This study is for subjects that have been diagnosed with lung cancer and the disease has progressed on prior therapy. The purpose of the study is to determine the safety and efficacy of Fingolimod and whether it can aid in treatment against lung cancer tumors. Fingolimod is not FDA approved and is considered an investigational drug. Subjects can expect to be in this study for about 8 months, with routine visits occurring at MUSC.

This phase III study is for participants with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) that is negative for a type of virus called human papilloma virus (HPV). If decided to participate in this study, participants will be receive either ficlatuzumab in combination with cetuximab, or placebo in combination with cetuximab. There are 3 Arms participants will be randomly assigned to: Arm 1 will receive ficlatuzumab (10mg/kg by intravenous (IV) infusion) and cetuximab (by IV infusion), Arm 2 will be given ficlatuzumab (20mg/kg by IV infusion) and cetuximab, and Arm 3 will receive placebo and cetuximab. The effectiveness of the 2 different ficlatuzumab groups (Arm 1 and Arm 2) will be compared at the first on-study scan, and the more successful Arm will began enroll more participants. This study is estimated to last approximately 5 years. Study drugs will be administered until disease progression or unacceptable toxicity, withdrawal of consent, death, or until the Sponsor terminates the study (whichever comes first). The main risk associate with the trial are swelling of your lower legs or arms, fatigue, shortness of breath, vomiting, diarrhea or constipation, bone pain, decreased appetite, anemia, high level of liver enzymes in your blood, dizziness, infusion reactions, cardiopulmonary arrest, pulmonary (lung) toxicity, skin reactions, inflammation of the mouth, low blood counts, liver problems, infection, headache, allergic reactions, and there may be unknown risks. This treatment can not guarantee the cancer will get better, since it may stay the same or get worse. What is discovered from this study may help other people in the future. The alternative to this study is not participating in this study and receiving HNSCC in patients who have previously received immunotherapy including chemotherapy with other treatment drugs or another clinical trial.

This study is for patients with hypertrophic cardiomyopathy (HCM). HCM is a condition where the heart muscle becomes abnormally thickened, which can sometimes block the blood flow out of the heart and results in the heart muscle working harder to pump blood to the body. Participants who have completed participation in a previous HCM study investigating the study drug, called aficamten (CK-3773274), will be eligible to participate in this study.

The study is done to collect long-term safety and tolerability data, including assessments of cardiac structure and function during chronic dosing with aficamten. Aficamten is a tablet taken by mouth. This is an open label study (the participants and study team will know the dose of aficamten taken at any given time). If your screening results show you are eligible to continue in the study, you will visit the research site for the "first dosing day" (Day 1), followed by visits at Weeks 2, 4, 6, 12, then every 12 weeks thereafter. Study related procedures include blood work, echocardiograms (ultrasound test of the heart), electrocardiogram (recording of heart's electrical activity), physical exams, and questionnaires. Risks associated with this study include shortness of breath, nausea, diarrhea, headaches and dizziness.

This study is for patient that have been diagnosed with rhabdomyosarcoma (RMS). The investigational drug in this study is vinorelbine, vincristine, dactinomycin, and cyclophosphamide (together called VINO-AC with vincristine). The purpose of this study is to compare the effects, good and/or bad, vinorelbine has on people with high risk RMS. Also, to find out if adding maintenance therapy will help get rid of the cancer and/or lower the chance that the cancer comes back. Participants can expect to be in this study for 12 months and will include chemotherapy, radiation and possibly surgery.

This is a Phase 2 study measuring the effectiveness and safety of an antibody treatment called AK117 combined with a drug called azacitidine in patients with newly diagnosed higher-risk myelodysplastic syndromes (HR-MDS). AK117 is an "investigational" (not yet FDA approved) treatment, azacitidine is FDA approved. The primary purpose of the study is to find the best dose of AK117 for future trials. The study will enroll approximately 90 patients randomized in 3 groups (like flipping a coin), with each group receiving either AK117 in doses of 30mg/kg, 20mg/kg, or a placebo, in combination with azacitidine. The study includes a screening period, treatment period, and follow-up period over the course of 3 years. Patients will receive AK117 or a placebo every 2 weeks in combination with azacitidine every 4 weeks. The main risk is that medical treatments often cause side effects. Patients may have none, some, or all of the effects listed or not listed in the protocol, and they may be mild, moderate, or severe. There is no direct benefit in participating in this study.

