ECG Device for LQTS Screening in Newborns, Phase II

用于新生儿 LQTS 筛查的心电图设备，第二阶段

基本信息

批准号：
8781751
负责人：
RUEY-KANG R CHANG
金额：
$ 98.36万
依托单位：
QT MEDICAL, INC.
依托单位国家：
美国
项目类别：
财政年份：
2014
资助国家：
美国
起止时间：
2014-08-18 至 2016-12-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8781751
关键词：
Accounting Adopted Affect Age Anxiety Award California Cardiac Cessation of life Child Clinical Clinical Research Clinical Trials Cost Effectiveness Analysis Data Development Device Designs Devices Early Diagnosis Effectiveness Electrocardiogram Electrodes Epidemiology Event Funding Genes Genetic screening method Goals Hearing Infant Investments Lead Learning Logistics Long QT Syndrome Marketing Measurement Morbidity - disease rate Mutation National Heart, Lung, and Blood Institute Neonatal Screening Newborn Infant Nucleotides Outcome Parents Phase Population Prevalence Production Publishing Research Risk Sampling Schedule Screening Result Secure Services Small Business Technology Transfer Research Specific qualifier value Sudden Death Sudden infant death syndrome Testing United States National Institutes of Health Variant base commercialization cost cost effectiveness design genetic variant mortality phase 1 study prevent public health relevance screening success tool user-friendly young adult

项目摘要

DESCRIPTION (provided by applicant): The congenital long QT syndrome (LQTS) is thought to occur in 1 in 2,000 infants and cause 10-15% of deaths from SIDS (sudden infant death syndrome). Electrocardiograms (ECGs) can detect LQTS before fatal cardiac events happen. In our Phase I STTR project, we successfully developed and tested an ECG device designed for LQTS screening in newborns. This device, now called QTScreen, was produced exactly as specified in our Phase I proposal. Both Specific Aims in Phase I were completed and all milestones were reached on schedule. In this Phase II proposal, our goal is to demonstrate the effectiveness of the QTScreen for LQTS screening in newborns in the U.S., so that the QTScreen can become a commercialized product and service. To achieve our goal, we propose a clinical trial to validate the QTScreen device for LQTS screening, assess strategies for newborn screening, and define the epidemiology of LQTS in the Southern California -- and thus the potential market. In Aim 1, we will perfect the QTScreen for use in a large clinical trial. The refinements will enhance QTScreen function and make it more user-friendly. The design will comply with FDA standards and allow scalable production. In Aim 2, we will show the effectiveness of QTScreen for LQTS screening in newborns in Southern California. We will conduct a clinical trial on 4,000 babies. Results will establish the prevalence of LQTS in Southern California, and validate QTScreen as a newborn screening tool. In Aim 3, we will identify strategies to understand and minimize false positive screens. False positive results cause unneeded parental anxiety and raise costs due to expensive genetic testing. Our strategies to minimize false positive results are to: (1) identify the optimal QTc threshold for rescreening and gene testing; and (2) identify common single nucleotide variants that affect QT intervals in newborns, apart from LQTS mutations. Expected outcomes of Phase II are a validated QTScreen device for newborn screening, a marketable product ready for FDA filing, and valuable data on LQTS occurrence in the U.S. With Phase II results, we will seek investments and apply for an NHLBI Phase IIB Small Market Award. In Phase IIB, we will propose a multi-center trial for nationwide newborn LQTS screening in the U.S. and cost effectiveness analyses.

描述（由申请人提供）：据认为先天性长QT综合征（LQT）发生在2,000名婴儿中的1个，并导致SIDS死亡的10-15％（猝死综合症）。心电图（ECGS）可以在致命心脏事件发生之前检测LQT。在我们的I阶段STTR项目中，我们成功开发并测试了一种用于LQTS筛查的ECG设备。该设备现在称为QTScreen，完全按照我们的I阶段建议中的指定生产。第一阶段的两个具体目标都已完成，并按计划达到所有里程碑。在这一第二阶段的提案中，我们的目标是证明QTSCREEN在美国新生儿筛查LQTS筛查的有效性，以便QTScreen可以成为商业化的产品和服务。为了实现我们的目标，我们提出了一项临床试验，以验证LQTS筛查的QTSCREEN设备，评估新生儿筛查的策略，并定义南加州LQT的流行病学，从而成为潜在市场。在AIM 1中，我们将完善用于大型临床试验的QTSCREEN。这改进将增强QTScreen功能，并使其更加用户友好。该设计将符合FDA标准，并允许可扩展生产。在AIM 2中，我们将展示QTScreen在南加州新生儿进行LQTS筛查的有效性。我们将对4,000名婴儿进行临床试验。结果将确定南加州LQT的患病率，并验证QTScreen作为新生儿筛查工具。在AIM 3中，我们将确定理解和最大程度减少误报屏幕的策略。假阳性结果会导致不需要的父母焦虑，并因昂贵的基因检测而提高成本。我们最大程度地减少假阳性结果的策略是：（1）确定重新分组和基因测试的最佳QTC阈值；（2）确定除LQTS突变外影响QT间隔的常见单核苷酸变体。 II期的预期结果是一种验证的QTSCREEN设备，用于新生儿筛查，可销售的产品可用于FDA备案，以及有关美国LQT发生的有价值数据，并获得了II期结果，我们将寻求投资并申请NHLBI IIB阶段IIB小型小型市场。在IIB期中，我们将提议在美国进行全国新生儿LQTS筛查和成本效益分析的多中心试验。