A Phase 2, Open-label, Uncontrolled, Single-dose Study to Evaluate the Safety and Tolerability, Pharmacokinetics and Occurrence of Antidrug Antibody for Nirsevimab in Immunocompromised Children < 24 Months of Age.

Date Added
August 10th, 2021
PRO Number
Pro00113136
Researcher
Andrew Atz

List of Studies


Keywords
Infant, Infectious Diseases
Summary

Respiratory syncytial virus (RSV) is the most common cause of lower respiratory tract infection (LRTI) among infants and young children, resulting in annual epidemics worldwide. Children with congenital or acquired immunodeficiencies, transplant recipients, and those receiving immunosuppressive therapy are at increased risk for severe RSV-associated disease resulting in prolonged hospitalizations, admissions to the intensive care unit (ICU), and the need for mechanical ventilation. Nirsevimab is not a vaccine, but a monoclonal antibody. Monoclonal antibodies are laboratory-made proteins that mimic the immune system's ability to fight off harmful antigens such as viruses. Nirsevimab is being developed as a cost-effective long acting product to protect all infants from RSV disease in a once-per-RSV-season dosing as opposed to the only current FDA approved RSV specific monoclonal antibody which requires 5 monthly injections. Approximately 100 subjects will be enrolled. Subjects will be followed for approximately 1 year after a single dose of nirsevimab and will have 4 in person visits and numerous telephone check ins during the year of follow up.

Institution
MUSC
Recruitment Contact
Megan Bickford
843-876-3394
bickfome@musc.edu

Combining taVNS with early CIMT to improve health outcomes of infants

Date Added
May 4th, 2021
PRO Number
Pro00109558
Researcher
Dorothea Jenkins

List of Studies


Keywords
Brain, Infant, Movement Disorders, Physical Therapy, Stroke Recovery
Summary

Newborns who are born premature or suffer brain injury at birth are at risk for motor problems that may cause weakness in reaching and grasping on one side of the body. In older children, therapists may use a hand mitt and restraint for the stronger arm, to encourage use of the weaker side, called constraint-induced movement therapy (CIMT). Even with the high intensity therapy of CIMT, it typically takes between 40-120 hours total treatment time for most children to improve their motor skills. A non-invasive form of nerve stimulation, transcutaneous auricular vagus nerve stimulation (taVNS), stimulates a nerve by the ear that enhances learning motor skills. The purpose of this study is to evaluate the safety and effectiveness of taVNS to improve motor skills when paired with CIMT in infants with one-sided weakness at 6-18months of age.

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

A randomized, double blind, parallel-group, placebo controlled study to evaluate the efficacy and safety of IBP-9414 in premature infants 500-1500g birth weight in the prevention of necrotizing enterocolitis – The Connection study

Date Added
February 23rd, 2021
PRO Number
Pro00105738
Researcher
Carol Wagner

List of Studies


Keywords
Children's Health, Infant, Pediatrics
Summary

Babies that are born extremely prematurely are at higher risk of developing a serious inflammatory condition, called necrotizing enterocolitis (NEC), that damages parts of the intestines. It is the most common cause of illness of the intestinal tract in premature infants in the first few weeks of life. It is a very serious condition that can lead to death in up to half of affected infants.The main purpose of this study is to see if IBP-9414 ( a probiotic) can prevent NEC and if it is safe and tolerable.

IBP-9414 (Lactobacillus reuteri) is a live bacterial therapy given to infant's through a feeding tube or it is given by mouth once daily starting at or before 48 hours of life. IBP-9414 will be given daily until the infant reaches 34 weeks + 6 days Post-Menstrual Age (PMA) (the the baby's gestational age at birth plus the postnatal days of life), or until treatment is permanently discontinued if earlier.

