The Role of Minimal Residual Disease Testing before and after Hematopoietic Cell Transplantation for Pediatric Acute Myeloid Leukemia + save

Date Added
June 4th, 2013
PRO Number
Pro00011017
Researcher
Michelle Hudspeth
Keywords
Adolescents, Cancer, Cancer/Leukemia, Children's Health, Infant, Pediatrics, Transplant
Summary

This study is being offerred to patients that have acute myelogenous leukemia (AML) which is a cancer of the blood and these patients are going to have a stem cell transplant. This study is looking to determine how accurate two different laboratory tests are at detecting residual, or small numbers of cancer cells in the body before and after stem cell transplant, as well as whether or not results of these two tests show how well a recipient might do after transplant.

Institution
MUSC
Recruitment Contact
Karen Rogers
843-792-2740
rogersk@musc.edu

A multicenter, randomized, double-blind, 8 week study to evaluate the dose response, efficacy and safety of aliskiren in pediatric hypertensive patients 6-17 years of age + save

Date Added
May 14th, 2013
PRO Number
Pro00016193
Researcher
Ibrahim Shatat
Keywords
Adolescents, Drug Studies, Hypertension/ High Blood Pressure, Pediatrics
Summary

This is an 8 week drug study for children 6 to 17.5 years of age who have been diagnosed with hypertension. The purpose is to test the safety, tolerability and effictiveness of the study medication, Aliskiren. The study has 3 phases:

Screening Phase: All subjects will "wash out" (be on a placebo and no hypertensive medication) for a period of 7 - 21 days, during which they will be closely monitored the the physicians and research study staff.

Phase 1: (4 weeks/28 days) After the wash out period, all subjects will given the study drug, Aliskiren, with dosing based on weight.

Phase 2: (4 weeks/days). Patients will be randomized to receive either placebo or continue the aliskiren treatment assigned during Phase 1.

Once subjects have completed the 8 week study, they will be eligible to enroll in a 52 week extension study which looks at the long term safety, efficacy and tolerability of the study drug.

Institution
MUSC
Recruitment Contact
Barron Bryant
843-792-4837
bryantcb@musc.edu

VCUGs: What is the Big Deal? + save

Date Added
May 13th, 2013
PRO Number
Pro00008937
Researcher
Elizabeth Peacock
Keywords
Adolescents
Summary

Voiding cystourethrograms (VCUGs) are a radiology study used to evaluate the urinary tract for a variety of conditions in the child. This can be particularly stressful and anxiety-provoking to a child and his/her parent. This research study is designed to identify what particular factors cause the most stress and anxiety.

Institution
MUSC
Recruitment Contact
Jessica Jenkins
843-876-0630
jenkijn@musc.edu

What are the Transition Experiences of Parents of Chronically Ill Pediatric Research Participants at the Conclusion of a Clinical Trial? + save

Date Added
April 24th, 2013
PRO Number
Pro00024368
Researcher
Teresa Atz
Keywords
Adolescents, Infant, Pediatrics
Summary

The goal of this study is to understand parents' experiences at the end of their child's clinical trial in order to ease the transition process from clinical trials to standard of care for children and their families.This study is open to parents or the legal guardian of a pediatric research patient with a chronic illness who has completed a clinical trial within a 12 month period. Participation includes one brief interview and completion of a short questionnaire.

Institution
MUSC
Recruitment Contact
Teresa Atz
843-813-3931
atz@musc.edu

ACNS0821: Temozolomide with Irinotecan versus Temozolomide, Irinotecan plus Bevacizumab (NSC# 704865, BB-IND# 7921) for Recurrent/Refractory Medulloblastoma/CNS PNET of Childhood, A COG Randomized Phase II Screening Trial + save

Date Added
April 19th, 2013
PRO Number
Pro00024598
Researcher
Jacqueline Kraveka
Keywords
Adolescents, Cancer, Pediatrics
Summary

There is no current standard treatment for recurrent/refractory
medulloblastoma/PNET. The combination of the drugs temozolomide and
irinotecan has been used to treat adults and children with other types of cancer.
The combination has also been used in previous studies to treat a small number
of children with recurrent or refractory medulloblastoma/PNET as well as other
recurrent tumors, with encouraging results. This study uses the results of these
earlier studies, and looks at how well giving temozolomide and irinotecan daily
for 5 days every 28 days works when given to children and young adults with
recurrent or refractory medulloblastoma/PNET.

