of commencement.
Objectives:
- To determine the safety and tolerability of infusing escalating doses of autologous MSCs for patients with acute or chronic GVHD.
- To assess the overall response rate of acute and chronic GVHD to autologous MSC infusion. These data will be used to plan future, larger clinical trials to evaluate the efficacy of autologous MSCs for the treatment of GVHD.
- To determine the effect of MSC infusion on lymphocyte phenotype, inflammatory biomarkers and GVHD specific biomarker levels
日期
| 最后验证: | 04/30/2020 |
| 首次提交: | 02/04/2015 |
| 提交的预估入学人数: | 02/04/2015 |
| 首次发布: | 02/09/2015 |
| 上次提交的更新: | 05/10/2020 |
| 最近更新发布: | 05/12/2020 |
| 实际学习开始日期: | 12/31/2014 |
| 预计主要完成日期: | 10/31/2020 |
| 预计完成日期: | 04/30/2025 |
状况或疾病
干预/治疗
Biological: Autologous mesenchymal stromal cells (MSCs)
相
手臂组
| 臂 | 干预/治疗 |
|---|---|
| Experimental: Dose Level 1: Infusion of MSCs First three subjects enrolled will receive a single infusion of mesenchymal stromal cells based on their individual weight | |
| Experimental: Dose Level 2: Infusion of MSCs Subsequent subjects enrolled will receive two infusions (a week apart) of mesenchymal stromal cells based on their individual weight | |
| Experimental: Dose Level 3: Infusion of MSCs Subsequent subjects enrolled will receive four infusions (a week apart) of mesenchymal stromal cells based on their individual weight |
资格标准
| 有资格学习的年龄 | 12 Years 至 12 Years |
| 有资格学习的性别 | All |
| 接受健康志愿者 | 是 |
| 标准 | Inclusion Criteria: - Age: patients must be ≥12 years old and weigh > (25 kg) at the time of study entry. - Patients must have received an allogeneic stem cell transplant for a hematologic malignancy. - Must have one of the following diagnoses: - Acute GVHD (grade II-IV) requiring systemic therapy and refractory/unresponsive to glucocorticoid (>1 mg prednisone-equivalent/kg x 1 week) - Chronic GVHD that is extensive and not improved despite therapy with glucocorticoid (> 0.5 mg prednisone-equivalent/kg/day) and therapeutic doses of a calcineurin inhibitor for at least 4 weeks, or worsened within 2 weeks, or overlap syndrome not responding to glucocorticoid treatment (>1 mg prednisone-equivalent/kg x 1 week) Exclusion Criteria: - Active invasive fungal infection requiring treatment with anti-fungal medication. - Active viral infection requiring treatment with anti-viral medication. - Persistence/relapse at the time of study entry of the primary malignancy for which the transplant was performed. Patients with a history of relapsed malignancy who have achieved a remission at the time of evaluation for study participation will not be excluded. - Known T-cell donor chimerism of <50%. - Documented DLCO <50% (if performed within 90 days of enrollment) or requirement for supplemental oxygen. - Pregnancy or breastfeeding. Patients of childbearing capability should agree to use contraception. |
结果
主要结果指标
1. Safety and Tolerability of EPIC MSC2014-002 based on dose limiting toxicities (DLTs) [6 months]
次要成果指标
1. Overall Response Rate for acute GVHD subjects [6 months]
2. Overall Response Rate for chronic GVHD subjects [6 months]
3. Transplant-related mortality [6 months]
4. Incidence of Relapse [6 months]
5. Disease-free survival [5 years post transplant]
6. Overall-survival [5 years post transplant]
