A multicenter safety study of unlicensed, investigational cryopreserved cord blood units (CBUs) manufactured by the National Cord Blood Program (NCBP) and provided for unrelated hematopoietic stem cell transplantation of pediatric and adult patients Save

Date Added
May 6th, 2014
PRO Number
Pro00017387
Researcher
Jennifer Jaroscak
Keywords
Cancer, Children's Health, Men's Health, Minorities, Pediatrics, Transplant, Women's Health
Summary

This study if for patients that have a blood disease and it's been determined that the best option for treating that blood disease is a cord blood transplant. Cord blood (CB) is blood that is taken from the umbilical cord and placenta of healthy newborn babies after childbirth. The cord blood collected from a newborn baby is called a cord blood unit. The United States Food and Drug Administration (FDA) considers cord blood to be a biological drug. These are considered “investigational” products. This study will evaluate the safety of administration of the investigational cord blood units by carefully documenting all infusion-related problems.

Institution
MUSC
Recruitment Contact
Jessica Simons
843-792-2708
simonsjl@musc.edu

The Role of Minimal Residual Disease Testing before and after Hematopoietic Cell Transplantation for Pediatric Acute Myeloid Leukemia Save

Date Added
May 6th, 2014
PRO Number
Pro00011017
Researcher
Michelle Hudspeth
Keywords
Adolescents, Cancer, Cancer/Leukemia, Children's Health, Infant, Pediatrics, Transplant
Summary

This study is being offerred to patients that have acute myelogenous leukemia (AML) which is a cancer of the blood and these patients are going to have a stem cell transplant. This study is looking to determine how accurate two different laboratory tests are at detecting residual, or small numbers of cancer cells in the body before and after stem cell transplant, as well as whether or not results of these two tests show how well a recipient might do after transplant.

Institution
MUSC
Recruitment Contact
Karen Rogers
843-792-2740
rogersk@musc.edu

GECHO Trial: Genetic Determinants of Congenital Heart Disease Outcomes Save

Date Added
May 6th, 2014
PRO Number
Pro00016329
Researcher
Andrew Atz
Keywords
Cardiovascular, Children's Health, Genetics, Heart, Pediatrics
Summary

Genetic Testing of neonates undergoing surgery for single ventricle cardiac defects (SVCD) and other congenital cardiac defects. DNA testing with an aim to identifying genetic factors that aid survival and recovery in SCD patients.

Genetic contribution to patient outcomes: Over the past two decades, there has been dramatic improvement in the survival and functional outcome of patients with all forms of congenital cardiac defects. Yet, there exists significant variability in outcomes that becomes more pronounced as the level of surgical intervention increases and the exposure to adverse hemodynamic conditions becomes more prolonged and more profound. This is particularly noticeable in the SCD patient group where there are continued high levels of mortality and levels of disability that can be quite severe. While these poor outcomes can on occasion be attributed to technical difficulties, complex cardiac anatomy or patient co-morbidities, more commonly they occur in patients that do not superficially appear to be any different than those that will ultimately have excellent outcomes. What is becoming increasingly apparent is that every patient differs in their ability to tolerate the challenges presented by the peri-operative environment. Therefore, significant improvements in outcomes may depend on identification of the genetic factors that place some patients at greater risk and designing treatment protocols to minimize those risks.

Institution
MUSC
Recruitment Contact
Patricia Infinger
843-792-7857
infingep@musc.edu

The Development and Preliminary Reliability Study of the Pediatric Sensory Modality Assessment and Rehabilitation Techniques (SMART) Save

Date Added
May 6th, 2014
PRO Number
Pro00034042
Researcher
Cynthia Dodds
Keywords
Brain, Children's Health, Hearing, Vision/ Eye
Summary

The purpose of this project is to develop and test preliminary reliability of a newly developed pediatric tool, the Pediatric Sensory Modality Assessment and Rehabilitation Techniques (SMART), which will measure cognitive awareness for children with severe brain damage. Five children, between the ages of 3-12 years, with physician-documented severe brain damage and considered medically stable are needed for this study. Recruitment flyers will be disseminated at MUSC and the greater Charleston area community to recruit parents/legal guardians of children with severe brain damage. Once parental/legal guardian and physician consents are in place, participants will be evaluated using the Pediatric SMART 5 times within 10 days. The Pediatric SMART is made up of 5 domains that are olfactory, visual, auditory and vestibular, gustatory, and tactile. Test administration requires approximately 1 hour and can be completed in settings convenient for parents/legal guardians. The potential benefit to study participants is that the findings from the Pediatric SMART may identify sensory and motor strengths of participants. Knowledge of these strengths may enhance current rehabilitation and treatment plans, which may lead to functional improvements; although, this cannot be guaranteed. It is a goal of this study to evaluate the preliminary Pediatric SMART reliability. Once reliability has been substantiated for the Pediatric SMART in further and future study, future children with severe brain injury, being evaluated with the Pediatric SMART, may have rehabilitation and treatments opportunities that are better informed, leading to greater improvement in functional and participatory outcomes.

Institution
MUSC
Recruitment Contact
Cynthia Dodds
843-792-5731
doddscb@musc.edu

Antibiotic Safety in Infants with Complicated Intra-Abdominal Infections (ABS01) Save

Date Added
April 1st, 2014
PRO Number
Pro00030944
Researcher
Andrew Atz
Keywords
Children's Health
Summary

The most commonly used drugs in infants with complicated intra-abdominal infections are not labeled for use in this population because safety and efficacy data are lacking. The proposed study will provide the safety information required for labeling. In addition, the PK of the study drugs has been or will be characterized in premature infants under an IND mechanism.

