CLINICAL TRIAL: PI TRIAL OF IMMUNOSTIMULATION JVRS-100 FOR RELAPSED OR REFRACTOR

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 这是一项I期方案，旨在评估JVRS-100（一种阳离子脂质DNA复合物（CLDC））治疗复发性/难治性白血病患者的安全性和耐受性。 目前，这方面的预测令人沮丧。JVRS-100由阳离子脂质和质粒DNA的复合物组成，不含基因插入物（非编码DNA），是先天性和适应性免疫的有效激活剂，诱导NK、T和B细胞活化，并刺激干扰素-、干扰素-、IL-12和其他细胞因子的分泌。这是一项2阶段I期试验。 在第1阶段，将遵循加速滴定方案，每个剂量水平各有1例患者。 第2阶段将在第1阶段观察到剂量限制性毒性（DLT）后或达到第1阶段的最大剂量后开始。第2阶段将遵循改良的Fibonacci方案，每个剂量水平3-6例受试者，直至确定推荐的2期剂量（RP 2D）。 然后将队列扩展至最多12例患者，以更全面地评价RP 2D。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is a Phase I protocol to evaluate the safety and tolerability of JVRS-100, a cationic lipid DNA complex (CLDC), for the treatment of patients with relapsed/refractory leukemia. This currently has a dismal prognosis. JVRS-100 consists of complexes of cationic lipid and plasmid DNA without a gene insert (non-coding DNA) which are potent activators of innate and adaptive immunity, induce NK, T and B cell activation, and stimulate the secretion of interferon-¿, interferon-¿, IL-12 and other cytokines. This is a 2 stage Phase I trial. In stage 1, an accelerated titration schema will be followed with one patient at each dose level. Stage 2 will commence after a dose limiting toxicity (DLT) is observed in stage 1 or after the maximum dose for stage 1 is reached. Stage 2 will follow a modified Fibonacci schema with 3-6 subjects at each dose level until a Recommended Phase 2 Dose (RP2D) is determined. The cohort will then be expanded to a maximum of 12 patients to more fully evaluate the RP2D.