The purpose of this study is to test a new drug, called ST-920, to see if it is safe and if it works to treat Fabry disease. ST-920 is a gene therapy treatment, which means that ST-920 replaces the missing or broken gene you have because you have Fabry disease, with a version or copy that works.

This study is looking at glucose (blood sugar) patterns in participants with Turner Syndrome between the ages of 3-80 years old. This will be done by obtaining 2 hours frequent blood sampling by completing an Oral Glucose Tolerance Test and a Mixed Meal Tolerance Test. Participants will also wear a continuous glucose monitor for 2 weeks. Along with the OGTT and MMTT, participants are asked to provide a blood sample for DNA and RNA testing. Participant’s parents are asked to provide a saliva sample for DNA testing

This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

There is no consensus on the radiation dose required to relieve the pain from bone lesions from multiple myeloma. Usually, patients receive between 10 and 12 radiation treatments to achieve pain relief. But a shorter course of radiotherapy may be more effective. This study will evaluate whether pain relief can be achieved using only 1 or 2 radiation treatments, which will be delivered to a painful bone lesion. Your study doctor will decide whether you receive 1 or 2 treatments. The total amount of radiation you will receive will be the same whether it is done in one or two treatment sessions.

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.

Aromatase inhibitors (AIs) are commonly used in treating hormone-positive breast cancer. Unfortunately, many patients receiving this treatment experience Aromatase Inhibitor-Associated Musculoskeletal Syndrome (AIMSS), with symptoms like joint and bone pain and joint stiffness. The current therapies used to improve AIMSS symptoms have limited effectiveness and can cause their own side effects. In this research study, we are examining the feasibility of topical medical cannabis cream as a treatment option for AIMSS.

This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients (365 Days to 31 Years) with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in body's immune system and may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better then combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.

CGD disorders are a group of diseases that cause the immune system to function abnormally, leading to infections, autoimmunity and/or inflammation that can begin early in life. CGD is usually caused by changes in certain genes in DNA. Researchers are trying to learn what types of medical problems patients with CGD have and how these respond to treatment, including bone marrow transplantation and gene therapy. Researchers also want to learn which genes cause CGD and how specific gene abnormalities lead to medical problems.

This is a study designed to evaluate the safety, tolerability, and efficacy of a one-time gene therapy (BBP-631) for adult patients diagnosed with classic congenital adrenal hyperplasia (CAH). The goal of gene therapy for CAH is to give the body a functioning CYP21A2 gene using a vector (an agent used to deliver a gene into the body). Having a functioning CYP21A2 gene in the adrenal gland may allow the body to naturally produce its own cortisol and aldosterone. The study treatment and follow-up lasts 1 year with a long-term follow-up of 4 more years.

This research study is designed to compare a new gene therapy, known as OTL-203 (study drug), with a standard treatment called “allogeneic hematopoietic stem cell transplant” (allo-HSCT), to find out which is better for the treatment of MPS-IH.

The purpose of this study if to determine if stool or blood can be used to detect colon cancers as early or earlier than colonoscopy. The researchers plan to use these samples to learn about specific proteins (also known as biomarkers) that may indicate colon polyps, colon cancer or an increased risk of developing colon cancer. In order to learn more about preventing and detecting colon and rectal cancer, we are collecting samples from subjects with cancer, adenomas, and colonoscopies who may be at risk for polyps.

We are enrolling people who have had complete surgical removal of a stage II or stage III colorectal cancer. We will draw blood samples for circulating tumor DNA (ctDNA) to find out if this blood test can be used to detect recurrence of the cancer. People will be followed for at least 3 years and up to 5 years.

Regular participation in physical activity helps maintain a healthy weight, improves energy levels and overall health. Children and teenagers who have received treatment for cancer are often less active, may gain weight and have more health problems as compared to children and teenagers who have not received treatment for cancer. This study looks at physical activity and its effect on your health. This study will use a variety of interventions to see if they affect how active you are over time.

This project is a data and specimen repository for developmental disorders. Participants provide biological samples and permission to store their health-related data. The purpose is collect and manage these materials for use in biomedical research related to developmental disorders.

We are studying how well radiation therapy with or without olaparib works in treating people with inflammatory breast cancer. Olaparib may keep cancer cells from repairing themselves, making them die. We want to see if adding this drug to radiation therapy is more effective.

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).

We are studying an internet-based pain coping skills program with pain education for cancer survivors who have persistent pain. Pain severity and interference will be compared to people who receive only pain education. The pain coping program has 8 sessions that are completed in the first 10 weeks of starting the study. The group that receives only pain education will receive access to the program after 6 months. Everyone is followed for 9 months

Nephrotic syndrome is a condition which affects the kidneys causing them to leak protein from the blood into the urine. Nephrotic syndrome is a disease that can improve (remission) and worsen (relapse) at different times throughout childhood. By collecting health information and laboratory samples, our goal is to learn more about these kidney diseases and find better ways to prevent and treat people with nephrotic syndrome. New knowledge will be shared with researchers and the public.

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

We are studying the addition of a drug to the treatment for people who have triple-negative breast cancer. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells. Some people will receive the current treatment and others will have the current treatment with carboplatin added. The results of the two treatments will be compared.

This study will enroll individuals with cancer to test a prototype mobile application which will measure and summarize the time spent on cancer care related tasks.

We are comparing treatment of early stage, hormone positive breast cancer with and without radiation therapy. One group will receive radiation therapy with endocrine therapy and the other group will receive endocrine therapy alone. We want to find out if there is any difference in how often breast cancer recurs in the same breast.

We are looking at how well letrozole with or without paclitaxel and carboplatin works in treating patients with stage II-IV low-grade serous carcinoma of the ovary, fallopian tube, or peritoneum. Letrozole is an enzyme inhibitor that lowers the amount of estrogen made by the body and may stop the growth of tumor cells that need estrogen to grow. We will compare the effectiveness of the two different treatments.

This study is looking at how well serum tumor markers work to monitor people who have hormone receptor positive Her2 negative breast cancer that has spread to other places in the body. We want to see if using the markers (from a blood sample) is as good as using scans to monitor disease.

We are looking at adding lomustine to temozolomide and radiation therapy when compared to temozolomide and radiation therapy alone (usual care). We will compare the effect (shrinking or stabilizing) and side effects when treating newly diagnosed MGMT methylated glioblastoma. Each of the drugs and radiation work in a different way to stop the growth of tumor cells.

This is a research study to find out if giving a drug called pembrolizumab after lung cancer surgery does a better job at keeping the cancer from coming back than surgery alone.

The purpose of this study is to learn about the effectiveness and safety of a new study drug called AFM13 when used in combination with a new cell therapy called AB-101. AFM13 is an antibody designed to bind to cancer cells and to “natural killer” cells. AB-101 refers to natural killer cells that were obtained from human umbilical cord blood. Natural killer cells are part of your immune system and their primary function is fighting infections and cancer. AFM13 binds the natural killer cells and links them with the cancer cells, so they can eliminate the cancer cells.

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.

This trial compares the effect of bevacizumab and osimertinib combination vs. osimertinib alone for the treatment of non-small cell lung cancer that has spread outside of the lungs and has a change (mutation) in a gene called EGFR. Sometimes, mutations in this gene cause EGFR proteins to be made in higher than normal amounts on some types of cancer cells. This causes cancer cells to divide more rapidly.

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.