Pathway To Prevention Save

Date Added
November 8th, 2018
PRO Number
Pro00080045
Researcher
Deborah Bowlby
Keywords
Diabetes, Insulin, Pediatrics
Summary

This study is providing annual blood tests looking at diabetes autoantibodies for first degree relatives of patients diagnosed with type 1 diabetes who are between the ages of 1 and 45. It will also provide "diabetes screening" for second and third degree relatives age 1-20. Often parents and/or relatives wonder "if they will get diabetes" down the road or if additional offspring are "at risk" for type 1 diabetes. This study aims at assisting families with free blood testing. If positive, participants will be eligible for "prevention studies". MUSC is currently only participating in the "screening" portion of TrialNet.

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

A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379) Save

Date Added
October 8th, 2018
PRO Number
Pro00082422
Researcher
Jacqueline Kraveka
Keywords
Adolescents, Cancer, Cancer/Brain, Pediatrics
Summary

This study is for patients who have been diagnosed with a neuroblastoma (NBL). The purpose of this study is to find out if we can improve the treatment for subjects with high-risk NBL by adding the experimental drug 131I-MIBG or the experimental drug Crizotinib to recommended therapy. The secondary purpose is to find out if we can reduce the number of stem cell transplants from two to one if we give the experimental drug 131I-MIBG during Induction, and use different drugs as part of the transplant chemotherapy given prior to stem cell infusion during Consolidation. Participants can expect to be in this study for up to 2 years. Researchers would like to continue to follow participants health for up to 10 years.

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

A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors Save

Date Added
October 8th, 2018
PRO Number
Pro00082427
Researcher
Jacqueline Kraveka
Keywords
Adolescents, Cancer, Pediatrics
Summary

This study is for patients that have been diagnosed with Germ Cell Tumors. The purpose of this study is to evaluate whether a strategy of complete surgical resection followed by surveillance can maintain an overall survival rate of at least 95.7% at two years for pediatric, adolescent and adult patients (ages 0- 50 years) with Stage I (low risk) malignant germ cell tumors, and at least 98% for patients with ovarian pure immature teratoma. The drugs used in this study are carboplatin and cisplatin. 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

International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones Save

Date Added
October 3rd, 2018
PRO Number
Pro00082665
Researcher
Jacqueline Kraveka
Keywords
Adolescents, Cancer, Cancer/Leukemia, Pediatrics
Summary

This study is for patients who have been diagnosed with a form of Acute Lymphoblastic Leukemia (ALL) called Philadelphia chromosome positive (Ph+) ALL. The purpose of this study is to compare disease free survival (DFS) of Standard Risk (SR) pediatric Ph+ ALL treated with continuous imatinib combined with either a high-risk COG ALL chemotherapy backbone or the more intensive EsPhALL chemotherapy backbone. A drug called imatinib in combination with chemotherapy will be given. Participants can expect to be in this study for up to 2 years and their health followed for up to 10 years.

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

Optimal Resuscitation in Pediatric Trauma - an EAST Multi-center Study Save

Date Added
August 15th, 2018
PRO Number
Pro00078600
Researcher
Christian Streck
Keywords
Pediatrics, Surgery
Summary

This study will focus on collecting coded patient data from pediatric traumas prospectively. Data will be collected to assess inclusion of patients that fit the inclusion criteria of <18 years old with an elevated shock index based on age (Form A). There will also be a post-resuscitation data collection to examine products given to pediatric patients with an elevated shock index (Form B).
These data collection forms will help us determine the impact on primary outcomes of crystalloid administration volume and timing prior to blood products in children presenting in shock following trauma.
We will assess patient factors that predict need for early transfusion of blood products in pediatric trauma patients presenting in shock. We will also relate primary outcomes to ratio of blood products in pediatric trauma patients in shock who are transfused. This study will help define variability in pediatric trauma fluid and blood resuscitation practices between centers.

Institution
MUSC
Recruitment Contact
Denise Garcia
843-754-8555
garciad@musc.edu

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Save

Date Added
August 14th, 2018
PRO Number
Pro00078283
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

The purpose of this stuThe purpose of this study is to learn more about the safety and effectiveness of VX-445 used in combination with TEZ and VX-561 in patients with cystic fibrosis. Participation could last for approximately 100 weeks. This will include a treatment period that will last approximately 96 weeks, and a safety follow-up period that will last approximately 4 weeks after the last dose of Study Drug. Approximately 400 people will participate in this study.

Institution
MUSC
Recruitment Contact
Robyn Do
(843) 792-1221
dorobyn@musc.edu

LEAHRN (Late Effects After High-Risk Neuroblastoma) Study Save

Date Added
July 14th, 2018
PRO Number
Pro00080522
Researcher
Jacqueline Kraveka
Keywords
Cancer, Cancer/Brain, Pediatrics
Summary

This study is for patients who have been diagnosed with high-risk neuroblastoma. The purpose of this study is to learn if the treatment you received for your high-risk neuroblastoma has affected your health overtime. Participants can expect to be in this study for up to 12 weeks.

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

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F) Save

Date Added
June 12th, 2018
PRO Number
Pro00078198
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

The purpose of this study is learn more about the effectiveness and safety of VX-445 used in combination with TEZ, IVA and VX-561 in patients with cystic fibrosis.
Participation could last for approximately 16 weeks. This will include a Screening Period that will last up to 4 weeks, a Run-in Period that will last up to 4 weeks, a Treatment Period that will last up to 4 weeks, and a Follow-up Period that may last up to 4 weeks after the last dose of Study Drug. Approximately 100 people will participate in this study.

Institution
MUSC
Recruitment Contact
Robyn Do
(843) 792-1221
dorobyn@musc.edu

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Save

Date Added
May 22nd, 2018
PRO Number
Pro00078126
Researcher
Patrick Flume
Keywords
Cystic Fibrosis, Lung, Pediatrics, Pulmonary
Summary

The purpose of this study is to learn more about the effectiveness and safety of VX-445 used in combination with TEZ and VX-561 in patients with cystic fibrosis. Participation could last for approximately 32 weeks. This will include a Screening Period that will last up to 4 weeks, a Treatment Period that will last up to 24 weeks, and a Follow-up Period that may last up to 4 weeks after the last dose of Study Drug. Approximately 360 people will participate in this study.

Institution
MUSC
Recruitment Contact
Robyn Do
(843) 792-1221
dorobyn@musc.edu

Pharmacokinetics of Understudied Drugs Administered to Children per Standard of Care Save

Date Added
May 7th, 2018
PRO Number
Pro00077156
Researcher
Lisa Knight
Keywords
Children's Health, Infant, Pediatrics
Summary

The majority of drugs administered to children are used off label and PK studies to define appropriate dosing are lacking across pediatric age groups. Challenges associated with clinical trials in children limit the ability to conduct PK and dosing trials in this population. Studies capitalizing on standard of care procedures have proven successful in characterizing the PK of drugs used in children. The purpose of this study is to characterize the PK of understudied drugs administered to children per standard of care as prescribed by their treating caregiver.
This study will serve as a tool to better understand drug exposure in children receiving drugs per standard of care. The data collected through this initiative will provide valuable PK and dosing information drugs in different pediatric age groups as well as special pediatric populations (i.e. obese).

Institution
Palmetto
Recruitment Contact
Sarah Newman-Norlund
803-216-3377
Sarah.Newman-Norlund@uscmed.sc.edu

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