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

AALL1131: A Phase III Randomized Trial for Newly Diagnosed High Risk B-precursor Acute Lymphoblastic Leukemia (ALL) Testing Clofarabine (IND# 73789, NSC# 606869) in the Very High Risk Stratum + save

Date Added
March 14th, 2013
PRO Number
Pro00016822
Researcher
Jacqueline Kraveka
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

Multimodal investigation of brain structure and function in Tourette's syndrome + save

Date Added
February 5th, 2013
PRO Number
Pro00021403
Researcher
Colleen Hanlon
Keywords
Adolescents, Central Nervous System, Healthy Volunteer Studies, Movement Disorders, Nervous System, Pediatrics, Psychiatry
Summary

The purpose of this pilot study is to investigate alterations in neural activity among individuals with Tourettes Syndrome. This will be acheived with single-pulse transcranial magnetic stimulation (TMS) and magnetic resonance imaging (MRI). These techniques are non-invasive. TMS is a brain stimulation method that allows us to measure the speed of information processing between brain regions as well as between the brain and the muscles. Combine with MRI, these techniques allow us to create a dynamic image of brain activity which may help guide future treatments. It is important to note that this will be used for research purposes and is not diagnostic.

Institution
MUSC
Recruitment Contact
William DeVries
843-792-6402
devriesw@musc.edu

ANHL1131: INTERGROUP TRIAL FOR CHILDREN OR ADOLESCENTS WITH B-CELL NHL OR B-AL: EVALUATION OF RITUXIMAB EFFICACY AND SAFETY IN HIGH RISK PATIENTS + save

Date Added
January 10th, 2013
PRO Number
Pro00021704
Researcher
Jacqueline Kraveka
Keywords
Adolescents, Cancer, Pediatrics
Summary

A large randomized trial is necessary to evaluate whether rituximab can add benefit to the current chemotherapy regimen. Two pilot studies in children provide preliminary evidence of safety and activity of rituximab in this disease setting that support such a study. A Berlin-Frankfurt-Münster group (BFM) study, which tested a single dose of rituximab administered prior to chemotherapy, has shown tumor responses. A COG pilot study tested the safety and tolerability of the combination of rituximab with LMB chemotherapy and showed no increased short term toxicity.

Institution
MUSC
Recruitment Contact
Jacqueline Kraveka
843.792.2957
kravekjm@musc.edu

Pressure Volume Loop Analysis in Fontan Patients + save

Date Added
January 2nd, 2013
PRO Number
Pro00021772
Researcher
Ryan Butts
Keywords
Adolescents, Children's Health, Heart, Pediatrics
Summary

The goal of this study is to better understand single ventricle circulation after its final palliation, the Fontan procedure. Each specific aim of the project directly affects current knowledge and/or aids future research in this population. In regards to Aim1, a better understanding the impaired responses to stress in single ventricle circulation will help direct future research on ways to improve quality of life as well as help direct medical and surgical therapies in this population. In regards to Aim 2, the mechanism that leads to improved exercise capacity with PDE5 inhibition will be elucidated. These three aims all compliment the overall goal of understanding the mechanisms that lead to heart failure in this high risk population.

Institution
MUSC
Recruitment Contact
Ryan Butts
843-792-9146
butts@musc.edu

A Randomized Controlled Trial of Varenicline for Adolescent Smoking Cessation + save

Date Added
December 4th, 2012
PRO Number
Pro00014398
Researcher
Kevin Gray
Keywords
Adolescents, Smoking
Summary

This is a placebo-controlled smoking cessation treatment study for adolescents ages 14-21. After assessment and inclusion into the study, participants will be randomized to receive a 12-week double blind course of varenicline or placebo. All participants will concurrently participate in a contingency management (CM) intervention, specifically designed to reinforce participant retention.

Institution
MUSC
Recruitment Contact
Christine Horne
843-792-5807
hornechr@musc.edu