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.
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 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.
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 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.
This is for patients aged 18 and older who have Sickle Cell Disease. The main purpose of this study is to find out how safe IMR-687 is for treating people with Sickle Cell, and whether it is has any unwanted effects. Participation in this study will last about 8 months.
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.
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 16 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.
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.