IGF::OT::IGF BCR-ABL 1 RT-QPCR TEST FOR IMPROVED MONITORING OF CML

由于BCR-ABL 1相对表达水平的预后价值，接受酪氨酸激酶抑制剂治疗的CML患者每3个月进行一次监测。早期识别治疗失败允许修改，可能挽救生命，治疗策略。本项目的长期目标是获得BCR-ABL 1 RT-qPCR检测作为FDA批准的IVD的监管批准，以提高准确、可靠检测的可用性，从而监测CML患者的残留疾病。批准涉及内部检测活动、多个批次的生产以及多个独立临床实验室的精密度和准确度检测。Asuragen已与FDA就研究设计、临床终点和统计方法达成一致，并将其纳入本提案。在I期和II期合同之间的过渡期间，承包商支持并开展了一项临床研究，以验证BCR-ABL 1 RT-qPCR检测。 在II期研究中，Asuragen将进行必要的研究，以开发和临床验证BCR-ABL 1次要断点（e1 a2）检测，以及主要和次要断点BCR-ABL 1 RT-qPCR检测的生产工艺改进和平台扩展研究。

CML patients undergoing tyrosine kinase inhibitor treatment are monitored every 3 months because of the prognostic value of BCR-ABL1 relative expression levels. Early identification of treatment failure allows for a revised, potentially life-saving, therapeutic strategy. The long-term objective of this project is to gain regulatory approval for the BCR-ABL1 RT-qPCR Test as an FDA-cleared IVD to improve the availability of accurate, reliable testing to monitor residual disease in patients with CML. Clearance involves internal testing activities, manufacturing of multiple lots and precision and accuracy testing at multiple independent clinical laboratories. Asuragen has achieved agreement with the FDA on the study design, clinical endpoints and statistical approaches, which are integrated into this proposal. In the interim between the Phase I and Phase II Contracts, the contractor had supported and performed a clinical study for the validation of the BCR-ABL1 RT-qPCR Test. In Phase II, Asuragen will perform essential studies to develop and clinically validate a test for BCR-ABL1 minor breakpoint (e1a2) as well as studies for manufacturing process improvements and platform expansion studies for both the Major and minor breakpoint BCR-ABL1 RT-qPCR Tests.