Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

Date Added
April 6th, 2021
PRO Number
Pro00106369
Researcher
Alexandra Ritter

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 Ritter
7577776673
ritteral@musc.edu

Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

Date Added
February 17th, 2021
PRO Number
Pro00107343
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Cancer, Pediatrics
Summary

This study is for patients that have been diagnosed with a hepatic malignancy. The purpose of this research is to reduce therapy associated toxicity for patients with non-metastatic hepatoblastoma (HB) and hepatocellular carcinoma (HCC) without adversely affecting long term outcomes. Participants can expect to receive treatment for up to 3 months. After treatment, participants will be followed for up to 10 years.

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

67Cu-SARTATE Peptide Receptor Radionuclide Therapy Administered to Pediatric Patients With High-Risk Neuroblastoma: A Multi-center, Dose-escalation, Open-label, Non-randomized, Phase 1-2a Theranostic Clinical Trial

Date Added
February 9th, 2021
PRO Number
Pro00105726
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Brain, Pediatrics
Summary

This study is for participants that have been diagnosed with a high-risk neuroblastoma that cannot be treated or did not improve with existing therapies, or the cancer came back after treatment with existing therapies. This study involves investigational drugs called 64Cu-SARTATE and 67Cu-SARTATE. The investigational drugs will be given as an IV injection. The study is divided into 2 parts: The Dose Escalation Phase and the Cohort Expansion Phase. The phase that participants will enroll to will depend on when they enter the study. The age range for participants is from 12 months to 25 years. Participants can expect to be in this study for approximately 14 months.

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

Obstructive Sleep Apnea in Pediatric Populations Treated with Growth Hormone Therapy

Date Added
February 4th, 2021
PRO Number
Pro00097104
Researcher
Kristal Matlock

List of Studies


Keywords
Hormones, Pediatrics, Sleep Disorders
Summary

Growth hormone may increase the size of tonsils in the airway. Children who take growth hormone may be at an increased risk for sleep apnea. The goal of this study is to check for sleep problems in children before and after starting growth hormone. This research will also help us know if and when other children who start growth hormone need to be checked for sleep problems.

Institution
MUSC
Recruitment Contact
Kristal Matlock
8437792233
matlock@musc.org

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

An Observational Cohort Study to Determine Late Outcomes and Immunological Responses after Infection with SARS- CoV-2 in Children with and without Multisystem Inflammatory Syndrome (MIS-C)

Date Added
October 6th, 2020
PRO Number
Pro00104091
Researcher
Natasha Ruth

List of Studies


Keywords
Coronavirus, Pediatrics
Summary

We will aim to enroll children who have had a nasal swab test showing infection with SARS-CoV-2, the virus that causes COVID-19, or children with a serious complication of COVID-19 called the Multi-system Inflammatory Syndrome in Children (MIS-C). This study does not offer any treatment for COVID-19 or MIS-C. The purpose of the study is to collect information that will help us understand what happens to children and young adults who get infected with SARS-CoV-2, and how their immune system responds. Most of the information that we will collect is part of usual care for young people who have COVID-19 or MIS-C. In addition, we will do some extra clinical and blood tests. Children and young adults who are enrolled in the study will be followed for one year. Our goal is to enroll up to 250 children and young adults (up to the time of your 21st birthday) from about 20 hospitals in the United States. At our center we plan to enroll about 20 participants.

Institution
MUSC
Recruitment Contact
Ebony Dawkins Mikell
843-876-8692
panacceb@musc.edu

Long-TerM OUtcomes after the Multisystem Inflammatory Syndrome In Children: MUSIC

Date Added
September 30th, 2020
PRO Number
Pro00103636
Researcher
Kimberly McHugh

List of Studies


Keywords
Coronavirus, Pediatrics
Summary

This observational study will determine the spectrum and time course of coronary artery involvement, left ventricular (LV) systolic function, and arrhythmias or conduction system disturbances within the first year from illness onset, and to define associated clinical and laboratory factors in a cohort of MIS-C affected patients.

Institution
MUSC
Recruitment Contact
Ebony Dawkins Mikell
843-876-8692
panacceb@musc.edu

Influenza Vaccine Effectiveness for Preventing Laboratory-Confirmed Severe Influenza-Associated Illness and Understanding Coronavirus Disease 2019 (Overcoming COVID-19) in US Children

Date Added
September 17th, 2020
PRO Number
Pro00102181
Researcher
Elizabeth Mack

List of Studies


Keywords
Coronavirus, Pediatrics
Summary

The Overcoming Cohort study is a studies looking for variations in DNA,for populations less than or 25 years of age, that either protect people from COVID-19 disease or make them more susceptible. The study will sequence patients' entire genome or the smaller group of genes that code for proteins (exome). The study would gain a better understanding of COVID-19 that would improve diagnostic, prevention, and treatment measures.

Institution
MUSC
Recruitment Contact
Ebony Dawkins Mikell
843-876-8692
panacceb@musc.edu

Prospective Non-Interventional Study in Patients with Locally Advanced or Metastatic TRK Fusion Cancer Treated with Larotrectinib

Date Added
September 17th, 2020
PRO Number
Pro00090298
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Pediatrics
Summary

This is an observational study, which means that only information is collected on treatment, examinations or diagnostic tests which are a part of the routine standard of care. The participant is being asked to volunteer because they have been diagnosed with locally advanced or metastatic TRK fusion cancer treated with larotrectinib. The purpose of this study is to learn more about the safety profile and the effectiveness of VITRAKVI® under routine practice conditions.
Participants will be followed for a minimum of 5 years from the time they agree to participate in the study.

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



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