Safety of Allogeneic Cardiosphere-Derived Cells for Dilated Cardiomyopathy

同种异体心肌球衍生细胞治疗扩张型心肌病的安全性

• 批准号: 8878331
• 负责人: Rachel Ruckdeschel Smith
• 金额: $ 88.08万
• 依托单位: CAPRICOR, INC.
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-01 至 2016-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8878331
• 关键词: Acute; Acute myocardial infarction; Adenosine; Affect; Aftercare; Allogenic; American; Animal Model; Anterior; Arrhythmia; Attenuated; Autologous; Award; Biological Assay; Blood flow; Businesses; Cardiac; Cardiotonic Agents; Cardiovascular Diseases; Cause of Death; Cell Therapy; Cells; Chronic; Cicatrix; Clinical; Clinical Data; Clinical Protocols; Clinical Trials; Coronary artery; Cryopreservation; Cyclosporine; Data; Development; Dilated Cardiomyopathy; Disease; Double-Blind Method; EFRAC; Enrollment; Ensure; Enzymes; Event; Funding; Future; Glean; Goals; Gold; HLA Antigens; Health; Heart failure; Hour; Immune; Immunosuppression; Infarction; Inflammation; Infusion procedures; Injury; Intervention; Investigational New Drug Application; Lead; Left; Left Ventricular Dysfunction; Left Ventricular Remodeling; Licensing; Life; Magnetic Resonance Imaging; Marketing; Multicenter Studies; Multicenter Trials; Myocardial Infarction; Myocardial perfusion; Myocardium; National Heart, Lung, and Blood Institute; Natural regeneration; Patients; Phase I Clinical Trials; Placebo Control; Population; Positioning Attribute; Preclinical Testing; Prevention; Process; Product Approvals; Protocols documentation; Quality Control; Randomized; Reaction; Reperfusion Therapy; Resolution; Safety; Secure; Series; Small Business Innovation Research Grant; Source; Surrogate Endpoint; Symptoms; System; Testing; Time; Tissues; United States; United States Food and Drug Administration; United States National Institutes of Health; Ventricular; abstracting; base; clinical research site; cohort; commercialization; design; effective therapy; experience; falls; immunological status; man; manufacturing process; meetings; mortality; myocardial damage; open label; patient population; percutaneous coronary intervention; pre-clinical; regenerative; regenerative agent; regenerative therapy; response; restoration; safety testing; scale up; sound; standard care; success

DESCRIPTION (provided by applicant): Project Abstract: Capricor, Inc. has an allogeneic cell therapy product, CAP-1002, presently in clinical development. CAP-1002 consists of cardiosphere-derived cells (CDCs), grown from a donor unrelated to the recipient, using a proprietary process exclusively licensed to Capricor. Autologous CDCs have demonstrated their utility as a regenerative agent: able to reduce infarct size (a.k.a scar size), add viable tissue mass, and attenuate adverse left ventricular (LV) remodeling when administered to patients several months after a myocardial infarction (MI). The magnitude of benefit seen in CDC-treated patients could foreshadow a reduction in future adverse clinical events. Allogeneic CDCs are currently being tested in a similar post-MI population in a randomized, double-blinded, placebo-controlled, multi-center safety and efficacy study. The current proposal aims to test the safety of CAP-1002 administered in the acute MI setting, adjunctive to the gold-standard treatment (i.e. PCI [percutaneous coronary intervention]), rapidly after restoration of blood flow (or reperfusion), as a cardioprotective agent. Capricor has an approved IND (Investigational New Drug application) for the use of CAP-1002 in the treatment and/or prevention of LV dysfunction following MI, and the proposed clinical trial falls under the umbrella of this indication. Preclinial data support the safe use of CAP-1002 in acute MI and reveal the product's ability to limit infarct size and reduce acute myocardial damage. CAP-1002 is manufactured in an existing GMP (Good Manufacturing Practice compliant) facility and will be stored at the clinical site for immediate availability. CAP-1002 is formulated such that it can be prepared for use within minutes and administered within minutes. Patients will be treated open-label with CAP-1002 within hours of successful reperfusion. Safety endpoints will include assessments of blood flow in the coronary arteries and microvasculature, resolution of myocardial damage, immune reaction, arrhythmias, and MACE (major adverse cardiac events). Efficacy assessments will examine infarct size, LV volumes, and ejection fraction by MRI (magnetic resonance imaging). Funding for a follow-on trial that would demonstrate efficacy will be pursued concurrently. Capricor is well-positioned to secure financing for the next study, particularly with an ongoing multi-center study utilizing the same product in a complementary patient population. The company has a sound regulatory strategy and advisors in place to guide interactions with the FDA (Food and Drug Administration) as CAP-1002 is advanced toward approval. The current manufacturing scale and facility will suffice through early commercialization and plans to scale up are underway. Capricor has identified several potential avenues by which to enter the public market and maintains active business development efforts. The company anticipates being revenue generating within a few years of project completion. The ongoing and proposed clinical trial series encompass nearly the full continuum of disease states following MI. CAP-1002 developed successfully for all MI patients would offer both a cardioprotective and regenerative therapy, to be administered as needed.

项目摘要：Capricor， Inc.有一种同种异体细胞治疗产品CAP-1002，目前处于临床开发阶段。CAP-1002由心球衍生细胞（cdc）组成，由与受体无关的供体生长，使用独家授权给Capricor的专有工艺。自体cdc已经证明了其作为再生剂的实用性：在心肌梗死（MI）几个月后给药时，能够减少梗死面积（又称疤痕大小），增加活组织质量，并减轻不良左心室（LV）重构。在cdc治疗的患者中看到的获益幅度可能预示着未来不良临床事件的减少。同种异体cdc目前正在类似的心肌梗死后人群中进行一项随机、双盲、安慰剂对照、多中心安全性和有效性研究。目前的提案旨在测试…的安全性