A Multicentre, Randomised, Double-blind, Parallel Group, Placebo-controlled, Time-to-first Asthma Exacerbation Phase III Efficacy and Safety Study of Benralizumab in Paediatric Patients with Severe Eosinophilic Asthma (DOMINICA)

Date Added
June 6th, 2023
PRO Number
Pro00126684
Researcher
Kelli Williams

List of Studies


Keywords
Allergy, Asthma, Pediatrics
Summary

The main purpose of this study is to see if taking benralizumab is effective in treating Severe Eosinophilic Asthma in patients 6 to 17 years old compared with placebo. Benralizumab is administered through a subcutaneous injection (shot). Participation in the study is divided into two parts. The first part will last up to 16 weeks or until an asthma attack or flare is experienced. The second part will last up to two years.

Institution
MUSC
Recruitment Contact
Natalie Naylon
8437925824
naylon@musc.edu

Phase II Trial of Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

Date Added
May 2nd, 2023
PRO Number
Pro00125254
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Brain, Pediatrics
Summary

This study is for patients that have been diagnosed with relapsed/refractory neuroblastoma. The investigational drug given is eflornithine (DFMO) along with etoposide. DFMO is the investigational drug being used along with etoposide for treatment of neuroblastoma. Participants will undergo a number of standard tests and research-related procedures before being able to enroll in this study. Some risks include but are not limited to: fewer red and white blood cells, diarrhea, abdominal pain, loss of appetite, skin rash, seizure, difficulty swallowing and blurred vision. Participants can expect to be on this study for approximately 2 years. Participants will then be followed for up to 5 years after study completion.

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

An Open-Label Extension Study of BPN14770 in Subjects with Fragile X Syndrome

Date Added
May 1st, 2023
PRO Number
Pro00125951
Researcher
Caroline Buchanan

List of Studies

Keywords
Genetics, Pediatrics, Rare Diseases
Summary

This is an OLE study for subjects completing from one of two double-blind clinical
trials. Subjects must have completed the Week 13 visit from one of these two parent
clinical trials to be eligible for this OLE.

Institution
Self Regional Healthcare
Recruitment Contact
Caleb Hinzman
864-672-6912
chinzman@ggc.org

Self-Management for Youth Living with Sickle Cell Disease: SMYLS Multi-site Trial

Date Added
April 4th, 2023
PRO Number
Pro00127137
Researcher
Shannon Phillips

List of Studies


Keywords
Adolescents, Blood Disorders, Minorities, Pain, Pediatrics, Rare Diseases
Summary

The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms. 272 teens and adults with SCD will be enrolled in this study which is being conducted at the Medical University of South Carolina in Charleston SC., East Carolina University in Greenville NC., University of Miami in Miami FL., and the University of Alabama in Birmingham AL.

Institution
MUSC
Recruitment Contact
Shannon Phillips
843-792-9379
phillipss@musc.edu

A Phase 3b/4 Randomized, Open-label, Efficacy Assessor Blinded Study, Comparing the Safety and Assessor Blinded Efficacy of Upadacitinib to Dupilumab in Subjects with Moderate to Severe Atopic Dermatitis (Level-Up)

Date Added
March 22nd, 2023
PRO Number
Pro00125074
Researcher
Lara Wine Lee

List of Studies


Keywords
Children's Health, Pediatrics, Skin
Summary

This is a global, Phase 3b/4, randomized, open-label, efficacy assessor-blinded, multi-center study that will evaluate upadacitinib compared with dupilumab, as monotherapy, in adolescents and adult subjects who have inadequate response to systemic therapy. The study will consist of a 35-day Screening Period; Period 1, a 16-week randomized, open-label, efficacy assessor blinded treatment period for all subjects, and a 30-day or 12-week follow-up visit for subjects on upadacitinib and dupilumab respectively, who will not enter the Period 2; Period 2, a 16-week open-label, efficacy assessor blinded extension period for those subjects with a < EASI 75 response at Week 16 (total duration 32 weeks) and a 30-day follow-up visit.

