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.
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.
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.
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.
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.
This trial is a first-in-human clinical trial with subcutaneous injections of turoctocog alfa pegol (N8-GP). You must be male, age 12 or older with a diagnosis of hemophilia A to participate. The purpose of the trial is to investigate the potential effect of N8-GP
on prevention of bleeds in previously treated patients with severe hemophilia A. This study would require approximately 10 visits to the research clinic over 5 months. Compensation will be provided for time and travel.
GBT440 is an study drug that has been shown to increase oxygen in the blood. This study is recruiting patients with sickle cell disease to establish efficacy and safety of GBT440 as compared with placebo.
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 this research study, there will be up to 5 children 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.
This study is meant to compare transplant to standard care (regular care) for sickle cell patients. By comparing the health outcomes for patients who receive bone marrow transplant to those patients who receive standard of care, this study will be able to determine whether the two treatments are the same, or if one is better than the other.
Sickle cell disease (SCD) is a genetic blood disorder present at birth characterized by painful vaso-occlusive episodes. Pain episodes typically result in approximately one to two severe pain crises requiring hospitalization per year along with another ten pain episodes managed at home. Pain crises can emerge as early as six to twelve months and most children with the most common form of the disease (HbSS), experience a pain episode requiring hospital care by the age of five years. Promoting better coping with pain in preschool age children likely requires additional adaptation of methods and consideration of a developmental framework. Very young children have limited coping skills, a limited ability to communicate pain, and it is up to the primary caregiver to correctly interpret and treat the pain that a child may be experiencing. Despite the frequency of painful episodes early in life in SCD, there has been little work to-date addressing pain management in young children with SCD. Management of pain episodes early in life by parents may be an important developmental context for later child and family coping with pain, as well as coping with other aspects of this chronic health condition. Consequently, the current study will target parents of preschool age children with SCD through an online video-based platform in order to teach pain management skills to help to identify and manage SCD-related pain episodes.