Institution
MUSC
Recruitment Contact
Della MacNicholas
843-792-8385
macnichd@musc.edu

A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Transplantation & Cellular Therapy Consortium (PTCTC) PIDTC "CSIDE" Protocol (Conditioning SCID Infants Diagnosed Early) PTCTC NMD 1801

Date Added
November 5th, 2019
PRO Number
Pro00091878
Researcher
Michelle Hudspeth

List of Studies


Keywords
Cancer, Infant, Pediatrics
Summary

This study is for children age 0-2 who have been diagnosed with severe combined immunodeficiency (SCID). In this study, participants will be randomized (select by chance) into groups that will decide the dose of chemotherapy they will receive, receive chemotherapy prior to a blood stem cell transplant, and have blood drawn for research tests. The purpose of this research study is to find out if lower doses of a chemotherapy drug called busulfan before stem cell transplant can help patients with SCID, and to see if the device the CliniMACS® is effective in preparing donor stem cells before the transplant. Participants can expect to be in this study for up to 3 years.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinical-trials@musc.edu

A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of MEDI8897, a Monoclonal Antibody With an Extended Half-life Against Respiratory Syncytial Virus, in Healthy Late Preterm and Term Infants (MELODY)

Date Added
September 24th, 2019
PRO Number
Pro00090069
Researcher
Andrew Atz

List of Studies


Keywords
Infant, Infectious Diseases, Pediatrics
Summary

The purpose of this study is to evaluate how effective MEDI8897 is at preventing lung disease caused by Respiratory Syncytial Virus (RSV) and to evaluate the safety and tolerability of MEDI8897 in healthy infants compared with placebo. A placebo is a saline solution that looks like the study drug but it does not contain the active ingredient.

Institution
MUSC
Recruitment Contact
Kalyan Chundru
8437921213
choudhar@musc.edu

Predicting the Emergence of Social Communication Across the First Three Years of Life

Date Added
November 9th, 2018
PRO Number
Pro00081992
Researcher
Jessica Bradshaw

List of Studies

Keywords
Autism, Children's Health, Infant, Language, Pregnancy
Summary

Early intervention for infants and toddlers with or at-risk for autism spectrum disorder can promote developmental skills and improve lifelong outcomes. Yet, many children with ASD are not diagnosed until after age 3. In order to improve early detection of ASD, we are investigating very early predictors of social communication challenges in infants as young as 1 week to 6 months of age.

This research study examines how the development of attention and motor skills in the first year of life is associated with the emergence of social and communication skills in three groups of infants: infants who are first born or who have a sibling with no developmental delays, infants who have an older sibling diagnosed with autism spectrum disorder, and infants who were born preterm.

Institution
USC
Recruitment Contact
Emma Platt
803-993-8356
esdilab@mailbox.sc.edu

National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children with Complex Congenital Heart Disease

Date Added
June 23rd, 2016
PRO Number
Pro00056522
Researcher
Frances Woodard

List of Studies


Keywords
Cardiovascular, Children's Health, Heart, Infant, Non-interventional
Summary

Transforming health care and outcomes for children with rare diseases is difficult within the current health care system. There is great variation in care delivery, inadequate and slow application of existing evidence, and ineffective use of available data to generate new knowledge. Individual care centers have inadequate numbers of patients for robust learning and improvement. In order to redesign the system, changes must take place at multiple levels, including the patient and family, clinician, practice and the network. The purpose of this project is to design, develop, and test further refinements to an improvement and research network focused on HLHS, the most severe congenital heart defect, and to use a registry to simultaneously improve clinical care, redesign care delivery systems and to conduct quality improvement, health services, outcomes, and comparative effectiveness research. The purpose of this initiative, specifically, is to improve care and outcomes for infants with HLHS by: 1) expanding the established NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, 2) improving implementation of consensus standards, tested by teams, into everyday practice across pediatric cardiology centers, and 3) engaging parents as partners in improving care and outcomes. We utilize a quality improvement methodology, known as the adapted learning collaborative model, which expedites the implementation of tools and strategies that facilitate changes such as systematic care coordination, cardiovascular monitoring, and nutritional monitoring into every day practice. The NPC-QIC registry is used to document the impact of these changes on various care processes and outcomes (e.g., mortality rate, readmissions, and weight gain).

Institution
MUSC
Recruitment Contact
Frances Woodard
843-792-3292
klinefl@musc.edu