A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway–Mutant Acute Lymphoblastic Leukemia

Date Added
February 13th, 2018
PRO Number
Pro00071934
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Cancer, Cancer/Leukemia, Pediatrics
Summary

This study is for patients that have been diagnosed with High Risk B-Lymphoblastic Leukemia (HR B-ALL). The investigational drug in this study is Ruxolitinib. The purpose of this study is to find out if the study drug, ruxolitinib, in combination with standard HR B-ALL treatment is safe and effective in children, adolescents, and young adults with HR B-ALL. Participants can expect to be in this study for the treatment period of approximately 26 months (females) or 38 months (males) plus the post-treatment follow-up. Subjects are considered on study during the post-treatment follow-up period until the subject is deceased, lost to follow-up, or until the study is completed. Subjects in this study will be followed until all enrolled subjects have been followed for 3 years from Day 1 or are deceased or lost to follow-up.

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

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease

Date Added
February 28th, 2017
PRO Number
Pro00062418
Researcher
Shayla Bergmann

List of Studies


Keywords
Drug Studies, Pediatrics
Summary

This is a study to determine the use of recombinant Von Willebrand Factor (rVWF) in the treatment and control of nonsurgical bleeding episodes and bleeding during elective and emergency surgery in children with severe Von Willebrand Disease. The study will last approximately 14 months and will involve regular visits to a research clinic.

Institution
MUSC
Recruitment Contact
Lauren Card
843-792-5935
cardl@musc.edu

Treatment of Drug-resistant Adult and Pediatric Primary Focal Segmental Glomerulosclerosis Using the LIPOSORBER® LA-15 System

Date Added
January 10th, 2017
PRO Number
Pro00059528
Researcher
Katherine Twombley

List of Studies


Keywords
Blood Disorders, Kidney, Pediatrics
Summary

A device called the "Liposorber LA-15 System" has been approved by the United States Food and Drug Administration for treating kids with focal segmental
glomerulosclerosis (FSGS). The "Liposorber LA-15 System" can only be used if other treatment options, like drugs, don't work or can't be used, but the kidneys are still working okay. It can also be used if the subject has had a kidney transplant and the FSGS comes back after the transplant. Although the Liposorber System can be used for FSGS, we are not sure how well the Liposorber System works. So, we are doing this study to find out how well the treatment works.

In this research study, there will be up to 5 children who have FSGS enrolled at MUSC. Subjects will come back for up to 12 treatments over 9 weeks and then 5 visits to their study doctor over the next 2 years.

Institution
MUSC
Recruitment Contact
Katherine Twombley
843-792-8904
twombley@musc.edu

Observational Study for Pediatric Rheumatic Diseases: The CARRA Registry

Date Added
November 3rd, 2015
PRO Number
Pro00048606
Researcher
Natasha Ruth

List of Studies


Keywords
Arthritis, Autoimmune disease, Fibromyalgia, Lupus, Pediatrics, Rheumatoid, Sarcoidosis, Scleroderma
Summary

The purpose of this study is to create and maintain a registry, which is a database (a searchable collection of information) about children, adolescents and young adults with pediatric onset of rheumatic diseases. This data may help in the evaluation of the safety and benefit of medications that are prescribed to patients who have rheumatic diseases.

Institution
MUSC
Recruitment Contact
Shameeka Bowman
843-792-8317
bowm@musc.edu

ACNS1022: A Phase II Randomized Trial of Lenalidomide (NSC # 703813, IND # 70116) in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytoma and Optic Pathway Gliomas

Date Added
August 23rd, 2013
PRO Number
Pro00026888
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Pediatrics
Summary

The overall goal of this study is to find out what effects, good and/or bad, a low
dose and a high dose of lenalidomide have on children, adolescents and young
adults with recurrent (has come back after being treated), refractory (has not gone
away with previous treatment), or progressive (is not responding to previous
treatments) Juvenile Pilocytic Astrocytomas (JPA) and Optic Pathway Gliomas
(OPG).

