ATHN 9 is a natural history study to assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.
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 adults.
In this research study, there will be up to 5 adults 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.
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.
The purpose of this study is to evaluate the likely safety and effectiveness of danaparoid sodium versus argatroban for patients suffering from heparin-induced thrombocytopenia (HIT). Patients with HIT will have low blood platelet counts due to being given the medication heparin. Adult and pediatric participants who are determined to be eligible to participate will be assigned by chance to treatment with danaparoid sodium or argatroban. The study drugs are administered through intravenous therapy (IV). Study and safety assessments, including blood draws and compression ultrasounds, will be completed at study visits. Participants will be in this study for at least 45 days, but could receive treatment longer depending on HIT severity. You will be hospitalized while you are receiving study drug treatment for a minimum fo 14 days. You will have 4 study follow-up visits after they have been discharged from the hospital.
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.
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.
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.
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.
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.Participants in the SCDIC implementation research protocols may include either patients enrolled in SC2 or other MUSC patients with SCD
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.