The study will test dordaviprone (also known as ONC201), a new drug that is being developed for treating people with glioma. ONC201 may help to remove tumor cells without affecting normal cells of the body. ONC201 is a first-in-class drug, which means it is a new drug having unique action.

The purpose of this study is to test the safety of FT825 at different doses and to understand the way the body processes and responds to FT825. The study will also find out what effects FT825, when given with or without a monoclonal antibody (cetuximab) and different chemotherapy regimens, have on cancer. FT825 is a type of cell product made up of “T cells.” T cells are part of your immune system and are important in helping fight infections. T cells are also important in eliminating cancer cells.

We are comparing the results of stool sample testing to colonoscopy for people who have Cystic Fibrosis (CF). We want to find out how effective stool sample testing is in detecting adenomas, including colorectal cancer.

This research is being done to determine if pectoserratus block with bupivacaine will provide improvement in pain control and decrease opioid use, relative to pectoserratus block with a saline solution, among patients undergoing total shoulder arthroplasty.

We are doing this study to see if we can lower the chance of endometrial cancer coming back and causing death by adding a drug or drugs that target HER2 proteins in addition to the usual combination of chemotherapy drugs. We want to find out if this approach is better or worse than the usual approach for your endometrial cancer. The usual approach is defined as care most people get for endometrial cancer, which in this case would be chemotherapy.

The study is divided into two portions. In the first phase, we want to test the safety of a drug called ipatasertib, by testing different doses of the drug to see which dose is safer for people when given in combination with a fixed dose of a drug called megestrol acetate (MA). In Phase II, we are studying how safe the treatment is and how well it works. We are doing this study because we want to find out if this approach is better or worse than the usual approach for endometrial cancer.

This study is for people who have emphysema (a disorder where too much air collects deep in the lungs) that is caused by the lack of a protein called alpha-1 antitrypsin (AAT) in the body. A deficiency of AAT (AATD) can damage lung and liver if not treated. The goal of this study is to evaluate the safety and study the therapeutic effects of INBRX-101 in subjects with AATD emphysema when compared with current approved AATD therapy with A1PI, known as Zemaira®.

The treatment (chemotherapy and transplant procedures) is considered standard clinical care that are usually given to the patients with this disease. The research aspect of this study is to collect data on the patients who are being treated on this plan. Patients will be followed throughout the course of their clinical care and for three years after their transplant.

The main purpose of this study is to check how safe the study drug is and how well your body handles taking it. We will also check if the study drug works to improve your kidney function, if has an impact on your daily life and the amount of the study drug in your blood over a period of time (called pharmacokinetics)

The objective of this study is to describe how activation of distinct pathways in and around the subthalamic nucleus (STN) and internal segment of the globus pallidus (GPi) correlate to changes in sleep outcomes in movement disorders patients after deep brain stimulation (DBS) surgery targeting these structures.

This is an early study of TAK-280 for people diagnosed with a type of cancer that cannot be treated or removed by surgery and the tumor is growing or has spread during or after other standard cancer treatment.

This study investigates a new treatment (TAR-200) for Muscle-Invasive Urothelial Carcinoma (bladder cancer). It assesses whether TAR-200 + Cetrelimab (experimental drug), is effective for patients scheduled for bladder removal surgery who can't undergo standard chemotherapy. The study compares two groups: one receiving TAR-200 + Cetrelimab, and the other receiving only Cetrelimab. The goal is to determine if this combination can provide an alternative treatment option for these patients with bladder cancer.

We are studying FT522 - a new product that is made by modifying cells in a laboratory - both with and without additional drugs, to see if it can help treat people with B-cell lymphoma. This study is for people who have had at least one treatment for their lymphoma, but the cancer either returned or did not respond to the treatment. We are testing this product to see what side effects it might have, as well as to see whether it is effective at treating B-cell lymphoma.

The research aspect of this trial is the use of a new machine to remove specific lymphocytes from the donor’s peripheral blood stem cells (PBSCs). This is called T cell receptor alpha/beta T cell depletion. This machine does such a good job at removing the unwanted donor T cells, and as a result we think patients will need fewer drugs to suppress their immune system.

We are studying the effects of stimulating the vagus nerve. The vagus nerve connects the brain to many organs in the body. Vagus nerve stimulation (VNS) is already approved by the United States Food and Drug Administration (FDA) to treat depression and epilepsy. We want to learn more about how it affects other parts of our bodies, such as the heart, metabolism, the immune system, and the nervous system. We hope that by understanding how VNS affects the body as a whole, we can develop new treatments for other conditions, or help to improve its use for depression and epilepsy.

The purpose of this study is to compare the effectiveness of megestrol alone, or combined with metformin, on the growth of Endometrial Intraepithelial Neoplasia (EIN). Participants will receive medication directed against EIN prior to the planned procedure (hysterectomy or progestin IUD placement). Women will receive either megestrol acetate pills by mouth twice a day for 3 to 5 weeks, or megestrol acetate and metformin pills twice a day by mouth for 3 to 5 weeks.

We are studying a new topical drug, Clascoternone, to treat adult males who have male pattern baldness. Participants randomly (by chance) receive the drug or an inactive solution that is applied to the area of hair loss twice a day for six months. We will compare the effectiveness and side effects of the two groups. Men who have regrowth of hair may also participate in a second six-month treatment to look at the long-term effectiveness of the drug.

