Multicenter Phenotype-Genotype Analysis of Vascular Anomalies and Related Syndromes

Date Added
December 24th, 2021
PRO Number
Pro00116848
Researcher
Lara Wine Lee

List of Studies


Keywords
Genetics, Pediatrics, Skin, Vascular
Summary

The purpose of this research study is to develop a better understanding of the cause and natural history of vascular anomalies and related syndromes. This study is being done in order to develop a better understanding of the cause of vascular anomalies in order to to improve care for people who are affected by these anomalies and related syndromes.

This study is being done at the University of Wisconsin-Madison (UW-Madison) and other sites in North America and Europe. A total of about 1000 people will participate in this study. About 20 – 30 people will take part in the study here at the Medical University of South Carolina.

Institution
MUSC
Recruitment Contact
Chelsea Shope
8437549577
shopec@musc.edu

Tele-PCIT: Internet Delivered PCIT for Young Children with Neurodevelopmental Disorders

Date Added
December 11th, 2021
PRO Number
Pro00114624
Researcher
Rosmary Ros-Demarize

List of Studies


Keywords
ADD/ADHD, Autism, Pediatrics
Summary

The purpose of this research study is to evaluate Parent Child Interaction Therapy (PCIT) delivered via tele-health for young children with a developmental diagnosis (ex. suspected or diagnosed autism, ADHD, global developmental delay, etc.) and disruptive behavior problems. Participants will go through a screening to determine eligibility. Once screening is complete, participants will complete a pre-therapy assessment in clinic, followed by 10 telehealth sessions one time per week, at no cost. Participants will then be asked to complete an in-person post-therapy assessment and follow-up questionnaires will be re-administered 3 months following the completion of therapy. Families will be compensated for their time.

Institution
MUSC
Recruitment Contact
Jessie Montezuma
843-714-1352
autismresearch@musc.edu

THRIVE-ASD: Telehealth Rapid Intervention for Externalizing Behaviors in ASD

Date Added
December 3rd, 2021
PRO Number
Pro00114600
Researcher
Rosmary Ros-Demarize

List of Studies


Keywords
Autism, Children's Health, Pediatrics
Summary

The purpose of this research study is to evaluate Parent Child Interaction Therapy (PCIT) delivered via telehealth for young children with autism spectrum disorder (ASD) and disruptive behavior problems. Participants will go through a screening to determine eligibility. Eligible families who choose to participate will complete 3 in-person visits at MUSC and may receive 10 telehealth therapy sessions, at no cost. Families will be compensated for their time.

Institution
MUSC
Recruitment Contact
Jessie Montezuma
843-714-1352
autismresearch@musc.edu

Immunobiology of Influenza Critical Illness in Young People (PICFLU2)

Date Added
November 24th, 2021
PRO Number
Pro00112536
Researcher
Sara Rhodes Proctor Short

List of Studies

Keywords
Infectious Diseases, Pediatrics
Summary

The Pediatric Intensive Care Influenza Study #2 (PICFLU2) is a multiyear, multicenter prospective observational study in patients aged ≤ 21 years hospitalized in pediatric Intensive Care Units (PICUs) and Stepdown (or intermediate) Care Units (SDUs) in the US designed to evaluate of the immunobiology of influenza virus-related critical illness in young hosts.

Institution
MUSC
Recruitment Contact
Lauren Wakefield
843-792-0603
wakefila@musc.edu

A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518) in Children and Young Adults With Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)

Date Added
November 23rd, 2021
PRO Number
Pro00116587
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Pediatrics
Summary

This study is for patients who have been diagnosed with relapsed or refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL). The overall goal of this study is to find out what effect, good and/or bad, the drug inotuzumab ozogamicin has on children and young adults with relapsed or refractory B-ALL. Participants can expect to be on this study for up 2 months and followed for up to 5 years.

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

A 52-week, open-label, single arm study to investigate the efficacy and safety of mepolizumab SC in participants aged 6 to 17 years with hypereosinophilic syndrome.

Date Added
September 8th, 2021
PRO Number
Pro00113422
Researcher
Kelli Williams

List of Studies


Keywords
Adolescents, Allergy, Pediatrics
Summary

The purpose of this study is to see if taking mepolizumab is safe and effective in treating Hypereosinophilic syndrome (HES) in children and adolescent patients ages 6 to 17 years who are receiving standard of care (SoC) therapy. Mepolizumab is administered through a subcutaneous injection (shot). Participation in the study will last for about 64 weeks approximately (1 year and 3 months).

Institution
MUSC
Recruitment Contact
Natalie Naylon
843-792-5824
naylon@musc.edu

Perinatal Arterial Stroke: A Multi-site RCT of Intensive Infant Rehabilitation (I-ACQUIRE)

Date Added
August 26th, 2021
PRO Number
Pro00111341
Researcher
Cynthia Dodds

List of Studies


Keywords
Brain, Infant, Movement Disorders, Pediatrics, Physical Therapy, Rehabilitation Studies, Stroke, Stroke Recovery
Summary

Constraint-induced movement therapy (CIMT) is the most efficacious treatment for children with hemiparesis from a perinatal arterial stroke but instead, weekly low-dose OT and/or PT is typical. The aims of this study are to compare 2 high doses of treatment to usual care in helping infants improve skills on the hemiplegic hand/arm and to improve bimanual activities. In addition, the association with gross motor, language and cognition will be explored.

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

Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

Date Added
April 6th, 2021
PRO Number
Pro00106369
Researcher
Chelsea Shope

List of Studies

Keywords
Adolescents, Pediatrics, Skin, Vascular
Summary

In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.

Institution
MUSC
Recruitment Contact
Alexandra Richmond
7577776673
ritteral@musc.edu

A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy

Date Added
December 22nd, 2020
PRO Number
Pro00106138
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Leukemia, Pediatrics
Summary

This study is for patients that have newly diagnosed High-Risk B-ALL, Risk-Adapted Post-Induction therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy. The treatment involves medicine called chemotherapy, which fights cancer. Some patients may also need radiation therapy depending on whether the cancer has spread to the brain and spinal fluid, or the testes for males. The investigational drug on this study is inotuzumab ozogamicin. Participants can expect to be on this study for a little over 2 years and followed for up to 10 years.

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

Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)

Date Added
November 19th, 2020
PRO Number
Pro00105634
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Pediatrics
Summary

This study is for patients that have been diagnosed with Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT). The treatment involves cancer fighting medicine called chemotherapy plus radiation therapy and/or surgery. Participants can expect to be on this study for up to 10 months and be followed by the treatment team for up to 5 years.

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



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