A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia Save

Date Added
September 24th, 2019
PRO Number
Pro00091452
Researcher
Charles Greenberg

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

The purpose of this study is to test the safety and effectiveness of a new study drug in adults who have warm antibody autoimmune hemolytic anemia (wAIHA). This study aims to determine if participants receiving the study drug, called fostamatinib, respond better than participants receiving placebo (an inactive substance) measured by an increase in red blood cell count. Participation in the study will be for a duration of at least 30 weeks with a total of approximately 15 visits to the study clinic.

Institution
MUSC
Recruitment Contact
Elizabeth Szwast
843-792-5935
hinsone@musc.edu

Monotherapy anticoagulation to expedite home treatment of venous thromboembolism (MATH VTE) Save

Date Added
May 21st, 2019
PRO Number
Pro00085403
Researcher
Andrew Matuskowitz

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

Previous studies have shown that many patients diagnosed with blood clots in the lungs and limbs can be safely discharged home from the emergency department with blood thinner therapy. However, physician discomfort and prescription medication cost results in many patients being admitted to the hospital. This study aims to show that the majority of low risk patients with blood clots who are started on a blood thinner called apixaban can be safely discharged from the emergency department without having a bad outcome. Specifically, we expect to find that these patients have a very low risk of worsening blood clot or bleeding within the first month after starting apixaban.

Institution
MUSC
Recruitment Contact
Aja Bayo
843-792-7944
bayo@musc.edu

SMYLS: A Self-Management Program for Youth Living with Sickle Cell Disease Save

Date Added
March 17th, 2019
PRO Number
Pro00084400
Researcher
Shannon Hudson

List of Studies


Profiles_link
Keywords
Adolescents, Blood Disorders, Pain, Pediatrics
Summary

The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of their sickle cell disease. The intervention lasts 12 weeks with a 3-month follow up period, and uses a smartphone or a tablet. For more information, please contact Shannon at 843-792-9379.

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

Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE)- Part 2 Save

Date Added
August 25th, 2018
PRO Number
Pro00080391
Researcher
Shannon Hudson

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This study is to better understand barriers to and resources for transcranial Doppler (TCD) screening in children with sickle cell anemia (SCA) ages 2 to 16 years. Surveys and interviews are being conducted with children with SCA and their parents/caregivers, healthcare providers, and other stakeholders to learn about their thoughts and experiences.

Institution
MUSC
Recruitment Contact
Joannie Hayes
843-876-8767
hayesj@musc.edu

A phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis Save

Date Added
August 14th, 2018
PRO Number
Pro00079784
Researcher
Shayla Bergmann

List of Studies

Silhouette
Keywords
Blood Disorders
Summary

This study will assess the appropriate dosing and evaluate the safety of crizanlizumab in pediatric sickle cell disease patients. The study is for male and female subjects between the ages of 6 months to 17 years old 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 has the potential to reduce the amount of sickle cell pain crisis a participant may experience. Participants can expected to participant in this study for up to 2 years.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A Randomised, Double-Blind, Parallel-Group, Multicentre, Phase III Study to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease (HESTIA3) Save

Date Added
July 24th, 2018
PRO Number
Pro00078866
Researcher
Shayla Bergmann

List of Studies

Silhouette
Keywords
Blood Disorders
Summary

This study will assess the effect of ticagrelor versus placebo in reducing the rate of pain crisis in sickle cell disease patients. The study is for male and female subjects between the ages of 2 to 17 who have experienced at least two pain crisis within a 12 month period. The drug is given by mouth in an outpatient setting and consists of 13 visits over an 18 month period.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

An Open Label Extension Study of GBT440 Administered Orally to Patients with Sickle Cell Disease Who Have Participated in GBT440 Clinical Trials Save

Date Added
April 10th, 2018
PRO Number
Pro00076264
Researcher
Temeia Martin

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This open label extension study is being conducted to assess the long-term safety and efficacy of GBT440 by providing participants from GBT440-031 continued access to treatment with GBT440 after completion of their participation in GBT440-031 and prior to the product potentially being available commercially. All participants enrolled into this study will receive GBT440.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A Phase IIb Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riociguat in Patients with Sickle Cell Disease Save

Date Added
January 9th, 2018
PRO Number
Pro00074247
Researcher
Temeia Martin

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This study is for patients 18 years and older who have Sickle Cell Disease. The study will be evaluating the safety and tolerability of treatment with riociguat to potentially improvement in blood pressure, blood circulation, and disease symptoms. Participants can expect to be in this study for 20 weeks.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@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

List of Studies


Profiles_link
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

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
Shayla Bergmann

List of Studies

Silhouette
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

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