This study is enrolling participants with symptomatic ATTR-CM (transthyretin amyloidosis cardiomyopathy). ATTR-CM is a rare and serious disease that occurs when a protein in the blood called transthyretin (TTR) builds up throughout the body, including in the heart and nerves. When the abnormal protein, known as amyloid, deposits in the heart, the heart muscle thickens and stiffens, causing the heart to fail. This research study is designed to test whether the medication nucresiran is safe and helps people with ATTR-CM, in comparison to the effects of placebo.

Nucresiran is considered investigational, meaning it is not currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of ATTR-CM. Nucresiran is a TTR silencer. It is like a "quiet button" that turns down the amount of the disease-causing protein that is made. Because there is less TTR, there may be less buildup in the heart and other organs over time.

This is a randomized study meaning once eligibility is confirmed, participants will be assigned by chance, like drawing straws, to either receive nucresiran or placebo. You will have a 2 out of 3 chance of being assigned to nucresiran and a 1 out of 3 chance of being assigned placebo. Placebo is a substance that looks like the actual medication and is given the same way but contains no active substance. The study drug, which can be either nucresiran or placebo, will be given as an injection under the skin in the abdomen (avoiding the area around the navel), thigh, or the side or back of the upper arms. Neither the participants nor the study doctor will know who is assigned to nucresiran or placebo but this information can be made available if need be.

Participation in this study is expected to last for 5-8 years, Study related procedures include physical exams, vital signs, echocardiograms (ultrasound test of the heart), electrocardiograms, (ECG, a tracing of the heart's electrical activity), blood work, urine samples and questionnaires. Participants will also take Vitamin A daily. Study related risks include risks related to the study drug including injection site reactions, abnormal liver function or an allergic reaction. There may be risks related to study procedures including loss of confidentiality. There may not be any direct benefit, but the information learned may benefit others with ATTR-CM in the future.

This study is for male subjects that have been diagnosed with metastatic prostate cancer (that has spread to other parts of the body) and progressed following standard hormonal/radiation therapy and surgery. Subjects are expected to remain in the study for a minimum of 48months or longer. There will be a total of 10 subjects locally enrolled.

This study is for subjects who has been diagnosed with radioactive iodine refractory (RAIR) differentiated thyroid cancer. Subjects are expected to remain in the study for a minimum of 96 months. Drugs are FDA approved and is given in the form of Tablet to subjects. The procedures include urine protein test, CT, MRI. Risks include diarrhea, nausea, vomiting, tiredness, weight loss, loss of appetite, changes in taste, redness, pain or peeling of palms and soles, High blood pressure. There is evidence that dabrafenib, trametinib and cabozantinib are effective in stabilizing and shrinking the type of cancer, we do not know which of these approaches are better at prolonging time until tumor growth. However, information learned from the trial may help other people in the future.

This study is for subjects that have been diagnosed with low-grade upper tract urothelial cancer (LG-UTUC). This study is to evaluate the tumor ablative effect of the study drug (UGN-104). Subject are expected to reman in the study for a minimum of 15months or longer.

This study is for patients that have been diagnosed with previously untreated, locally advanced, and metastatic pancreatic ductal adenocarcinoma PDAC. This study is testing two treatment regimens: NALIRIFOX (5-fluorouracil, liposomal irinotecan, oxaliplatin, and leucovorin) vs mGAP (gemcitabine, nab-paclitaxel, and cisplatin).
The primary purpose of this study is to see which of the two regimens is more effective in PDAC. Participants will continue on study medications if seeing clinical benefit, and can expect to be on the study for a maximum of 6 months.

This phase 2 study is enrolling patients who have acute myeloid leukemia (AML) with certain biomarkers. This study is being done to see the effectiveness of different combinations of drugs to treat AML. It will involve 3 groups of patients receiving different combinations of Gilteritinib, Azacitidine and Venetoclax. Gilteritinib is an investigational drug, Azacitidine and Venetoclax are FDA approved. The main purpose of the study is to see if the amount of leukemia in the patient's body can be lowered by adding the drug Gilteritinib to the Standard of Care of treating AML with Azacitidine and Venetoclax. The study will include approximately 147 patients. The patients will be randomized into the three groups (like flipping a coin), Group 1 will receive just Azacitidine + Venetoclax, Groups 2 and 3 will also receive Gilteritinib but Group 2 will receive it for more time within a treatment cycle. Patients will complete screening after participating in this treatment trial or SOC therapies. Patients will continue treatment until disease progression, unacceptable toxicity, study closure, death, or withdrawal of consent. The main risk is that the study drugs may not be as good as the usual approach for their cancer or condition at shrinking or stabilizing their cancer. Patients may have none, some, or all of the side effects listed or not listed in the protocol, and they may be mild, moderate, or severe. There is no direct benefit for them in participating in this study.

This study is patients that have been diagnosed with high-risk renal cell carcinoma. This study will compare disease free survival (DFS) in patients treated with adjuvant pembrolizumab and tivozanib versus those receiving pembrolizumab alone. Participants can expect to remain in the study for 10years. There will be a total of 14 patient enrolled locally.

This phase 3 study is recruiting patients who are at risk of graft-versus-host disease (GVHD) after a bone marrow transplant. This study will measure the safety and effectiveness of a prevention treatment combination called Tacrolimus/Methotrexate/Ruxolitinib compared to Standard of Care (SOC), Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil. Ruxolitinib (Rux) is an approved treatment for GVHD. This study is divided into two parts. The first part, called the run-in phase, will investigate the best dose of Ruxolitinib. The second part of the study will compare the SOC combination therapy with the investigational combination therapy (which will include Ruxolitinib). Participants will be randomly assigned to one of the two groups (like flipping a coin). The study will enroll up to 572 patients nationwide and 5 at MUSC. The participants can expect to be involved in the study for at least 24 months. The main risk is that medical treatments often cause side effects. The most common side effects expected from the investigational combination therapy are high cholesterol, increased liver enzymes, low platelet levels, and low red blood cell counts. There is no direct benefit for them in participating in this study.

The purpose of this study is to test the effectiveness and safety of the combination of an investigational vaccine, PDS0101, given by subcutaneous (beneath the skin) injection and the standard of care, Pembrolizumab (KEYTRUDA), given intravenously ("IV;" into the vein) compared to pembrolizumab (KEYTRUDA) alone for those with unresectable recurrent or metastatic head and neck cancer and high risk papillomavirus infection. "Investigational" means the combination of the study vaccine and pembrolizumab being tested has not been approved by the US Food and Drug Administration (FDA) nor any other Health Authority.

This study is for patients that have been diagnosed with advanced solid tumors. This study is testing an investigational drug called BNT317. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to evaluate the safety, tolerability, absorption of BNT317. BNT317 is administered via intravenous (IV) infusion. Participants can continue to receive this study drug until it no longer gives them benefit. Researchers will continue to follow-up with patients long-term.