People with certain types of lung conditions that cause scarring—such as those linked to autoimmune diseases, allergic reactions, or unknown causes—will take part in this study. They'll be followed closely for two years, with regular check-ins and sample collections. During these visits, the doctor will gather health information and take blood samples to help understand how these conditions progress. After the two-year period, participants can choose to stay in touch for future updates or follow-ups by phone or mail. The goal is to learn more about these lung diseases and improve how they're diagnosed and treated over time.
This study is investigating how efimosfermin, compared to a placebo, might improve liver fibrosis in patients with biopsy-proven stage F2- or F3 Metabolic Dysfunction-Associated Steatohepatitis (MASH), also known as "fatty liver disease".
This study is for patients diagnosed with relapsed or refractory T-cell lymphomas. These include:
Peripheral T‑cell lymphoma, not otherwise specified (PTCL-NOS)
Follicular helper T‑cell lymphoma (TFH lymphoma)
Systemic anaplastic large‑cell lymphoma (sALCL)
The study is testing a drug called Soquelitinib. This drug is investigational, which means it has not been approved by the U.S. Food and Drug Administration (FDA).
The purpose of the study is to see how well soquelitinib works compared with standard treatment chosen by a physician. Standard treatment may include:
Belinostat (BELEODAQ®)
Pralatrexate (FOLOTYN®)
The study may last up to 6 years. Study treatment may last up to 2 years (104 weeks). Study visits will happen based on the treatment schedule for the assigned study drug.About 180 people will be screened for the study. About 150 participants will be randomly assigned to a treatment group.Soquelitinib works by blocking an enzyme called ITK. ITK is part of a signaling pathway that helps certain cancer cells grow. Blocking ITK may slow or stop the growth of cancer T cells.
Soquelitinib has not been approved for use in humans and is considered experimental. It is not known if the drug will work for this cancer. The possible side effects are also not fully known. About 70 patients have received soquelitinib in another clinical trial. In those patients, the drug was generally tolerated.In this study, treatment with soquelitinib will be compared with standard treatments used for this type of cancer.Belinostat is approved in the United States for adults with relapsed or refractory PTCL.Pralatrexate is approved in several countries, including the United States, Canada, Australia, and South Korea, for patients with relapsed or refractory PTCL.
Participants will be placed into treatment groups by randomization. Randomization means assignment to a group by chance, similar to flipping a coin. Each participant has an equal chance of receiving any of the study treatments.
This study aims to improve our understanding of Cystic Fibrosis Transmembrane Conductance Regulator(CFTR)-related metabolic syndrome(CRMS), a term used for some infants whose newborn screening results suggest they may be at risk for cystic fibrosis but who do not currently meet the criteria for a cystic fibrosis diagnosis. CFTR stands for cystic fibrosis transmembrane conductance regulator and refers to a gene that provides instructions for making a protein that helps control the movement of salt and water in and out of cells. Changes in this gene can increase a person's risk of developing cystic fibrosis or related health concerns.
The study has two goals. First, we will review existing medical records to better understand the health conditions, symptoms, and long-term health of children with CRMS and adults with cystic fibrosis diagnosed later in life who receive care at the Medical University of South Carolina pulmonary clinic. Second, we will survey parents of children with CRMS to better understand how receiving a CRMS designation affects families emotionally and in their daily lives.
This study is for patients diagnosed with acute graft-versus-host disease that did not get better with steroids (steroid-refractory) . This study is testing an investigational drug called remestemcel-L-rknd ("remestemcel") with ruxolitinib to placebo with ruxolitinib as treatment for steroid-refractory acute graft-versus-host disease (SR-aGVHD). The study treatment is randomized. This means that a computer assigns you to a treatment group by chance, like flipping a coin or drawing a name out of a hat.The primary purpose of the study is If steroids don't work, patients get a second line of therapy. Doctors and researchers want to find a good treatment for SR-aGVHD as these patients are at a very high risk for medical complications and death. This study will compare patients who receive remestemcel with ruxolitinib and patients who receive placebo with ruxolitinib as a second-line treatment.
The ATHNdataset Registry is the largest blood disorders real-world data set in the United States and is used to answer important clinical and scientific questions about the specific causes, prevention, treatment, and the social and economic impact of blood disorders.
The registry will involve collecting health information from the medical records, bleeding and treatment records of participants, and completion of questionnaires. This information will be part of the ATHNdataset registry which will be used to answer scientific, public health, and policy questions about better ways to treat blood disorders.
This research study is being done to compare the effectiveness and safety of frexalimab, a medication given as an injection under the skin every 4 weeks, with tacrolimus, a commonly used anti-rejection medication taken by mouth. The study will include adults ages 18 to 70 who are receiving their first kidney transplant and have a low to moderate risk of rejecting the transplanted kidney. Participants who qualify may take part in the study for up to 5 years.
In our preclinical mouse studies we have identified immune dysfunction in mouse models of osteogenesis imperfecta, also known as brittle bone disease. We then successfully restored bone strength in these mice with our patented adoptive cell therapy treatment.
In this study we will collect small volume blood samples from patients with osteogenesis imperfecta. We will then analyze these blood samples through a variety of methods to determine if osteogenesis imperfecta patients have the same immune dysfunction as we have seen in our mice studies.
AKI is a condition where the kidneys are not able to make enough urine. Therefore, another way to remove waste from the body is needed. One way of removing waste from the body is called Continuous Kidney Replacement Therapy (CKRT). There are different types of CKRT, but all allow waste to be removed from your body because the kidneys can not do this any longer. Acute kidney injury can also cause your immune system to not work correctly and this may be one of the reasons that AKI is such a deadly condition. White blood cells, which are key parts of the immune system, are overactive in AKI and may be the reason the immune system doesn't work the way it should. The experimental device is a Selective Cytopheretic Device (SCD). It is a set of tubing and a cartridge that is connected in series to a CKRT circuit. The SCD binds and stops overactive white blood cells. The purpose of this study is to test if the SCD helps someone with AKI recover faster than without it.
The purpose of the study is to better understand the experience of loved ones whose family members were treated for violence injuries at MUSC, and what services could potentially be improved. This study will involve administering surveys to loved ones of patients who are treated for injuries due to intentional violence at MUSC. They will be recruited via flyers and information sheets. Survey questions will address perceptions of their loved ones healthcare experience, interactions with staff members, impact of the Turning the Tide Violence Intervention Program (TTVIP), what perceived needs they have, and what services have been provided. Responses of those that have loved ones who enrolled in TTVIP compared to those who did not will be compared to assess for differences.