Long QT Syndrome Screening in Newborns

Long QT Syndrome Clinical Trial

Official title: ECG Device for Long QT Syndrome Screening in Newborns

Status Recruiting

Clinical Trial Summary

The purpose of this project is to test a new, very compact, 12-lead ECG device as a way to detect long QT syndrome (LQTS) in infants. The device -- called QTScreen -- was developed in Phase I of this project.

In Phase II, the goals are to test the capacity of the device for LQTS screening in newborns and to obtain prevalence data on LQTS in California.

The 4 main objectives are:

1. To validate the capacity of QTScreen for LQTS screening in newborns.

2. To determine the extent to which parents are able to use QTScreen on their babies at home.

3. To survey end-user experience and opinions.

4. To estimate the LQTS prevalence in California.

Clinical Trial Description

Long QT syndrome (LQTS) is a genetic disorder characterized by a prolonged QT interval on the ECG and occurrence of syncope, ventricular arrhythmias, and sudden death. LQTS is a major cause of sudden death in infants, children, and young adults. Treatment by β-blockers and/or placement of an implantable cardioverter defibrillator (ICD) are effective in preventing sudden death, if the diagnosis of LQTS is made early.

The efficacy of ECG screening of newborns for LQTS has been demonstrated in Italy. Furthermore, within the national healthcare system in Italy, universal ECG screening in newborns (<30 days old) is cost-effective. The U.S. debate for universal LQTS screening started in the mid-2000's. In a 2007survey completed by North American pediatric cardiologists, 27% favored optional ECG screening of newborns, whereas 11% supported mandatory screening (363 responses, 35% response rate). There may be stronger support for universal screening, now that more data are available.

Obtaining a good standard 12-lead EKG on a neonate in the clinic is difficult and time consuming. If an ECG device for newborn screening were readily available, reliable, easy to use, and cost-effective, then universal screening of all 4 million newborns in the U.S. each year would identify 2,000 infants with LQTS who are at risk for sudden death, assuming the prevalence is the same as in Italy. Perhaps more than 300 deaths per year from SIDS could be prevented, as well as many more sudden deaths in children, adolescents, and young adults.

During Phase I of this project, a new device was developed to meet this clinical need (called QTScreen). In Phase II, the aim is to test the capacity of QTScreen for LQTS screening in newborns and the feasibility of home screening by parents in an ethnically diverse population of Northern and Southern California. The results of this clinical trial may validate the device as a screening tool for LQTS, and also provide important scientific data for supporting newborn screening for LQTS in the U.S.

The 4 main objectives are:

1. To validate the capacity of QTScreen for LQTS screening in newborns. This will be a clinical trial on 4,000 newborns, conducted at the Los Angeles Biomedical Research Institute (LABioMed).

2. To determine the extent to which parents are able to use QTScreen on their babies at home.

3. To survey end-user experience and opinions. The target users of QTScreen are parents. Participating parents will be asked to complete a survey, to obtain data for further development of the device.

4. To estimate the LQTS prevalence in California. To date, the only population-based estimate of LQTS prevalence is 1 in 2,000 in Italy. In a recent study in Japan, 4,285 1-month-old infants had ECG screening. The LQTS prevalence was not reported, because only 10% of subjects with QTc values >450 msec had gene testing. There has been no population-based study of LQTS in the US. In a study of 707 children with hearing loss in California, 2 subjects had potassium voltage-gated channel KQT-like subfamily member 1 (KCNQ1 potassium channel) mutations, truncation or splicing. This trial will provide data on the LQTS prevalence in California.

Study Sites:

Study procedures will be conducted at subjects' homes. Subjects will be recruited from LA BioMed at Harbor-UCLA Medical Center, Providence Little Company of Mary Medical Center-Torrance, Santa Clara Valley Medical Center, and St. Francis Medical Center. Approval from each of the recruitment sites will be obtained prior to commencing recruitment.

Consent:

Consent will be sought from one parent only. Parents will be given the opportunity to thoroughly read the consent form and to ask questions.

Risk/Benefit:

ECG is a routine test performed on infants, children and adults every day in the hospital for many years with no problems or adverse events. The QTScreen system used in this study is a simplified way of performing an ECG test on infants. Some subjects may undergo genetic testing as part of this research and there are some risks associated with genetic testing, such as emotional and confidentiality.

There is a very small risk of infection or rash related to the procedure involved in ECG testing. The subject may feel some discomfort from the sticky patches placed on and removed from his/her chest. Discomfort will be minimized by using an adhesive remover wipe which is commercially available and used by hospitals. Although infection is very unlikely to occur, rules and procedures of the nursery and clinic will be followed. This includes cleaning the ECG equipment and sanitizing it using a disinfecting wipe before and after each use to prevent infection.

The effects of drawing blood are usually pain, bleeding and/or a bruise where the needle is inserted. Occasionally the area around the vein may swell. Serious complications such as a blood clot or infection may occur but these are rare. Some people feel faint when having blood drawn. Necessary precautions, such as gloves and proper sterilization, will be taken to minimize pain and infection. A small amount of local anesthetic cream may be placed on the child's forearm.

Appropriate precautions will be used to minimize the risks associated with this project and feel that in general the risks associated with this project are low.

The information gained from this study will help to find ways to possibly simplify the ECG test. This research study may also lead to a better understanding of LQTS which can provide important insight for future treatments and research. Therefore the general feeling is that the benefits of this project outweigh the risks. ;

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Screening

Related Conditions & MeSH terms

• Long QT Syndrome
• Syndrome

• NCT number: NCT02412709
• Study type: Interventional
• Source: QT Medical, Inc.
• Contact: Sandra Sedano, M.S.
• Phone: 424-558-3500
• Email: ssedano@qtmedicalinc.com
• Status: Recruiting
• Phase: Phase 2
• Start date: June 2015
• Completion date: March 2017