Institution
MUSC
Recruitment Contact
Jacqueline Kraveka
843-792-2957
kravekjm@musc.edu

ANBL12P1: Pilot Study Using Myeloablative Busulfan/Melphalan (BuMel) Consolidation Following Induction Chemotherapy for Patients with Newly Diagnosed High-Risk Neuroblastoma

Date Added
July 17th, 2013
PRO Number
Pro00026760
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Pediatrics
Summary

the United States, it is standard treatment for patients with high-risk neuroblastoma (NBL) to receive the drugs carboplatin, etoposide and melphalan (CEM) as the preparative regimen in Consolidation therapy prior to Autologous Stem Cell Transplant (ASCT). BuMel Consolidation therapy has recently been studied in patients with high-risk NBL in some European countries. The findings from those studies indicate that the use of BuMel prior to ASCT may be linked to an increase in the survival rate for patients when compared to CEM. Those studies also indicate that the chance of the disease coming back (a relapse) may be lower among the patients who received BuMel Consolidation therapy. In North America the BuMel combination is considered experimental. In this study, researchers want to find out if a combination of busulfan and melphalan (BuMel) can be given as Consolidation therapy prior to ASCT for subjects with newly diagnosed high-risk NBL. The main goal of this study is to find out what effects, good and/or bad, a BuMel preparative regimen given before ASCT has on people with newly diagnosed high-risk NBL.

Institution
MUSC
Recruitment Contact
Jacqueline Kraveka
843-792-2957
kravekjm@musc.edu

ACNS0831: Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma Ages 1 to 21 years

Date Added
June 25th, 2013
PRO Number
Pro00024678
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Pediatrics
Summary

Ependymoma is a type of rare childhood cancer that occurs in the brain and spinal cord. Survival statistics are generally disappointing with a 5-year survival of 50-64%. The standard of care for ependymoma is maximal surgical resection followed by radiation therapy directed at the primary site of disease.

Radiation therapy is associated with immediate and long-term toxicities in children, especially young children. For this reason, it has been the practice of some doctors not to give radiation therapy to children with ependymoma when the tumor has been completely surgically removed. The investigators who designed this study have created strict measures to choose those who will not receive additional treatment after surgery and careful follow-up to minimize the risks to those who are assigned to observation only.

Institution
MUSC
Recruitment Contact
Jacqueline Kraveka
843-792-2957
kravekjm@musc.edu

AALL1131: A Phase III Randomized Trial for Newly Diagnosed High Risk B-Lymphoblastic Leukemia (B-ALL) Including a Stratum Evaluating Dasatinib (IND#73789, NSC#732517) in Patients with Ph-like Tyrosine Kinase Inhibitor (TKI) Sensitive Mutations

Date Added
June 22nd, 2012
PRO Number
Pro00016822
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Leukemia, Pediatrics
Summary

To collect information about the patient's leukemia and to seek the optimal treatment for children with ALL based on the individual level of risk of the cancer coming back after treatment. The risk groups are defined as a result of recent research conducted by the Children's Oncology Group (COG). We would like to learn if the use of an experimental intrathecal therapy (ITT), which has been given to many people with ALL and has been well tolerated, would be better at preventing relapse in the central nervous system and improve disease outcomes in children with High Risk ALL.

Institution
MUSC
Recruitment Contact
Jacqueline Kraveka
843-792-2957
kravekjm@musc.edu

A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) for transplantation in pediatric and adult patients with hematologic malignancies and other indications

Date Added
August 16th, 2011
PRO Number
Pro00011805
Researcher
Michelle Hudspeth

List of Studies


Keywords
Cancer, Children's Health, Men's Health, Minorities, Pediatrics, Transplant, Women's Health
Summary

The primary purposes of this study are to:
•Provide access to cord blood units for recipients whose best choice for a cord blood unit(s) do not meet all FDA standards, but do meet standards set by the NMDP on this study.
•Assess how well and how quickly blood counts return to normal after transplant in recipients on this study.

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



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