Safety of Autologous Marrow Stromal Cells After Stroke

描述(由申请人提供)：这项研究将是一项I期剂量递增试验，以评估自体骨髓间充质干细胞(MSC)输注在急性缺血性中风患者中的安全性。中风仍然是美国成人残疾的主要原因。大多数患者没有足够早地获得医疗保健，无法从超急性中风疗法中受益。在中风的动物模型中，在脑梗塞后几天给予MSC与改善行为结果有关。在几项对患有非脑血管疾病的人类的安全性研究中，已经发现MSC是安全的。因此，MSC可能代表了一种可以改善人类中风患者预后的治疗方法，而且干预的时间窗口很宽。 具体目标将根据特定的主要和次要终点来评估接受这种治疗的患者的发病率和死亡率是否高于预期。主要终点集中在治疗90天后的死亡率和主要血栓形成/感染性不良事件。次要终点从更长远的角度考虑这些事件，并进一步评估对全身和神经功能的潜在不良影响。 长期目标是确定一种安全的治疗方法，改善急性缺血性中风患者的长期结果，并可用于大多数新诊断为中风的患者。这项研究是一项I期安全性试验，因此是在这方面评估自体骨髓间充质干细胞输注的第一步。

DESCRIPTION (provided by the applicant): This study will be a Phase I, dose-escalation trial to evaluate the safety of autologous Mesenchymal Stromal Cell (MSC) transfusion in patients with acute ischemic stroke. Stroke remains the leading cause of adult disability in the U.S. Most patients do not access healthcare early enough to benefit from hyperacute stroke therapies. In animal models of stroke, administration of MSC days after infarct has been associated with improved behavioral outcome. MSC has been found to be safe in several safety studies of humans with non-cerebrovascular diagnoses. MSC may, therefore, represent a treatment that can improve outcome in human stroke patients with a wide time-window for intervention. The specific aims will evaluate whether the morbidity and mortality rates among patients receiving this treatment are higher than expected, according to specific primary and secondary endpoints. The primary endpoint focuses on mortality and major thrombotic/infectious adverse events 90 days after treatment. The secondary endpoints consider these events over a longer term, and further evaluate potential adverse effects on systemic and neurological function. The long-term objective is to identify a treatment that is safe, improves long-term outcome in patients with acute ischemic stroke, and can be administered to a majority of patients with a new diagnosis of stroke. This study is a Phase I safety trial, and thus represents the first step in evaluating autologous MSC transfusion in this regard.