This study is an open label extension study for those who participated in the ION 682884-CS2 clinical trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a disease caused by change in a protein called transthyretin (TTR) which can result in a build up of this protein in parts of the body including the heart. This build up is called an amyloid deposit, and when this occurs in the heart it can lead to a condition called cardiomyopathy. This study involves the medication eplontersen, which is considered investigational meaning it is not approved for commercial use by the Food and Drug Administration (FDA). Eplontersen is aimed at preventing production of the TTR protein to slow or reverse disease progression. Eplontersen is given as an injection under the skin in the upper arm, stomach or thigh. This study will last about 3 1/2 years and include 16 clinic visits. Study procedures include physical exams, blood work, questionnaires, hall walk tests, electrocardiograms (tracing of the heart's electrical activity), echocardiogram (ultrasound test of the heart) and taking a Vitamin A supplement.

This study is to treat cutaneous squamous cell cancer that cannot be removed with surgery and is relatively large or has spread beyond its original location with the combination of radiation therapy and cemiplimab. Cemiplimab is FDA approved for this head and neck cancer, but the combination of cemiplimab and radiation is investigational. This treatment consist of 17 weeks of cemiplimab followed by 7 weeks of cemiplimab + radiation followed by 28 weeks of cemiplimab alone. There will be 3 clinic visits during treatment so the study doctor can check on the participant's health and see if they are having any side effects. Some of the most common risk and side effects that the study doctors know about are lack of energy or feeling tired (fatigue), loose or watery stools (diarrhea), rash, allergic reaction to study drug, and possible effects on liver, kidney, heart, and blood. Treatment will last a year with a follow-up call at the end of year 2.

This phase I trial will determine the maximum tolerated dose of lenalidomide when given in combination with high-dose systemic methotrexate and rituximab, with or without nivolumab, as induction treatment of primary central nervous system lymphoma. In addition, whether the combination of drugs can extend the control of CNS lymphoma by being used as maintenance (prolonged treatment) after control is achieved with the initial chemotherapy regimen (induction) will be judged. If decided to take part in the study, participants will complete pre-study testing, and if allowed to participate in study different people will get different doses of the study drug lenalidomide during induction chemotherapy. If the drug does not cause serious side effects, the next group of people in the study will get a higher dose, and the doses will continue to increase for every new group until people have serious side effects that require the dose to be lower. Lenalidomide will be taken by mouth on days 5 to 14 of each induction cycle. Once the dose of lenalidomide is found, the next group of people in the study will receive nivolumab in combination with the other drugs (methotrexate, rituximab, and lenalidomide). The first drug administered in each cycle is rituximab, which is given as an intravenous infusion typically in the infusion center. The day after rituximab, participants will be admitted to the hospital for the infusion of methotrexate. Enrolled participants that present benefit after induction will receive lenalidomide and nivolumab as prolonged therapy (maintenance) for an additional 12 months (12 cycles and each cycle is 28 days) or until the disease gets worse or the side effects become too severe. After treatment is completed the study doctor will continue to follow up on participants condition for 2 years to observe side effects. After 2 years the doctor will continue to follow up either in clinic or by phone for up to 5 years after registration. The most common side effects known are kidney damage, infusion reaction, blood clots, birth defects, immune toxicity, fever and infections, and there may be some risks that the study doctor is not aware of yet. Once the combination is proven safe, this study will allow for future studies to determine whether the combination of these four drugs can improve the response to treatment and help increase the understanding of their use in primary CNS lymphoma treatment. It is unclear whether these drugs will help participants live longer than the usual approach alone.

This study is for participants who have acute leukemia, a type of cancer of the blood and bone marrow. This study will help us learn more about a newly discovered oral drug, DSP-5336, that is being tested in people with acute leukemia and other types of blood cancer. DSP-5336 is a drug that interferes with a protein (Menin) in the body that has been found to have a role in developing leukemia. The main purpose of this study is to evaluate how safe DSP-5336 is at different doses and to find a recommended dose that is safe for further study. The overall planned study duration, including the above visits and periods, is 50 months (4.2 years).

This study is for patients who have been diagnosed with a solid tumor cancer that has continued to grow despite treatments patients have already received (non-small cell lung cancer or urothelial cancer). The study drug is FF-10832 (gemcitabine liposome injection). Gemcitabine is a cancer treatment registered in the US for the treatment of ovarian, breast, non-small cell lung, and pancreatic carcinomas. The study drug is a new, liposomal formulation of Gemcitabine. This new formula was developed to increase the amount of gemcitabine that goes to tumor cells. The study drug will be given to patients by itself, or in combination with pembrolizumab. Pembrolizumab is an approved treatment for many types of cancer. There are two groups that a participant may be assigned to, which group a participant is assigned on will be determined randomly, in a 1:1 ratio, like flipping a coin. The drugs will be given via an infusion. There is a 50% chance of being assigned to either group. Participation in the study will likely last 12 months, but participants may stay on the study longer if the study treatment continues to benefit them. The study consists of a screening visit, treatment visits, end of study visit, and a long-term follow-up.