NCT05443464撤回1 期
A Phase I Safety Study of Single Dose Allogeneic Bone Marrow Derived MSCs for Steroid Refractory Acute Graft vs. Host Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 主要终点
- Safety events
研究概览
简要总结
The objective of this clinical study is to demonstrate safety and feasibility of single-dose infusion of Ossium MSCs product (OSSM-001) to treat steroid refractory acute GVHD (SR-aGVHD).
详细描述
The dose escalation will be used to establish a maximum tolerated dose, starting from 2M cells/kg. If no safety issues are seen, then MSCs dose maybe escalated to 6M cells/kg, 12M cells/kg and 24M cell/kg based on data review from each cohort. Additional studies will be conducted to establish a dosing regimen and to evaluate the safety and efficacy of OSSM-001 for SR-aGVHD patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Minimum 18 years of age who have undergone hematopoietic cell transplantation (HCT), from any donor source donor (including HLA-matched related and unrelated, haploidentical and umbilical cord donors) with any conditioning regimen
- •Clinically diagnosed Grades II to IV acute GVHD as per standard criteria occurring within 100 days of HCT
- •Confirmed diagnosis of steroid refractory aGVHD defined as patients administered high-dose systemic corticosteroids (methylprednisolone 2 mg/kg/day [or equivalent prednisone dose 2.5 mg/kg/day]), given alone or combined with calcineurin inhibitors (CNI) and either:
- •Progressing based on organ assessment after at least 3 days at the time of initiation of high-dose systemic corticosteroid +/- CNI for the treatment of Grade II-IV aGVHD, OR
- •Failure to achieve at a minimum partial response based on organ assessment after 7 days compared to organ stage at the time of initiation of high-dose systemic corticosteroid +/- CNI for the treatment of Grade II-IV aGVHD, OR
- •Patients who fail corticosteroid taper defined as fulfilling either one of the following criteria:
- •i. Requirement for an increase in the corticosteroid dose to methylprednisolone ≥2 mg/kg/day (or equivalent prednisone dose ≥2.5 mg/kg/day), OR
- •ii. Failure to taper the methylprednisolone dose to <0.5 mg/kg/day (or equivalent prednisone dose <0.6 mg/kg/day) for a minimum 7 days.
- •Minimum Karnofsky Performance Level of at least 30 or higher at the time of study entry.
排除标准
- •Has received more than one systemic treatment for steroid refractory aGVHD in addition to steroids.
- •Received stem cell therapy in the past
- •Presence of an active uncontrolled infection including significant bacterial, fungal, viral, or parasitic infection requiring treatment
- •Presence of relapsed primary malignancy, or who have been treated for relapse after the HCT was performed, or who may require rapid immune suppression withdrawal as pre-emergent treatment of early malignancy relapse.
- •Evidence of pulmonary infiltrate or hemorrhage based on imaging or requiring high flow oxygen via face mask
- •Patients who have had treatment with any other investigational agent, device, or procedure within 30days (or 5 half-lives, whichever is greater) prior to enrollment.
- •Patients who have received more than one HCT
- •Any medical or psychological condition or situation deemed by the Investigators to put the patient at increased risk of complications or non-compliance.
- •Unresolved veno-occlusive disease
- •HLA antibody screen positive for HLA antibodies specific against the MSCs products
- •ALT or AST > 5X of upper limit of normal
- •Serum Bilirubin >2 X of upper limit of normal
- •GFR <50 ml/min
- •SpO2 <94% despite being on continuous supplemental oxygen
- •Patients requiring continuous >4L/minute of supplemental oxygen (irrespective of oxygen saturation)
结局指标
主要结局
Safety events
时间窗: 112 days (16 weeks)
OSSM-001 related safety events
次要结局
- Maximum tolerated dose (MTD)(112 days (16 weeks))
研究者
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