The purpose of this study is to evaluate the safety and ability of a transplant with your own gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. The goal is to determine if a sufficient amount of hemoglobin that prevents red blood sickling can be produced after the gene modified stem cells are returned to your body. This study may provide information on the potential usefulness of bb1111 for treatment of sickle cell disease

The goal of this study is to test the safety and effectiveness of tildacerfont in children with congenital adrenal hyperplasia (CAH). When a child is enrolled in the study, in addition to taking the study drug (tildacerfont), he or she will continue to take his or her standard glucocorticoid doses. A part of the study will be to test different doses of the study drug and to measure adrenal hormones at each visit. Children will be in the study for 18 weeks and will have to visit the study clinic 5 times.

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 purpose of this study is to compare the treatment of carboplatin/paclitaxel and letrozole hormonal therapy to letrozole alone. Letrozole is a drug called an aromatase inhibitor, which indirectly stops the body from producing estrogen. The use of the hormonal therapy drug, letrozole without chemotherapy may shrink or stabilize your cancer in the same way that chemotherapy also does, but without the added side effects of chemotherapy. Half of women in this study will receive letrozole with paclitaxel/carboplatin and the other half will receive letrozole alone.

The purpose of this study to examine the effects of cigarette smoking and drinking alcohol on the formation of DNA damage in the mouth. The overall goal is to identify DNA damage that may be important to the development of head and neck cancers.

This research trial studies the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.

The purpose of this research study is to obtain information on the safety and effectiveness of PBCAR0191 to treat certain types of cancers, such as Non-Hodgkin Lymphoma and B-cell Acute Lymphoblastic Leukemia. It is made from a type of blood cells known as T cells. The T cells in PBCAR0191 came from people who have donated their blood. The donated T cells have been genetically changed, so that they may be able to kill specific cancer cells commonly present in Non-Hodgkin Lymphoma and B-cell Acute Lymphoblastic Leukemia.

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 see the efficacy (how well something works) of study treatment (tarlatamab/SOC chemotherapy) and whether it causes any side effects when treating small cell lung cancer (SCLC) that has progressed (become worse) after initial platinum-based treatment.

This purpose of this study is to identify a safe dose level for the study drug, E7777, when given with standard tisagenlecleucel therapy (also known by its brand name, Kymriah, is an immunotherapy that is made from the participants own blood cells) in participants with Diffuse Large B-Cell Lymphoma (DLBCL). Up to three dose levels of E7777 will be tested.

The purpose of this study is to establish a registry and biobank to study monoclonal gammopathy and amyloidosis. We aim to understand better the changes these diseases cause, the symptoms they present, and the clinical progression to be able to better prevent them and treat them in the future.

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 looking at the effectiveness of adding an immunotherapy drug, pembrolizumab, to usual treatment for people who have salivary gland cancer that can’t be treated with surgery or radiation. The cancer must be androgen receptor positive.

This study is looking at whether giving a new type of experimental medicine, called JSP191, can prepare the body to help the stem cell transplant work better, so the immune system can grow and fight infections. The study doctor and Sponsor also want to see how safe and well tolerated this experimental medicine is. They will study whether it is safe to give to patients and look at how much medication to give and what side effects may occur. During this study, the optimal dose of JSP191 will be determined and additional patients will be enrolled in this study using that dose level.

The investigational therapy in this study is referred to as Restorative Microbiota Therapy (RMT). It is prepared by extracting healthy bacteria from the stool of healthy human donors and making it into capsules taken by mouth. The donor stool samples are rigorously tested for harmful bacteria and viruses before processing. There is scientific evidence to suggest that RMT might make immunotherapy more effective. The primary goal of the study is to test if RMT makes durvalumab + tremelimumab treatment with chemotherapy more effective to control lung cancer.

The purpose of this study is to test the safety of FT576 at different doses and schedules and to understand the way the body processes and responds to FT576. The study will also find out what effects FT576, when given alone (also called monotherapy) or in combination with an anti-cancer drug (called a monoclonal antibody), may have on you and your cancer. FT576 is a type of cell product made up of “natural killer” or NK cells. NK cells are a type of immune blood cell that are known to attack cancer cells.

This protocol aims to characterize the safety and tolerability of loncastuximab tesirine in combination with gemcitabine, lenalidomide, polatuzumab vedotin, or umbralisib, and to identify the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) for any of the combinations in subjects with relapsed or refractory B-cell Non-Hodgkin Lymphoma. This project aims to address the resistance mechanisms to single agent therapies and enhance efficacy by engaging different targets, in synergistic or additive manner.

This study it to test an investigational vaccine called mRNA-1647 that is being developed for preventing cytomegalovirus (CMV) infection in people. CMV is a common virus that can spread easily through an infected person’s saliva or other body fluids such as blood, urine, and breast milk. We want see if the trial vaccine can prevent CMV infection in participants who have not been previously infected, to understand the safety (how many side effects you may have) of the trial vaccine, and to see if the trial vaccine results in participants making antibodies to CMV.

Researchers want to find out more about how physical, cognitive, and emotional factors affect eating.

We are doing this study to see if we can improve the standard treatment for OCD, Exposure with Response Prevention, by pairing it with Transcranial Magnetic Stimulation to the parts of the brain that cause OCD symptoms.

This study is being performed to determine the safety and tolerability of ZN-c3 alone and the combination of ZN-c3 and ZN-d5 in Acute Myeloid Leukemia (AML). We want to identify the best doses of the study drugs and learn if either drug effects the blood levels of the other. We will also assess how effective the Study Drugs are in treating AML and explore whether certain aspects of AML can predict whether leukemia responds to the study drug(s).

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 purpose of this study is to evaluate if a partial enteral nutrition diet, with a pea protein plant-based oral nutrition supplement (ONS; Kate Farms Peptide 1.5), combined with the Inflammatory bowel disease - Anti-Inflammatory Diet (IBD-AID) improves the nutritional intake of adult patients experiencing a CD flare initiating immunologic therapy compared to standard of care. Standard of care for patients experiencing a CD flare is commonly characterized by prescription of a low fiber diet and either lack of oral nutrition supplementation or use of an animal protein based supplement.

This study will look at an investigational study drug, called olorofim, to determine how safe the study drug is, how well it is tolerated and whether it is effective compared to AmBisome® (a standard of care treatment) to treat invasive fungal disease (IFD). We expect that you will be in this research study for up to 18 weeks or just over 4 months.

The purpose of this study is to determine the most effective ways to get rid of Staph aureus on body surfaces before surgery. We will determine if the participants can effectively get rid of the bacteria with the simple application of various treatment methods assigned to them. We will study if these methods are useful and cost effective in preventing the infections after surgery.

The purpose of this study is to implement an intervention using a Follower, Action plan, and remote Monitoring (FAM) of glucose data to reduce severe hyperglycemia in adults with Type 1 Diabetes Mellitus at risk for diabetic ketoacidosis. You and your chosen “follower” (family member, caregiver, or friend) will be asked to attend 6 visits (in- person or remote) as a pair (“dyad”) with the study team that will last up to 30 minutes to 2 hours each across a span of 4 months.

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.

This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.

Osteoarthritis (OA) is a leading cause of disability and chronic pain that reduces physical activity and daily activities. In this clinical research study, the goal is to learn more about geniculate artery (located in the knee) embolization (GAE) treatment to see if it will reduce pain as well as stiffness and difficulty performing daily activities caused by knee OA and if it can be performed safely.