The purpose of this study is determine the optimal dose, efficacy and safety of an investigational drug (a new drug not yet approved by the U.S. Food and Drug Administration) in adults with Advanced Systemic Mastocytosis. The investigational drug is known as CGT9486 and will be taken daily orally. Participation in the study is expected to be approximately 6 years.

This study will evaluate the safety and effectiveness of fazirsiran (investigational drug) compared with placebo (an inactive substance) in patients with alpha-1 antitrypsin deficiency associated liver disease (AATD-LD). If eligible, subjects will be randomized (assigned to a group by chance) to receive either fazirsiran or placebo to be administered subcutaneously (an injection under the skin). Subjects will be treated on Day 1, at Week 4, and then every 12 weeks for 196 weeks. Subjects will be followed for 6 months after their last dose of study drug or placebo for a total study duration of approximately 230 weeks (including 10 weeks of the screening period which is the time needed to assess if a subject is eligible for the study).

This study evaluates whether non-cigarette tobacco products (e-cigarettes) can help smokers quit smoking as compared to traditional quit methods (nicotine replacement therapy or varenicline/Chantix). Participants in this study will be randomly assigned to one of two groups, then will have a choice between the offered products of that group. Participants in the e-cigarette group will have a choice of e-cigarette brand and flavor. Participants in the medication group will have choice between nicotine replacement therapy (patches and lozenges) or varenicline, also known as Chantix. Participation will last 6 months and will include weekly phone calls for the initial 7-weeks plus a 11-week phone call and a 6-month follow-up visit. Participants will also complete electronic daily diaries during the first 7-weeks.

The purpose of the research is to test out a combined treatment for depression where we stimulate a nerve in the ear while at the same time stimulating the brain with magnets. These treatments are called transcutaneous (through the skin) auricular (ear) vagus nerve stimulation (taVNS) and transcranial (through the skull) magnetic stimulation (TMS). For patients who already have a cervical VNS device, we will not change their treatment and will use this in place of the taVNS. We think this combined method might treat depressive symptoms better than either alone. This study is in person at the Institute of Psychiatry in downtown Charleston on the MUSC campus. First, patients will have a screening session and then will have 6 treatment days total where they will receive either VNS treatment alone, TMS treatment alone, or both at the same time. The treatment they start with will be randomized, and they will have 2 treatment days of each combination.

This study is for women with endometrial cancer. This study is being done to see if by adding a drug or drugs that target HER2 proteins in addition to the usual combination of chemotherapy drugs.

The need to advance knowledge and increase access to treatment for co-occurring posttraumatic stress disorder (PTSD) and substance use disorders (SUD) is a priority for service members and Veterans. PTSD and SUD often co-occur and are two of the most common and debilitating mental health disorders among service members and Veterans. However, few providers are trained in this approach and therefore are not prepared to deliver best practice care to Veterans who need integrated PTSD/SUD treatment.
This project will directly address this critical gap in provider training to improve the care of Veterans with comorbid PTSD/SUD, by providing development and testing of a web-based provider training course for concurrent treatment of PTSD and SUD Using prolonged exposure (COPE).

The primary objective of this platform trial is to evaluate the efficacy and safety of differing treatments in CF pulmonary exacerbations during a planned 14 day course of IV antimicrobials. Primary efficacy will be evaluated as the difference in mean ppFEV1 (pulmonary function testing) changes from Visit 1 to Visit 2 (Day 28 ± 2 days) between intervention arms. The study will compare treatment of an intravenous aminoglycoside and β-lactams (AG) versus intravenous β-lactams only (non-AG) in people with CF diagnosed with a pulmonary exacerbation who will be treated for Pa.

The purpose of this study is to develop and test if upper limb task practice and muscle activity training improve upper limb function in stroke survivors. Participants will be asked to come to the laboratory 3 times a week for 6 weeks to receive upper limb task practice and/or muscle activity training. Participants will also come to the laboratory for additional 3 visits for assessments of upper extremity function. The total duration of the study will be 2.5 months.

This study is for patients that have been diagnosed with locally advanced head and neck squamous cell carcinoma. The purpose of this research study is to evaluate the effectiveness of using a combination of pembrolizumab and olaparib when given before and after standard chemoradiation therapy in treating locally advanced head and neck squamous cell carcinoma. Pembrolizumab and olaparib are drugs that are approved for treatment of different cancers including lung, head and neck, breast and prostate cancer. However, FDA has not approved use of these two drugs together in treating head and neck cancer.

Treatment will be offered in three phases. In the induction phase, participants will receive a single infusion of pembrolizumab and will take olaparib tablets twice daily for total of 21 days. Participants will move to the chemoradiation phase, where they will receive radiation therapy and chemotherapy per routine standard care, for a total of 7 weeks. Chemoradiation therapy is done on a daily basis (excluding weekends), and chemotherapy therapy will involve a cisplatin infusion once weekly. At the conclusion of this phase, participants start the maintenance phase, which involves treatment with pembrolizumab and olaparib in cycles that are 42-days long. Treatment will include a single pembrolizumab infusion during each cycle and taking olaparib tablets twice daily during each cycle. Total number of cycles to be completed in the maintenance phase are 8 cycles. Participants can expect to be in this study for about 6.5 years.

Over 2400 people who have sickle cell disease and are between the ages of 15 and 45 have been enrolled into the National Registry (SCDIC-I) of patients with Sickle Cell Disease (SCD). A rich resource of natural history data, the SCDIC-I Registry has longitudinal data collected yearly since 2016 from patient surveys (e.g. self reported pain incidences, sleep, barriers to care, experiences during and after pregnancy), medical record abstraction (e.g. medications, transfusion history, co-morbidities) and laboratory results. The 150 patients (or 1200 among the 8 sites) will be selected from both MUSC adult and pediatric SCD clinics starting at 12 years of age; those not previously enrolled in the SCDIC National Registry will be offered the possibility to enroll in SCDIC-II.
We will look at the following:
1- Compare the effect of new SCD medications – crizanlizumab, voxelotor, and L-glutamine – on clinical outcomes in individuals with SCD.
2 - Identify genetic and genomic predictors of response to crizanlizumab, voxelotor, and L-glutamine
3 - Integrate study data into the CureSCi metadata catalog (MDC) to enhance future cross-study analyses.