Institution
MUSC
Recruitment Contact
Hibah Al Nasiri
843-792-7857
alnasiri@musc.edu

COLLABORATIVE LEARNING PROJECT OF PERIOPERATIVE CARE OF INFANTS WITH CONGENITAL HEART DISEASE Save

Date Added
March 21st, 2014
PRO Number
Pro00031239
Researcher
Mark Scheurer
Keywords
Children's Health
Summary

To determine whether a collaborative learning-derived Clinical Practice guideline (CPG) for early postoperative ventilation and extubation results in a higher proportion of subjects extubated early after infant heart surgery. This will be tested in 5 Pediatric Heart Network (PHN) sites. A comparison will be made to 5 other PHN sites who will not undergo specific training in the CPG. Times until extubation after 2 surgeries (tetraoligy of Fallot repair and repair of coarctation of aorta) will be compared between the 5 sites with active CPG learning vs. the 5 control sites. MUSC is being asked to be a control site.

Institution
MUSC
Recruitment Contact
Harvey Frampton
843-792-7857
frampton@musc.edu

Early Power Mobility Study for Children with Neuromuscular Disorders Save

Date Added
March 19th, 2014
PRO Number
Pro00022440
Researcher
Patricia Coker
Keywords
Children's Health, Physical Therapy
Summary

Power wheelchairs are defined as ‘Wheelchairs powered by electricity that provide mobility and body support for individuals with limited ability to walk’ (Shoemaker et al., 2010). For the purposes of this study, the term power mobility is any battery powered equipment used for mobility by children with disabilities. This can include powered ride-on-toys (e.g. Boss car, Cooper car), powered scooter-boards and powered standing devices (e.g. Gobot). This study seeks to expand on the new literature being published on the use of commercially available powered ride-on-toys to assist with the early mobility of children born with movement disorders. Children with neuromuscular impairments have significantly decreased early mobility which greatly affects their opportunities to explore their physical and social environment (Tefft, Guerette, & Furumasu, 1999; Uchiyama, Anderson, & Campos, 2008). The commercially available ride-on-toys could be used in the clinic, home, community, or school settings to improve independent mobility and are a low cost alternative to other mobility devices (Huang & Galloway, 2012). In addition, these devices provide a novel therapeutic tool to examine and/or treat body function level impairments such as cause-effect learning and head/upper extremity/trunk/lower extremity strength and control (Ragonesi & Galloway, 2012). The utilization of early power mobility allows for important early exploration and learning and may have tremendous effect on later perceptual, cognitive, social, and quality of life outcomes for children with movement disorders.

Institution
MUSC
Recruitment Contact
Patricia Coker
843-792-7491
cokerpc@musc.edu

Using Health Status to Measure Care Coordination Efficacy Save

Date Added
March 12th, 2014
PRO Number
Pro00030602
Researcher
Johnette Simmons
Keywords
ADD/ADHD, Adolescents, Aging, Autism, Brain, Cancer, Cerebral Palsey, Children's Health, Dementia, Depression, Diabetes, Geriatrics, Heart, Hypertension/ High Blood Pressure, Mental Health, Obesity, Pediatrics, Rehabilitation Studies, Spinal Cord, Stroke
Summary

On March 23, 2010, President Barack Obama of the United States of America, signed into law the Patient Protection and Affordable Care Act commonly referred to as health reform. This bill calls immediate attention to better coordinated care between providers of health care and ancillary services. While the health reform law is new, the concept of care coordination in the practical sense is familiar to several health and human service disciplines, such as behavioral healthcare, developmental disability systems of care, medical home models, disease management programs, case management programs, long term care Medicaid waivers, and large healthcare organizations. The aim of this study is to understand what consumers and families expect from care coordination programs and services, and how programs should be designed to ensure the most benefit

Institution
MUSC
Recruitment Contact
Johnette Simmons
843-576-1408
sinjoh@musc.edu

South Carolina Children's Educational Surveillance Study Save

Date Added
March 4th, 2014
PRO Number
Pro00015374
Researcher
Laura Carpenter
Keywords
Autism, Children's Health
Summary

We will conduct a screening and direct assessment study in the general population in an area already undergoing monitoring by ADDM. Specifically, all 8-year-olds in a three county region of South Carolina (n=8,000) will be screened for ASD, and those found to be at risk for ASD will be invited to participate in an in-depth diagnostic assessment. Evidenced-based strategies will be implemented to maximize participation in both the screening and diagnostic phases of the study to improve the accuracy of the findings. ASD prevalence estimates will be calculated using the number of children aged 8 years residing in the study area as the denominator, and the number of children identified as cases as the numerator, with adjustments made for missing data from nonparticipants. Prevalence estimates will be provided using both traditional ADDM and SUCCESS methods. Factors contributing to
differences in ASD prevalence estimates by methodology (e.g. sex, race/ethnicity, SES, previous diagnoses, behaviors, degree of impairment, co-morbidities) will be examined. Additionally we will compare prevalence using the DSM-Iv and DSM-5 criteria.

Institution
MUSC
Recruitment Contact
Walter Jenner
843-532-4992
jennerw@musc.edu

Pharmacokinetics of Multiple Dose Methadone in Children Save

Date Added
January 7th, 2014
PRO Number
Pro00029787
Researcher
Andrew Atz
Keywords
Children's Health, Drug Studies, Pediatrics
Summary

Selection of the correct drug dose is the most important decision in assuring optimal pharmacotherapy. Defining an optimal regimen requires a clear understanding of the drug’s PK, pharmacodynamics (PD), and for methadone, pharmacogenomic profiles. Understanding these characteristics for drugs used in children is imperative to determine optimal dose regimens across the pediatric age continuum.

Institution
MUSC
Recruitment Contact
Hibah Al Nasiri
843-792-0080
alnasiri@musc.edu