A feasibility and Randomized phase 2/3 sutdy of the VEFGR2/MET inhibitor Cabozanitinib in combination with cytotoxic chemotherapy for newly diagnosed osteosarcoma

Date Added
June 2nd, 2026
PRO Number
Pro00150183
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Cancer, Children's Health, Pediatrics
Summary

This study is recruiting pediatric patients who have been diagnosed with osteosarcoma. Osteosarcoma is the most common primary bone malignancy of childhood and adolescence. The goal of the study is to determine the feasibility of adding cabozantinib to standard MAP (high dose methotrexate, doxorubicin, and cisplatin) chemotherapy in patients with newly diagnosed metastatic osteosarcoma with a resectable primary tumor and to compare the effects, good and/or bad, of cabozantinib in combination with MAP versus MAP alone on people with newly diagnosed OST to find out which is better. Common side effects of chemotherapy include nausea, vomiting, hair loss, and fatigue (tiredness). Participants will receive the medications through an IV in their arm and PO. Study participation is expected to last up to 16 months.

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

COMPASS Ancillary NeuroDevelopmental Outcomes (CAN-DO)

Date Added
April 15th, 2026
PRO Number
Pro00148202
Researcher
Sinai Zyblewski

List of Studies


Keywords
Cardiovascular, Pediatrics
Summary

The main reason for this research study is for researchers to evaluate the relationship between congenital heart disease and development. Currently, there is not enough long-term information available to researchers to predict a child's development if they have been diagnosed with Ductal Dependent Pulmonary Blood Flow (DDPBF), a type of congenital heart disease.

Institution
MUSC
Recruitment Contact
Susannah Wakefield
843-792-8317
wakefies@musc.edu

An Open-label, Phase 3 Study to Evaluate the Pharmacokinetics, Safety, and Immunogenicity of Vedolizumab Subcutaneous in Pediatric Subjects With Moderately to Severely Active Ulcerative Colitis or Crohn's Disease Who Achieved Clinical Response Following Open-label Vedolizumab Intravenous Therapy

Date Added
April 1st, 2026
PRO Number
Pro00148307
Researcher
Carmine Suppa

List of Studies

Keywords
Crohn's Disease, Pediatrics, Ulcerative colitis
Summary

Efficacy of the vedolizumab IV and SC formulations has been demonstrated in completed studies of adult subjects with moderately to severely active UC or CD. Clinical trial results are needed to affirm proper dosing, exposure, efficacy and safety for use of vedolizumab in pediatric patients. Subsequent to preliminary positive results from the completed pediatric phase 2, vedolizumab IV is being further evaluated in the phase 3 Studies MLN0002-3024 and MLN0002-3025 (subjects aged 2 to 17 years). Confirmatory positive results from these studies would support submission for registration of vedolizumab IV for pediatric patients. Vedolizumab SC is being evaluated in the proposed study to provide an alternative administration route of vedolizumab for maintenance treatment in the
same pediatric population

Institution
MUSC
Recruitment Contact
Trudy Spuller
843-792-0560
spuller@musc.edu

A PHASE IIIb MULTICENTER, SINGLE-ARM, OPEN-LABEL SURVEILLANCE STUDY OF SUSCEPTIBILITY TO BALOXAVIR MARBOXIL IN PATIENTS WITH INFLUENZA

Date Added
March 31st, 2026
PRO Number
Pro00146625
Researcher
Robert Clifford

List of Studies


Keywords
Children's Health, Drug Studies, Pediatrics
Summary

The flu is caused by a virus that can sometimes change. This can make the flu resistant to treatment, which means drugs, like baloxavir marboxil, can become less effective for treating the flu (also known as "resistance").

The purpose of this study is to monitor changes in the flu virus before and after study treatment with baloxavir marboxil in children. The resistance of the flu virus to study treatment with baloxavir marboxil will also be monitored.

Institution
MUSC
Recruitment Contact
Jasmine Dewitt
843-870-2225
dewitjas@musc.edu

A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-risk and Low-risk Fusion Negative Rhabdomyosarcoma

Date Added
March 11th, 2026
PRO Number
Pro00149531
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Cancer, Children's Health, Pediatrics
Summary

This study is recruiting pediatric patients who have been diagnosed with rhabdomyosarcoma (RMS). RMS is a type of cancer that occurs in the soft tissues of the body. The goal of the study is to compare the effects, good and/or bad, of giving less chemotherapy drugs to people with very low risk-RMS to find out which is better. Patients will be separated into two groups based on their tumor type: Low Risk and Very Low Risk. Participants in the Low Risk group will receive Vincristine, Dactinomycin and Cyclophosphamide. Participants in the Low Risk group will receive Vincristine and Dactinomycin. All participants will be tested for genetic differences (called MYOD1 and TP53). If any participant is found to have these genetic differences, they will receive Vincristine, Dactinomycin and Cyclophosphamide. Common side effects of chemotherapy include nausea, vomiting, hair loss, and fatigue (tiredness). Participants will receive the medications through an IV in their arm. Study participation is expected to last up to one year.

