A Phase 1, Open Label, Multicenter Trial to Assess the Safety, Tolerability and Efficacy of MB-106 in Patients with Relapsed or Refractory CD20+ B-Cell NHL or CLL

摘要 在此应用中，我们建议优化 Mustang Bio 专有 MB-106 的可行性及其对 B- 细胞非霍奇金淋巴瘤（NHL）和慢性淋巴淋巴瘤（CLL）患者并确定 复发或难治性 CD20 B 细胞 NHL 或 CLL 患者的最佳剂量。 虽然目前的分子靶向治疗疗程已导致长期缓解，但迄今为止 不能治愈 NHL 和 CLL 癌症。因此，需要一种创新方法 个性化和具体的治疗可以持续超过缓解日期。 CAR T 细胞疗法如提议的 这是一种细胞免疫疗法，使用表达嵌合抗原受体的过继性 T 细胞，该受体特异于 靶向肿瘤相关 B 细胞抗原。所提出的方法是高度个性化的，因为每个产品 是为每位患者单独制造的。 在这项直接进入 2 期临床试验中，MB-106 将进行功效、安全性测试，并确定最佳剂量 对于患者。提议开展一项开放标签介入性、非随机过继性 T 细胞免疫治疗研究 拓宽对 MB-106 对血液、骨髓和其他测量疾病部位的影响的了解 通过输注后的细胞因子水平。在该项目结束时，MB-106 的推荐剂量 患者将被确定，并将作为未来临床试验和最终临床治疗的基础 使用。

ABSTRACT In this application, we propose to optimize the feasibility of Mustang Bio's propriety MB-106 and its impact on B- cell Non-Hodgkin's Lymphoma (NHL) and Chronic Lymphatic Lymphoma (CLL) patients and determine an optimal dosage for patients with relapsed or refractory CD20+ B-cell NHL or CLL. While the current courses of molecular targeted therapies have resulted in prolonged remissions, to date there is no curative therapeutic for NHL and CLL cancers. Therefore, an innovative approach is needed for an individualized and specific therapy that can persist past remission dates. CAR T cell therapy such as proposed here, is a cellular immunotherapy that uses adoptive T-cells expressing a chimeric antigen receptor specific to targeting tumor-associated B- cell antigens. The methodology proposed is highly individualized, as each product is manufactured individually for each patient. In this Direct to Phase 2 clinical trial, MB-106 will be tested for efficacy, safety, and determine the optimal dosage for patients. Proposed is an open-label interventional, non-randomized adoptive T-cell immunotherapy study to broaden the understanding the impact of MB-106 in blood, bone marrow, and other sites of disease measured by cytokine levels post infusion. At the conclusion of this project, the recommended dosage of MB-106 for patients will be determined and will be used as the basis for treatment in future clinical trials and ultimate clinical use.