Institution
MUSC
Recruitment Contact
Mikayla DiDonato
8438762281
didonato@musc.edu

Examining Provider and Caregiver Perceptions of an Automated Clinical Support Tool to identify Child Risk in Primary Care

Date Added
February 12th, 2023
PRO Number
Pro00125560
Researcher
Hannah Espeleta

List of Studies

Keywords
Children's Health, Pediatrics
Summary

This study wants to understand how we can better identify children who have experienced or are at risk to experience child maltreatment. We will be interviewing caregivers of children and primary care providers (nurses, pediatricians, social workers) to understand their perspectives on screening for child safety risks in primary care settings. Interviews with providers will also focus on their feedback on a new tool to designed to detect child safety risks within health care settings. Findings will help inform best practice in integrated health care settings.

Institution
MUSC
Recruitment Contact
Hannah Espeleta
843-792-6098
espeleta@musc.edu

Abdominal ultrasound in staging pediatric inflammatory bowel disease as compared to endoscopy at diagnosis.

Date Added
February 12th, 2023
PRO Number
Pro00123592
Researcher
Hamza Khan

List of Studies

Keywords
Bowel, Children's Health, Inflammatory Bowel Disease, Pediatrics
Summary

The purpose of this study is to evaluate the findings of Abdominal Ultrasound (AU) and to determine the effectiveness and safety in diagnosing Inflammatory Bowel Disease (IBD) in children. An AU provides a cost-effective, radiation-free, noninvasive method while a colonoscopy, endoscopy, and biopsy are invasive procedures. A secondary aim of the study is to compare the AU findings with blood markers and advanced imaging, when available. The department of Radiology will be conducting this study at the Shaw Jenkins Children's Hospital and MUSC Children's Health R. Keith Summey Medical Pavilion.

Institution
MUSC
Recruitment Contact
Samuel Bidwell
8437922305
bidwells@musc.edu

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Assess the Efficacy, Safety and Pharmacokinetics of Lebrikizumab Compared to Placebo in Participants 6 Months to <18 Years of Age with Moderate-to-Severe Atopic Dermatitis

Date Added
December 8th, 2022
PRO Number
Pro00123135
Researcher
Lara Wine Lee

List of Studies


Keywords
Children's Health, Pediatrics, Skin
Summary

This study aims to evaluate the efficacy and safety of lebrikizumab when used in combination with topical corticosteroid (TCS) treatment, compared with placebo, in pediatric participants with moderate-to-severe atopic dermatitis. Participants found to be eligible according to all of the study entry criteria will be randomly assigned in a 2:1 ratio to receive either lebrikizumab or placebo. This study can last up to 32 weeks, with 4 study periods. Screening Period: up to 4 weeks (≤30 days), TCS Standardization Period: 2 weeks, Treatment Period: 16 weeks, Post-Treatment Safety Follow-up Period: 12 weeks.

Institution
MUSC
Recruitment Contact
Mikayla DiDonato
8438762281
didonato@musc.edu

Low-Interventional Cohort Study of Myocarditis/Pericarditis Associated With COMIRNATY in Persons Less Than 21 Years of Age

Date Added
November 16th, 2022
PRO Number
Pro00124654
Researcher
Kimberly McHugh

List of Studies


Keywords
Coronavirus, Heart, Pediatrics
Summary

This is a low-interventional cohort study to determine cardiac and non-cardiac long-term outcomes of persons <21 years of age with myocarditis/pericarditis after the administration of COMIRNATY, compared with similarly aged persons with myocarditis/pericarditis associated with COVID-19, including MIS-C.

Institution
MUSC
Recruitment Contact
Layla Al Sarraf
843-876-5203
alsarral@musc.edu

Identification of Differences in Verb Learning between Late Talkers and Typically Developing Children

Date Added
October 4th, 2022
PRO Number
Pro00123522
Researcher
Sabrina Horvath

List of Studies

Keywords
Language, Pediatrics, Speech Disorders
Summary

This study explores the best way to teach two-year-old toddlers new verbs, and whether there are differences in what is best between late talkers and typically developing children. In a series of two, one-hour visits, children will watch videos on an eye-tracker, which will capture their face and gaze patterns. This data will be analyzed to see how children are making sense of what they are hearing. In one task, we ask whether it is better for children to hear a new verb before they see the action it denotes, or whether it is better to see the new action before hearing the verb. In the second task, we consider how quickly children are able to make sense of the language they hear, and whether this has any relationship to how they learn new verbs (Task 1). Results will help shape new clinical interventions for late talkers.

Institution
MUSC
Recruitment Contact
Sabrina Horvath
843-792-9363
horvaths@musc.edu



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