This study is an open label extension of the ACT-EARLY study. which included those with no evidence of ATTR but are known carriers of disease causing TTR gene. ATTR stands for transthyretin amyloidosis. It is a condition in which a protein called transthyretin (TTR) accumulates in various organs, including the heart (known as ATTR-CM), kidneys, and nerves (known as ATTR-PN). This accumulation can lead to damage and dysfunction in these organs.

This study will continue using the study drug acoramidis (AG-10) to determine if it can help people with the genetic TTR variant slow the progression of ATTR. AG-10 is an investigational drug. Investigational means that AG-10 is not yet approved for use in any settings outside of clinical research studies like this one. Reducing the amount of TTR in your blood may reduce the amount of amyloid deposits in your body and may keep your cardiomyopathy from getting worse over time.

Participation in this study will last up to 60 month and will consist of about 13 clinic visits and about 11 telephone follow up visits. Some tests required include physical exams, medical and surgical history, bloodwork, questionnaire, electrocardiogram (test that records your heart's electrical activity), echocardiogram (ultrasound test of your heart) and study drug administration.

The purpose of this study is to test whether adding cetuximab to standard of care (pembrolizumab) is more effective in shrinking tumor size and increasing survival when compared to being treated with pembrolizumab alone. This study seeks to find if this approach is the same, better, or worse than standard of care for returning or spreading head and neck cancer after previous treatment.

Treatment and follow up for this study may be up to 5 years. The procedures include blood tests, CT or MRI scans, and chemotherapy. Risks include tiredness, anemia, constipation, loss of appetite, joint stiffness, cough, swelling and redness of the skin.

You may or may not receive a direct benefit from participating in this trial, however, information learned from the trial may help other people in the future. Both drugs, pembrolizumab and cetuximab, are already individually approved by the FDA for use in head and neck cancers. However, the benefit of combining the two drugs is being investigated in this study and this study approach is not FDA approved.

There will be about 158 people taking part in this study, approximately 4 subjects will be enrolled at MUSC.

This study is for subjects who have been diagnosed with recurrent or metastatic nasopharyngeal cancer. Subjects are expected to remain in the study for a minimum of 70 months. Drugs are FDA approved and is given through a vein (also called IV or intravenous). The procedures include blood and urine tests, troponin test. Risks include infection, bruising, bleeding, anemia, kidney damage, hearing loss, nausea, vomiting, numbness, pain, rash, blood in urine. You may not receive a benefit from participating in this trial, however, information learned from the trial may help other people in the future.

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.

The purpose of this research study is to find out if giving radiation therapy (RT) early to high-risk bone metastases that are not causing pain can reduce the chance of skeletal-related events (SREs) such as fractures, spinal cord compression, or surgery to bone. This will be compared to the current standard of care (SOC), which usually treats bone metastases only when symptoms like pain occur. The study will enroll 16 participants locally over 25 months, and each subject will remain in the study for at least 40 months. You are being asked to join because you have metastatic cancer that has spread to your bones and is considered "high-risk," though it is not causing pain at this time. High-risk bone metastases are typically located in the spine, hip, shoulder, or long bones, and larger tumors in these areas are more likely to cause complications. If you join this study, you will receive radiation therapy to high-risk bone sites in addition to standard care. The main risk is that radiation therapy may not work better than the usual approach at preventing bone complications. Radiation can also cause side effects, including skin changes, tiredness, and inflammation of the esophagus, bowel, or lungs. There may also be risks that are not yet known to the study doctors.

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.