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

Epidermolysis Bullosa Clinical Characterization and Outcomes Database

Date Added
February 26th, 2026
PRO Number
Pro00142696
Researcher
Lara Wine Lee

List of Studies


Keywords
Pediatrics, Skin
Summary

Epidermolysis Bullosa (EB) is a rare, inherited skin condition that makes the skin extremely fragile, causing painful blisters and wounds from even minor friction or injury. There is currently no cure, and because EB is uncommon, doctors still have limited high-quality data to guide the best treatment and long-term care. This study is part of a large North American effort to collect and organize health information from people with EB into a secure database. By tracking how the disease progresses over time, along with symptoms, complications, and treatments, researchers hope to better understand EB and improve care for future patients. Participation involves consenting to share medical record information and optionally completing brief questionnaires during routine clinic visits or by email. No experimental treatments or extra medical procedures are involved. While there is no direct benefit to participants, the knowledge gained may help improve care and support the development of new treatments in the future.

Institution
MUSC
Recruitment Contact
Lara Wine Lee
843-792-2890
winelee@musc.edu

A Phase 3, Double-Blind, Placebo-Controlled, Randomized Study to Assess the Safety of Epicutaneous Immunotherapy with DBV712 250 g in 1-through 3-year old Children with Peanut Allergy

Date Added
February 13th, 2026
PRO Number
Pro00149278
Researcher
Kelli Williams

List of Studies


Keywords
Allergy, Children's Health, Pediatrics
Summary

This study is a double‑blind, placebo‑controlled research study to evaluate the safety and effectiveness of a skin patch treatment for peanut allergy in children ages 1 to 3. The patch delivers a very small amount of peanut protein through the skin and is designed to help the immune system become less sensitive to peanuts over time.

Participation in the study will last approximately 34 weeks. Participation is voluntary, and participants may withdraw at any time.

Institution
MUSC
Recruitment Contact
Rohini Rao
(843) 792-0965
recruitment@musc.edu

A Phase 3a, observer-blind, randomized, controlled study to demonstrate lot-to-lot consistency and evaluate the immunogenicity and safety of an investigational varicella vaccine compared with Varivax, administered as a first dose to healthy children 12 to 15 months of age

Date Added
February 10th, 2026
PRO Number
Pro00145276
Researcher
Robert Clifford

List of Studies


Keywords
Adolescents, Children's Health, Pediatrics, Vaccine
Summary

The investigational varicella vaccine (hereafter referred to as VNS vaccine) is a new
candidate varicella vaccine derived from the Oka strain. The main rationale for the
development of VNS vaccine is to provide an additional alternative varicella vaccine as an advantage from a public health perspective to prevent varicella disease

Institution
MUSC
Recruitment Contact
Amelia Gaines
843-284-4254
gaineame@musc.edu

A Pragmatic Clinical Trial of the WE BEAT Well-Being Education Program in Adolescent Congenital Heart Disease: WE BEAT CHD Study

Date Added
January 21st, 2026
PRO Number
Pro00148198
Researcher
Stephanie Gaydos

List of Studies

Keywords
Pediatrics
Summary

The overall goal of this study is to learn how to best support teens with CHD to improve their health and quality of life. Adolescents with moderately and severely complex CHD experience considerable risk of mental health comorbidities. Psychological health is important to cardiovascular health and related outcomes. This study will evaluate the effectiveness of a well-being focused telemedicine intervention in adolescents with CHD. The study is to learn if the WE BEAT program helps teens with CHD improve their ability to cope with stress and anxiety. The study will also create a data bank to study connections between mental health, heart health and biomarkers (hair, saliva, urine, blood samples).

Institution
MUSC
Recruitment Contact
Terry Headley
843-494-3796
headleyt@musc.edu

A Randomized Trial of Dichoptic Treatment for Amblyopia in Children 4 to 7 Years of Age

Date Added
September 16th, 2025
PRO Number
Pro00141315
Researcher
James Bowsher

List of Studies

Keywords
Children's Health, Pediatrics, Vision/ Eye
Summary

Children with amblyopia (lazy eye) are often treated with patch therapy. Patch therapy often results in leftover amblyopia and has many challenges associated with its use. This study will determine how traditional patch therapy results compare to those achieved with the Luminopia digital therapeutic system.

Institution
MUSC
Recruitment Contact
Martha Long
843-792-6591
mas405@musc.edu



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