A phase 2, Multicenter, Open-Label Study to Assess PK/PD of SEG101 (crizanlizumab), with or without Hydroxyurea/Hydroxycarbamide, in Adult Sickle Cell Patients with Vaso-Occlusive Crisis Save

Date Added
October 24th, 2017
PRO Number
Pro00071740
Researcher
Julie Kanter washko

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Keywords
Blood Disorders
Summary

This study will assess the efficacy of crizanlizumab in sickle cell disease patients. The study is for male and female subjects between the ages of 18 to 70 who have experienced at least one pain crisis within a 12 month period. The drug is given via an IV infusion in an outpatient setting and consists of 52 visits.

Institution
MUSC
Recruitment Contact
Emily Warner
843-876-8614
warnere@musc.edu

A Family-Centered Self-Management Program for Young Children with Sickle Cell Disease: Phase II Save

Date Added
September 7th, 2017
PRO Number
Pro00068250
Researcher
Shannon Hudson

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Keywords
Blood Disorders, Children's Health, Infant, Pediatrics
Summary

This study tests an intervention to help children with sickle cell disease ages 0-7 years and their families. The intervention is available on a smartphone or tablet, and includes an app for keeping track of symptoms and messaging with a health care provider. For more information, please contact Shannon at 843-792-9379.

Institution
MUSC
Recruitment Contact
Shannon Phillips
843-792-9379
phillipss@musc.edu

Self-Management for Families and Youth Living with Sickle Cell Disease - SMYLS Save

Date Added
September 2nd, 2017
PRO Number
Pro00062837
Researcher
Shannon Hudson

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Keywords
Blood Disorders, Children's Health, Pediatrics
Summary

The purpose of this study is to find out whether a web-based intervention using a mobile device over a 12-week period is helpful in managing symptoms of Sickle Cell Disease in children, such as pain and fatigue and quality of life. For more information, please contact Shannon at 843-792-9379.

Institution
MUSC
Recruitment Contact
Shannon Phillips
843-792-9379
phillipss@musc.edu

Disseminating NIH Evidence Based Sickle Cell Recommendations in North Carolina and South Carolina Save

Date Added
August 22nd, 2017
PRO Number
Pro00064785
Researcher
Julie Kanter washko

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Keywords
Blood Disorders
Summary

The purpose of this project is to utilize implementation science to evaluate patient and system outcomes associated with the dissemination of a toolbox of decision support tools designed based on the NHLBI evidence-based guidelines for treating individuals with sickle cell disease (SCD). These tools will be disseminated to primary care physicians and emergency department providers across NC and SC. The overarching goal is to implore provider education and the implementation of a co-management model of care to improve the efficiency and quality of healthcare for individuals with SCD. The co-management model of care suggests that individuals with SCD benefit from visiting both their primary care clinician and their SCD specialist twice per year.

Institution
MUSC
Recruitment Contact
Ashley Green
843-876-8652
greenan@musc.edu

A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion Save

Date Added
August 8th, 2017
PRO Number
Pro00068449
Researcher
John Lazarchick

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Keywords
Blood Disorders, Drug Studies, Rare Diseases
Summary

This is a first-in-humans gene therapy study for subjects diagnosed with severe hemophilia A. A one time infusion of the gene therapy product (BAX888) will be given with the hope of eliminating or reducing the need for prophylactic and/or on-demand use of FVIII concentrate therapy. Up to 10 subjects will be enrolled study-wide with up to two subjects enrolled at MUSC. This study will require weekly visits to the study clinic after initial infusion for the first 15 weeks followed by monthly visits for the first year. Compensation will be provided.

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

Implementation of a Sickle Cell Enhanced Novel Care Network in South Carolina (iSCENSC): Needs Assessment and Registry Protocol Save

Date Added
August 8th, 2017
PRO Number
Pro00066242
Researcher
Cathy Melvin

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Keywords
Blood Disorders
Summary

iSCENSC is a three-part study that will use new methods (called implementation science) to identify ways to enhance care for people with sickle cell disease (SCD) who are in the (SC)2 South Carolina Sickle Cell network. The (SC)2 network is designed to help more people with SCD find a doctor with training in sickle cell disease and to improve their medical care. Part one of the study is a needs assessment where we look at the current barriers for people with SCD. Part two is a registry for individuals with SCD; and part three will be a study or studies to try and help improve the care for individuals with SCD.

