ECG Device for LQTS Screening in Newborns, Phase II

DESCRIPTION provided by applicant The congenital long QT syndrome LQTS is thought to occur in in infants and cause 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 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 we will show the effectiveness of QTScreen for LQTS screening in newborns in Southern California We will conduct a clinical trial on babies Results will establish the prevalence of LQTS in Southern California and validate QTScreen as a newborn screening tool In Aim 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 identify the optimal QTc threshold for rescreening and gene testing and 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 PUBLIC HEALTH RELEVANCE The long QT syndrome LQTS is a major cause of sudden death in infants children and young adults In our Phase I STTR project we successfully developed and tested an ECG device QTScreen designed specifically for LQTS screening in newborns In Phase II our goal is to prove the effectiveness of the QTScreen for LQTS screening in newborns in the U S so that QTScreen will be a marketable product and service for commercialization as a way to prevent deaths due to LQTS