Phase II Trial Evaluating the Safety and Efficacy of an Allogeneic Stem Cell for

评估同种异体干细胞的安全性和功效的 II 期试验

• 批准号: 8591725
• 负责人: MARC S PENN
• 金额: $ 33万
• 依托单位: ATHERSYS, INC.
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-08-01 至 2015-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8591725
• 关键词: Academic Medical Centers; Acute; Acute myocardial infarction; Admission activity; Adopted; Adult; Age; Allogenic; American; Biological Assay; Bone Marrow; Cardiac; Cardiovascular Diseases; Cardiovascular system; Caring; Catheters; Cause of Death; Cell Therapy; Cells; Cessation of life; Chemistry; Chronic; Clinical; Clinical Data; Clinical Trials; Data; Death Rate; Development; Diabetes Mellitus; Diagnosis; Direct Costs; Distal; Double-Blind Method; Elderly; Epidemic; Event; Facilities and Administrative Costs; Functional disorder; Health system; Heart; Heart Diseases; Heart failure; Hospitalization; Incidence; Individual; Infarction; Injection of therapeutic agent; Injury; Institution; Investigation; Ischemia; Lead; Left Ventricular Ejection Fraction; Marketing; Medical; Molecular; Mononuclear; Morbidity - disease rate; Myocardial Infarction; Obesity; Outcome; Patients; Phase; Phase I Clinical Trials; Phase II Clinical Trials; Phase III Clinical Trials; Placebo Control; Play; Population; Populations at Risk; Prevalence; Prevention; Process; Protocols documentation; Quality of life; Recording of previous events; Recurrence; Risk; Risk Factors; Role; Safety; Site; Stem cells; Stents; System; Testing; Time; Translating; Tunica Adventitia; United States; Validation; Venous; Work; base; cardiovascular injury; cell preparation; cell type; clinical efficacy; cost; design; heart function; improved; improved functioning; manufacturing process; mortality; novel; novel therapeutics; patient population; public health relevance; randomized placebo controlled trial

DESCRIPTION (provided by applicant): Cardiovascular disease (CVD) represents a significant unmet medical need with over 1 million patients in the United States suffering from acute myocardial infarction (AMI) each year. This epidemic of heart attacks also represents a risk factor for further cardiovascular disease, with over 5.9 million individuals suffering from chronic heart failure (CHF) and > 670,000 new patients diagnosed with CHF annually in the United States. CHF is associated with high morbidity and mortality as well as high cost of care. In fact, taken together, CVD is the leading cause of death in the United States and is responsible for an estimated $287 billion dollars annually in direct and indirect costs, according the American Heart As the population ages and the incidence of diabetes and obesity increase, the incidence and associated cost of care for CVD Is expected to rise as well. By 2030, the number of people in US age 65 and over will increase by 80%. Despite the high cost of care and prevalence, the medical options to treat both AMI and CHF are limited. Based on the current clinical data, it would appear highly likely that cell therapy will play a role in the prevention ad treatment of cardiac dysfunction in the ensuing years. In this study, we proposed to develop in the Phase I portion of this proposal, an adult bone-marrow derived cell therapy product, MultiStem, that can formulated in a cryovial for rapid thaw and delivery to treat acute ischemic injury. For the Phase II portion, we plan to investigate the effects of cell therapy in a specific nd novel population of patients with cardiovascular disease, those patients with heart attacks that present as non-ST elevated myocardial infarcts (NSTEMI) to determine if we can reduce the morbidity and mortality and provide functional benefit to the heart. Importantly, the NSTEMI population is growing in prevalence unlike the clinical population of first-time ST elevation AMI which has been steadily declining over the past decade. Successful completion of these studies will provide the data required for a Phase III registration trial that will be required to market MultiStem for the treatment of acute cardiovascular injury.

描述（由申请人提供）：心血管疾病（CVD）代表了一种尚未满足的重大医疗需求，美国每年有超过100万患者患有急性心肌梗死（AMI）。这种心脏病发作的流行也代表了进一步心血管疾病的风险因素，在美国每年有超过590万人患有慢性心力衰竭（CHF），并且有> 670，000名新患者被诊断患有CHF。CHF与高发病率和死亡率以及高护理成本相关。事实上，总的来说，心血管疾病是美国死亡的主要原因，每年直接和间接费用估计为2870亿美元，根据美国心脏协会的数据，随着人口老龄化以及糖尿病和肥胖症发病率的增加，心血管疾病的发病率和相关护理费用预计也会上升。到2030年，美国65岁及以上的人口将增加80%。尽管护理成本高且患病率高，但治疗AMI和CHF的医疗选择有限。根据目前的临床数据，细胞疗法很可能在随后的几年中在预防和治疗心功能不全中发挥作用。在这项研究中，我们建议在本提案的I期部分开发一种成人骨髓源性细胞治疗产品MultiStem，该产品可以在冷冻管中配制，用于快速解冻和输送，以治疗急性缺血性损伤。对于II期部分，我们计划研究细胞疗法在特定和新型心血管疾病患者人群中的作用，这些患者的心脏病发作表现为非ST段抬高型心肌梗死（NSTEMI），以确定我们是否可以降低发病率和死亡率并为心脏提供功能益处。重要的是，NSTEMI人群的患病率正在增长，而首次ST段抬高AMI的临床人群在过去十年中一直在稳步下降。这些研究的成功完成将提供III期注册试验所需的数据，该试验将需要上市用于治疗急性心血管损伤的MultiStem。