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

规划用于 ACL 修复的全生物细胞生成移植物 (CGEM) 的临床试验

• 批准号: 10724487
• 负责人: LISA M LARKIN
• 金额: $ 12.1万
• 依托单位: PRO THERAPEUTICS LLC
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-08-01 至 2025-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10724487
• 关键词: Acceleration; Acute; Animal Model; Animals; Anterior Cruciate Ligament; Applications Grants; Arthritis; Biological; Biological Products; Biomechanics; Budgets; Capital; Cells; Childhood; Clinical; Clinical Research; Clinical Trials; Clinical Trials Support Unit; Collaborations; Communities; Complex; Data; Degenerative polyarthritis; Development; Documentation; Economic Development; Ensure; Evaluation; Foundations; Fright; Gait; Goals; Golf; Grant; Healthcare; Human; Incidence; Injury; Institutional Review Boards; Investigational Drugs; Investigational New Drug Application; Investments; Joint Instability; Knee; Knee Injuries; Knee joint; Laboratories; Ligaments; Manuals; Measurement; Mechanoreceptors; Michigan; Monitor; Movement; Muscle function; Natural regeneration; Operative Surgical Procedures; Orthopedics; Outcome; Patient-Focused Outcomes; Patients; Phase I Clinical Trials; Play; Positioning Attribute; Probability; Procedures; Protocols documentation; Quality of life; Reconstructive Surgical Procedures; Regenerative Medicine; Regenerative capacity; Research; Research Personnel; Risk; Risk Factors; Safety; Site; Solid; Speed; Statistical Data Interpretation; Surgeon; Talents; Technical Expertise; Technology; Tendon structure; Testing; Tissue Engineering; Tissues; United States Food and Drug Administration; United States National Institutes of Health; Universities; acute care; anterior cruciate ligament injury; anterior cruciate ligament reconstruction; care costs; clinical trial readiness; clinically relevant; cost; design; early onset; first-in-human; follow-up; graft failure; high risk; improved; innovation; knee replacement arthroplasty; laboratory equipment; pre-Investigational New Drug meeting; procedure cost; programs; randomized, clinical trials; repaired; restoration; scaffold; skeletal tissue; soft tissue; standard of care; success; trial planning

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.

项目总结/摘要 简介：Skeleton组织工程实验室（STEL）技术有限责任公司是一家再生医学公司， 公司从密歇根大学剥离出来。STEL的第一个产品CGEM移植物是一种独特的全生物 用于修复前交叉韧带（ACL）的“现成”组织工程移植物， 身体再生原生ACL组织，从而改善ACL损伤的膝关节稳定性和功能。 重要性：美国每年进行超过35万例ACL重建手术[1]。此外 由于手术费用高昂，这些患者在十年内患骨关节炎（OA）的风险很高 手术后。随着儿童ACL损伤的增加，长期成本和生活质量的影响将 在创新的新治疗方法出现之前，这是一个日益增加的医疗保健问题[2]。目前的高尔夫标准 ACL修复的方法有非手术、保守治疗或手术修复。目前用于修复的移植物 无法恢复正常功能。早发性OA仍然是一种风险，在约37%的治疗病例中可见[2]。 拟议研究的目标：这项R34临床试验计划资助的重点是准备我们的团队， 成功进行首次人体临床试验的技术。再生医学项目 复杂且昂贵，尚未被风险投资（VC）社区所接受。迄今为止，我们的 研究得到了NIH和州经济发展赠款的支持，但为了确保 为了在下一个发展阶段取得成功，我们必须在团队和规划方面进行重大投资， 我们的临床试验项目整形外科界有很大的热情， 该项目将提高这项技术在人类患者中使用的可翻译性，并帮助我们的团队 通过清除三个关键里程碑来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 密歇根州。 ·目标2：建立研究者操作程序手册、数据安全和监测计划 （DASP）和统计分析计划，以支持IRB批准I期临床试验。 目标3：收集新药研究（IND）提交所需的文件 美国食品和药物管理局（FDA）。 成功完成上述里程碑将有助于我们的团队设计和准备一个高影响力的临床 试用程序。我们汇集了必要的管理人才和技术专长，帮助设计 临床研究，并为密歇根大学成功的首次人体试验准备文件。 STEL用于ACL修复的“现成”CGEM移植物代表了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。