Planning for clinical trial of fully-biologic, cell generated graft (CGEM) for ACLrepair

项目摘要/摘要 简介：骨组织工程实验室(STEL)技术有限公司是一种再生医学 这家公司是从密歇根大学分拆出来的。斯特尔的第一个产品，CGEM移植物，是一种独特的完全生物的 “现成”组织工程移植修复前交叉韧带(ACL)，使患者的 身体再生天然的前交叉韧带组织，从而改善膝关节的稳定性和功能，以应对前交叉韧带损伤。 意义：美国每年有超过350,000例前交叉韧带重建手术[1]。此外 由于手术费用高昂，这些患者在十年内发展为骨关节炎(OA)的风险很高 手术后。随着儿童前交叉韧带损伤的增加，长期成本和生活质量的影响将 在创新的新疗法问世之前，这将是一个日益令人担忧的医疗问题[2]。现行的高尔夫标准 对于前交叉韧带的修复，要么是非手术治疗，要么是手术修复。目前用于修复的移植物 不要恢复正常功能。早发性骨性关节炎仍是一种风险，约37%的治疗病例可见[2]。 建议研究的目标：这项R34临床试验计划拨款专注于为我们的团队和 成功进行首个人类临床试验的技术。再生医学项目是巨大的 复杂且昂贵，并未受到风险投资(VC)社区的欢迎。到目前为止，我们 这项研究得到了美国国立卫生研究院和州经济发展基金的联合支持，但为了确保 要取得下一阶段开发的成功，我们必须在团队和规划方面进行重大投资 我们的临床试验计划。整形外科社区和建议的 该项目将提高这项技术用于人类患者的可译性，并帮助我们的团队 通过清除三个关键里程碑获得更高的商业成功概率： ·目标1：为大学I期临床试验制定详细的临床研究方案和预算 来自密歇根州。 ·目标2：制定操作程序、数据安全和监测计划的调查员手册 (DASP)和统计分析计划，以支持IRB批准I期临床试验。 ·目标3：汇编提交研究新药(IND)所需的文件 给美国食品和药物管理局(FDA)。 上述里程碑的成功完成将有助于我们的团队设计和准备高影响力的临床 试验计划。我们已经汇集了必要的管理人才和技术专长，以帮助设计 在密歇根大学进行临床研究，并为成功的首例人体试验准备文件。 STEL用于修复前交叉韧带的现成CGEM移植物代表着再生医学的重大进步 以及软组织骨科损伤治疗的变革性创新。

PROJECT SUMMARY/ABSTRACT Introduction: Skeletal Tissue Engineering Laboratories (STEL) Technologies, LLC is a regenerative medicine company spun out of the University of Michigan. STEL’s first product, the CGEM graft, is a unique fully biologic “off-the-shelf” tissue-engineered graft for repair of the anterior cruciate ligament (ACL) that allows the patient’s body to regrow native ACL tissue, resulting in improved knee stability and function for the ACL injuries. Significance: More than 350,000 ACL reconstruction surgeries are performed each year in the U.S. [1]. In addition to the high cost of the procedure, these patients are at high risk of developing osteoarthritis (OA) within a decade after surgery. With the increase in pediatric ACL injuries, the long-term costs and quality of life implications will be an increasing healthcare concern until innovative new treatments are available [2]. The current golf standard for ACL repair is either non-surgical, conservative treatment or surgical repair. The current grafts used for repair don’t restore normal function. Early onset OA remains a risk and is seen in about 37% of treated cases [2]. Goals of Proposed Research: This R34 clinical trial planning grant is focused on preparing our team and technology for a successful first-in-human clinical trial. Regenerative medicine projects are tremendously complex and expensive and have not been embraced by the Venture Capital (VC) community. To date, our research has been supported by a combination of NIH and state economic development grants, but to ensure success with the next stage of development, we must make significant investments in the team and planning of our clinical trial program. There is significant enthusiasm from the orthopedic community and the proposed project will advance the translatability of this technology for use in human patients and help our team attain a higher probability of commercial success by clearing three key milestones: · AIM 1: Develop a detailed clinical study protocol and budget for a Phase I clinical trial at University of Michigan. · AIM 2: Establish Investigator Manual of Operating Procedures, data safety and monitoring plan (DASP) and statistical analysis plan to support IRB approval of Phase I clinical trial. · AIM 3: Assemble the Documentation Needed for the Investigational New Drug (IND) Submission to the Food and Drug Administration (FDA). Successful completion of the above milestones will help our team design and prepare for a high-impact clinical trial program. We have assembled the management talent and technical expertise necessary to help design the clinical study and prepare the documentation for a successful first-in-human trial at the University of Michigan. STEL’s “off-the-shelf” CGEM graft for ACL repair represents a significant advancement for regenerative medicine and a transformative innovation for the treatment of soft tissue orthopedic injuries.