Institution
MUSC
Recruitment Contact
Ashley Green
843-876-8652
greenan@musc.edu

STA-ImmunoDef VIII and STA-Immunodef IX Sample Stability and Fresh-Frozen Study Save

Date Added
August 1st, 2017
PRO Number
Pro00065916
Researcher
John Lazarchick

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Keywords
Blood Disorders
Summary

Patients with hereditary blood clotting protein deficiencies of Factor VIII (diagnosis of Hemophilia A) and FIX deficiency (diagnosis of Hemophilia B) will be asked to donate a blood sample to test new reagents from Stago to replace the ones currently used in our laboratory to measure the levels of these clotting factors in plasma. In part one of the study sample once drawn and processed will be immediately have levels of either FVIII or Factor IX measured and then the assay repeated hourly for the next 5 hours to assess sample stability (reproducibility). The purpose of this part of the study is validate the sample stability at room temperature known from the literature described in CLSL guideline. In part two of the study, aliquots of the same samples will be frozen at both -20C and -80C. The -20C samples will be thawed at day 7, 15 and 17 and the Factor VIII and Factor IX assays repeated. The -80C samples will be thawed at days 15, 30 and 32 and the assays repeated. The purpose of this part of the study is to validate the sample stability after freezing at -20C and -80C known from the literature and recognized by CLSI.

Institution
MUSC
Recruitment Contact
John Lazarchick
843-792-3424
lazarj@musc.edu

Evaluation of the CytoRADx SystemTM as a Biodosimeter for Special Human Populations Save

Date Added
July 14th, 2017
PRO Number
Pro00067066
Researcher
Gary Headden

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Keywords
Blood Disorders, Children's Health, Environmental Factors, HIV / AIDS, Non-interventional, Pediatrics
Summary

This is a study to develop a test for radiation exposure in the event of a nuclear event such as the detonation of a nuclear device or widespread radiation exposure. We will collect blood samples from a variety of different types of human subjects whose current medical status could possibly have an effect on the results of the test, such as trauma, burns, infections or a damaged immune system or young children. The goal is to ensure that these conditions do not affect the results of the test.

Institution
MUSC
Recruitment Contact
Robert Houck
(843) 792-3576
houckr@musc.edu

Sample Collection for Performance Evaluation for INNOVANCE® Free PS Ag* Save

Date Added
May 25th, 2017
PRO Number
Pro00064923
Researcher
Gary Headden

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Keywords
Blood Disorders, Circulation, Non-interventional, Rare Diseases
Summary

Protein S is a protein that is very important to the blood clotting process. When people don't have enough Protein S they run the risk of developing blood clots. This study is collecting samples from people who may have abnormal Protein S levels in order to develop a new way of checking the Protein S levels. This test is looking specifically for FREE Protein S. The free protein S is Protein S that is not bound or "tied up" and is therefore more ready to do its job in the clotting process.

Institution
MUSC
Recruitment Contact
Robert Houck
843-792-3576
houckr@musc.edu

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Finding Study of SC411 in Children with Sickle Cell Disease Save

Date Added
March 28th, 2017
PRO Number
Pro00064573
Researcher
Julie Kanter washko

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Keywords
Blood Disorders
Summary

The SCOT Trial is testing the safety, effectiveness and to find the optimal dose of the Investigational New Drug, called SC411. The study is for male and female subjects with sickle cell disease and are between the ages of 5 to 17 years of age. Subjects must have had at least two and no more than 10 documented episodes of clinical sickle cell crises within 12 months. This is an outpatient study that will involving visits the research site at least 7 times over a 12 week period.

Institution
MUSC
Recruitment Contact
Emily Warner
843-876-8614
warnere@musc.edu

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