A Phase 3 Randomized Study of Selumetinib (IND # 77782) Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

Date Added
February 23rd, 2022
PRO Number
Pro00118178
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Brain, Pediatrics
Summary

This study is for newly diagnosed previously untreated neurofibromatosis type 1 (NF1) associated with low-grade glioma (LGG). The purpose of this study is to see if selumetinib works just as well as the standard treatment of carboplatin/vincristine (CV) for subjects with NF1-associated LGG, and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway. Participants can expect to receive treatment for up to 24 months, until the cancer gets worse or until the side effects are too great. After study completion, participants will continue to be followed for up to 10 years.

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

A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)

Date Added
February 22nd, 2022
PRO Number
Pro00118193
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Brain, Pediatrics
Summary

This study is for newly diagnosed or previously untreated low-grade glioma (LGG). The overall goal of this study is to see if selumetinib works just as well as the standard treatment using carboplatin and vincristine (called CV) for subjects with LGG. Selumetinib is a drug that works by blocking a protein (a basic building block of the human body) that lets cancer cells grow without stopping. Participants can expect to receive treatment on this study for about 1–2 years. After study completion, participants will continue to be followed for up to 10 years.

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

Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Date Added
February 1st, 2022
PRO Number
Pro00113673
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Brain, Pediatrics
Summary

This study is for participants that have been diagnosed with Medulloblastoma. The purpose of this study is to test the effectiveness of using an investigational agent called DFMO for Medulloblastoma. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to either keep participants tumor in remission or if they have active tumor, for their tumor to respond to the treatment and will also look at the safety and tolerability of DFMO. After this first day participants will be seen in clinic once every 30 days for the first 6 months of the study, after that they will be seen once every 90 days for the remainder of the study. These visits will last about 2 hours.

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

Specimen Banking With Clinical and Genomic Data Registry With the Establishment of Tumor Models for Pediatric Cancers

Date Added
February 1st, 2022
PRO Number
Pro00115767
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Pediatrics
Summary

This study is for participants with tumors from pediatric cancers and genomic/molecular testing was done as part of standard of care treatment. This is an observational study; therefore, only information about the disease and medical treatment will be collected and participants will not receive any treatments or additional medications. The sponsor, Beat Childhood Cancer, will collect and store personal health information and molecular/genomic test results, tissue samples, and bodily fluids (examples: additional tube(s) of blood, urine, bone marrow or cerebral spinal fluid) that are left over after testing or treatment is completed in a data registry and a specimen bank, and make these available for future research. Database personnel will continue to collect and store participant information from future visits, as long as they do not withdraw from participation in this study.

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

An investigation of reward brain circuitry structure and function in individuals with co-occurring alcohol use disorder and bipolar disorder and their unaffected offspring

Date Added
October 5th, 2021
PRO Number
Pro00114068
Researcher
Will Mellick

List of Studies


Keywords
Adolescents, Alcohol, Brain, Depression, Mental Health, Substance Use
Summary

The purpose of this two-visit brain imaging (magnetic resonance imaging, MRI) study is to identify brain targets for improving treatment and preventative interventions for individuals at risk for co-occurring bipolar disorder and alcohol use disorder. The preliminary visit for a parent and his/her biological child will include completion of clinical interviews, surveys, and labwork to determine study eligibility. If they are considered eligible for the study, brain imaging visits will occur within 1-2 weeks at which a 1-hour MRI will be completed along with additional clinical interviews, surveys, and labwork. Brief follow-up phone call interviews will be completed with participants every 3 months for 1 year. Study participation is confidential and compensated.

Institution
MUSC
Recruitment Contact
Sara Hix
(843) 792-7500
hixs@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

Evaluation of Trauma Center-Based, Technology-Enhanced Stepped Care Intervention for Adolescent Traumatic Injury

Date Added
August 3rd, 2021
PRO Number
Pro00111879
Researcher
Tatiana Davidson

List of Studies


Keywords
Adolescents, Mental Health
Summary

Pediatric traumatic injury is the leading cause of death and morbidity among US adolescents and are associated with mental health and health risk outcomes, including posttraumatic stress and depression, deficits in physical recovery, social functioning and quality of life, which if unaddressed, may contribute to increased use of health care services. In 2015 our team launched the Trauma Resilience and Recovery Program (TRRP) at Medical University of South Carolina, a scalable and sustainable, technology-enhanced, multidisciplinary stepped model of care – one of the few in the US - that provides early intervention and direct services to improve access to evidence-based mental health care after traumatic injury for children, adults and families. We have found this model of care to be feasible and acceptable to adolescent patients (ages 12-17) at each level of service. TRRP includes 3 major steps: (1) in-hospital education, brief risk reduction session, and tracking patients' emotional recovery via an automated text-messaging system, (2) a 30-day screen via telephone to identify patients who are good candidates for psychological treatment, and (3) providing referral to best-practice telehealth-based or in-person assessment and treatment. We have partnered with three accredited Level I and II pediatric trauma centers, Prisma-Health Upstate, Children's of Alabama, and Boston Children's Hospital, and propose a multi-site hybrid 1 effectiveness-implementation randomized controlled trial with 300 adolescent (ages 12-17) traumatic injury patients to assess the extent to which TRRP promotes improvement in quality of life and emotional recovery and gather preliminary data on the potential for TRRP to be implemented in other Level I trauma centers. This study will provide valuable data on the efficacy, preliminary effectiveness and potential for implementation of an innovative, cost-effective, sustainable technology-enhanced intervention designed to address the unique needs of adolescent injury patients and mitigate short- and long-term impact of injury on mental health, quality of life, and overall well-being.

Institution
MUSC
Recruitment Contact
Olivia Eilers
843-792-0979
eilerso@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

Brief Computerized Intervention for Reducing Adolescent Cannabis and Alcohol Use

Date Added
March 2nd, 2021
PRO Number
Pro00106723
Researcher
Austin Hahn

List of Studies

Keywords
Adolescents, Alcohol, Substance Use
Summary

The study will test a computerized treatment with subjects ages 13-17 years who are interested in seeking treatment for alcohol and/or cannabis use. Follow-up assessments will be conducted at one- and three-months following treatment.

Institution
MUSC
Recruitment Contact
Amy Boatright
843-792-5200
boatrigh@musc.edu



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