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.

项目摘要/摘要 简介：骨骼组织工程实验室（Stel）Technologies，LLC是一种再生医学 公司从密歇根大学开发。 Stel的第一个产品CGEM Graft是一种完全独特的生物学 “现成的”组织工程移植物，用于修复前交叉韧带（ACL），该韧带允许患者的 身体以完善天然ACL组织，从而改善ACL损伤的膝关节稳定性和功能。 意义：在美国，每年进行35万多个ACL重建手术[1]。此外 对于该手术的高成本，这些患者在十年内有患骨关节炎（OA）的高风险 手术后。随着儿科ACL损伤的增加，长期成本和生活质量的影响将 在获得创新的新疗法之前，要越来越多[2]。当前的高尔夫标准 对于ACL修复是非手术，保守的治疗或手术修复。当前用于维修的移植物 不要恢复正常功能。 OA早期发病仍然是一种风险，在约37％的治疗病例中可以看出[2]。 拟议研究的目标：R34临床试验计划赠款旨在为我们的团队和 成功的首次人类临床试验的技术。再生医学项目非常重要 复杂而昂贵的，尚未受到风险投资（VC）社区的拥护。迄今为止，我们的 NIH和国家经济发展赠款的结合支持了研究，但要确保 在下一阶段发展的成功，我们必须在团队中进行大量投资和计划 我们的临床试验计划。骨科社区和拟议中的热情充满热情 项目将推动该技术用于人类患者的可翻译性，并帮助我们的团队 通过清除三个关键里程碑来实现更高的商业成功可能性： 目标1：为大学的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：建立操作程序，数据安全和监视计划的调查员手册 （DASP）和统计分析计划支持IRB批准I期临床试验。 ·目标3：组装研究性新药（IND）提交所需的文件 到食品和药物管理局（FDA）。 成功完成以上里程碑将有助于我们的团队设计并为高影响力的临床做准备 试用计划。我们组装了管理人才和技术专长，以帮助设计 临床研究并准备文档，以成功地在密歇根大学进行一项成功的人类试验。 Stel的“现成” CGEM GRAFT用于ACL维修是再生医学的重大进步 以及用于治疗软组织骨科损伤的变革性创新。