This study is for participants that have been diagnosed with Intermediate or High-risk Primary or Secondary Myelofibrosis. This study is testing an investigational drug called Nuvisertib. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The main purpose of this study is to evaluate the efficacy and safety of Nuvisertib. Nuvisertib is an oral PIM1 selective inhibitor. A PIM1 selective inhibitor is a drug that specifically targets and blocks the activity of the PIM-1 kinase, an enzyme implicated in cancer cell growth. This drug is given to participants by mouth. Participants in this study can expect to be in the treatment phase of this study for 19 months and the long term follow up phase for 3 years.
A 144-week clinical research study to look at how well an investigational obesity medicine works, along with a reduced-calorie diet and increased physical activity in participants with obesity.
The medicines are injected subcutaneously (under the skin) once a week.
This study also includes a placebo.
This study is for patients that have been diagnosed with metastatic or locally advanced unresectable solid tumors with tumor protein 53 (TP53) mutation/loss with or without brain metastasis. This study is testing an investigational drug called AO-252. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to determine the maximum tolerated dose (MTD), the recommended phase II does (RP2D), and the safety profile of AO-252. The drug is given to participants orally. Participants can expect to be on this study for approximately 24 months, followed by a 12-month follow-up period.
This study is for male subjects that have been diagnosed with prostate cancer. Subjects are expected to remain in the study for a minimum of 12years or longer. There will be a total of 6 subjects locally enrolled. Subjects may experience the following risks: Bladder or prostate capsule perforation, Bladder neck contracture, Bleeding or blood in the urine, Bruising, Cancer progression, Electric shock/burn, Embolism, Incontinence or overactive bladder, and Infection.
This study is being done to learn more about the study drug known as AZD0780. AZD0780 is investigational meaning it has not yet been approved for commercial uses by the Food and Drug Administration (FDA) but has been approved for use in this study. This study will evaluate the efficacy, safety and tolerability of AZD0780. AZD0780 is taken once daily by mouth. This is a randomized study which means you will be assigned by chance, like flipping a coin, and you will have a 1 in 2 chance of being assigned to the study drug or placebo. Placebo is a substance that looks like the study drug but has no active ingredients. Neither you nor your study doctor or study team will know if you are assigned to study drug or placebo.
Participation in this study will last up to 4.5 years and include up to 15 visits. Study related procedures include medical history collection, physical exams, electrocardiogram (ECG – a test that records the heart's electrical activity), questionnaire and bloodwork. There are risks related to the study drug including increased blood pressure, shortness of breath and vomiting.
This study is seeking participants with BAG3-associated dilated cardiomyopathy (DCM). BAG3-DCM is a rare genetic disorder. Dilated cardiomyopathy is a condition that causes the heart to have a harder time pumping blood to the rest of the body which can lead to heart failure. Current treatment for BAG3-DCM is focused on improving heart function and preventing advanced heart failure with medicines, procedures and devices.
This study involves gene therapy. This will be the first time that a BAG3 gene containing study drug will be tested in human volunteers. The purpose of this research is to learn whether the investigational gene therapy RP-A701 is safe and effective for patients with BAG3-DCM. Gene therapy involves the addition of one or more genes to your cells to replace a missing gene or correct malfunctioning genes. Investigational means it is not currently approved by the Food and Drug Administration (FDA). RP-A701 will be given as a one time infusion into a vein in your arm.
Participation in this study will last about 2 years and include at least 18 visits including an inpatient hospitalization stay of at least 5 days. Study related procedures include review of your medical records, study drug infusion, immunosuppressant and antibiotic medications, echocardiogram (ultrasound test of your heart) exercise testing, electrocardiogram (recording of your heart's electrical activity), heart biopsy (collecting a piece of heart tissue), cardiac MRI, questionnaires, heart rhythm monitoring and ICD interrogations, and collection of blood, saliva, urine and stool collection. Study related risks related to gene therapy and those related to study procedures including risks of the heart catheterization, radiation, and biopsy, exercise testing, blood draw risks, genetic testing risks, the risk of loss of confidentiality and unknown risks.
This study is for patients that have been diagnosed with breast cancer. The purpose of this research is to assess the safety and efficacy of sacituzumab tirumotecan (sac-TMT) in people with certain types of breast cancer who will have surgery to remove their breast cancer. This trial will compare two treatment plans that patients will receive before their surgery. One of these treatment plans will involve patients receiving Sac-TMT and pembrolizumab (pembro), followed by chemotherapy and pembro. Patients enrolled in the other treatment plan will receive chemotherapy and pembro. Patients will be given the drug intravenously (a needle in a vein). Patients may experience a decrease in white blood cells, platelets, and red blood cells (anemia). Patients are expected to remain in the study for a minimum of 30 months or longer. There will be a total of 12 patients locally enrolled.
This study is testing a new medicine called Visugromab (CTL-002) to see if it helps people with a type of lung cancer called metastatic non-squamous non-small cell lung cancer. Everyone in the study will get standard cancer treatment, but some will also get Visugromab while others get a placebo (a look-alike with no active drug), and who gets what is decided randomly—like flipping a coin. The goal is to find out if Visugromab makes the treatment more effective and safe. The study will last up to two years, with Treatment in cycles lasting 3 weeks, about once a month. Researchers will closely monitor participants to see how well the treatment works and how their bodies respond. Serious risks of treatment may include heart problems, low oxygen levels, infections, organ failure, and inflammation in various parts of the body such as the lungs, liver, intestines, pancreas, and thyroid.
This is a clinical use of an FDA-approved Humanitarian Use Device (HUD) under a Humanitarian Device Exemption (HDE) called Miltenyi Biotec CliniMACS CD34. The intent is to treat patients with Myelodysplastic Syndrome (MDS) in a clinical setting, not to conduct research or gather data on safety or effectiveness. No data will be collected beyond what is required for standard clinical care. Device-related processing will occur at an NMDP Advanced Cell Therapy Lab Solutions facility, and the resulting cell product will be transported to MUSC Hollings Cancer Center for infusion. Eligible patients are in first complete remission and undergoing allogeneic stem cell transplant (SCT) from an HLA-identical matched related donor. Patients will be provided MUSC's standard treatment consent per institutional policies. The HUD will be stored, dispensed, and used at NMDP in accordance with standard operating procedures.
This study is for patients who have non-small cell lung cancer that is stage IV or has returned after remission. The goal is to compare the usual treatment by itself to the usual treatment plus a drug called cemiplimab. "Investigational" means this drug combination has not been approved by the U.S. Food and Drug Administration (FDA). A computer will randomly assign patients to one of two groups. This process is called "randomization." Patients will be placed into a group by chance, like flipping a coin, and will have an equal chance of being in Group 1 or Group 2. The drug is given by infusion. Patients will keep getting treatment until the cancer gets worse. Each treatment cycle lasts 21 days. After the last cycle, patients will be followed for up to 3 years.