Study objectives: -To characterize spontaneous and movement-related LFP changes in STN and GP in externalized patients under conditions that modulates the severity of tremor, bradykinesia and rigidity (off meds/off stim; on meds/off stim; off meds/on stim, on meds/on stim). -To characterize and compare the relative effect of different forms of closed loop stimulation (e.g., triggered at specific thresholds of low beta/HFO PAC or beta band activity) to standard isochronal high frequency DBS on motor signs and performance during movement.

1.1 Purpose: This protocol will carry out Aim 2 (Experiments 1 and 3) of Udall Project 2, leveraging the novel (on-label, FDA-approved) local field potential measuring capability of the Medtronic Percept™ PC DBS system to study the effects of globus pallidus internus and globus pallidus externus (GPi, GPe) DBS on: the wash-out and wash-in dynamics of motor behavior and local field potentials (LFPs) and correlations between fluctuations in gait and LFPs during activities of daily living (recorded over 4 weeks).

The goal of this study is to provide comprehensive longitudinal assessments of a cohort of PD patients before, during, and after DBS surgery, including neurological, neurophysiological, and neuropsychological data.

This is a prospective, randomized control trial that will assess neutralizing antibody titers in solid organ transplant (SOT) recipients that receive 2 doses of the high-dose (HD) influenza vaccine during a influenza season. We will evaluate the immunogenicity of 2 doses of the high dose influenza vaccine utilizing neutralizing antibody assays. The control group will receive 1 HD influenza vaccine and a placebo injection.

A pilot study looking at an adult-based non-pharmacologic interventional trial in collaboration with psychologist who has expertise in cognitive behavioral therapy (CBT) for adolescents with chronic disease and intellectual disability and developing processes towards independence in non-Sickle Cell Disease settings.

CP is a progressive fibro-inflammatory disease where EPI develops due to destruction of pancreatic parenchyma or pancreatic duct distortion. EPI results in maldigestion, leading to fat-soluble vitamin deficiencies, weight loss, malnutrition, and impaired quality of life (Qol). Signs and symptoms of EPI include abdominal bloating and cramping, diarrhea, foul-smelling, greasy stools (steatorrhea), and unintentional weight loss. Pancreatic enzyme replacement therapy (PERT) is the mainstay of treatment of EPI. Treatment is aimed at reduction of maldigestion-related symptoms, and prevention of malnutrition and its related morbidity and mortality. CREON® is a PERT that has been FDA approved since 2009 for the treatment of EPI due to cystic fibrosis, CP, pancreatectomy, or other conditions

This is a study of adults with unexplained chronic cough between 18-80 years old. This study is trying to determine whether a noninvasive vibrotactile stimulation device can help reduce cough symptoms.

Stem cell transplants (sometimes referred to as a bone marrow transplants) have been done for over 40 years but research continues to further refine the method to reduce side effects without affecting transplant success. The purpose of this study is to improve on transplant outcomes while reducing the potential side effects based on what has been learned from previous transplant studies using a reduced intensity preparative regimen. Information collected during this study (transplant outcomes and side effects) will be compared with the outcomes of the previous reduced intensity conditioning transplant study that enrolled more than 300 patients since 2002.

There are certain genetic changes in the leukemia cell thought to drive the disease in patients with acute myeloid leukemia. Ziftomenib is an investigational drug that blocks the menin pathway in hopes of preventing or slowing the leukemia cells from growing and dividing. The purpose of this study is to determine the safe dose of an investigational new drug (ziftomenib) used in combination with other study drugs i.e., venetoclax and azacitidine, to treat cancer. This will include an evaluation of side effects associated with ziftomenib in combination with the other study drugs and how ziftomenib works in combination with the other study drugs (venetoclax and azacitidine).

The purpose of this study is to learn if removing the donor T cells from the donor product using this new method will be a better way to reduce the risk of GVHD. The benefit of removing these cells with this new method is that they will prevent GVHD without requiring drugs to suppress the immune system. Potentially, the immune system will recover from the transplant faster, which in turn will also lessen the risk of severe infections. As well, the patient will not have the other common undesired side effects of these immunosuppressive drugs.

The purpose of this study is to find out what dose of CABA-201 can be safely administered to patients with SLE, including those with lupus nephritis (LN). SLE is thought to involve B cells that cause the body to attack different tissues in the body including your skin, joints, kidneys, heart, lungs, brain, and blood cells. LN is a type of kidney disease caused by SLE. CABA-201 is a chimeric antigen receptor T cell (CAR T) therapy. In this study, we will take some of your T cells, a type of white blood cell, and genetically modify them (put in a “code”) so that they may find and remove the B cells in your body, including the B cells that are involved in causing your disease. Once your cells are modified, CABA-201 cells will be re-infused into your body intravenously (through the vein).

The purpose of this study is to understand if the study medication ONCOFID-P-B is effective and safe in treating patients with carcinoma in situ of the bladder who have not received benefit from standard BCG treatment and are not candidates for radical cystectomy.

This study is being done to learn how well ripretinib works against cancer as compared to sunitinib in patients with a specific GIST-gene mutation who have received imatinib. We will also learn more about the safety of ripretinib and look at how ripretinib may affect your body. The choice of whether you will be given ripretinib or sunitinib will be assigned by a computer, by chance, like the flip of a coin. You will have a 2 out of 3 chances of receiving ripretinib. You will know if you are receiving ripretinib or sunitinib.