There are currently two main ways of measuring nutrition in the Cystic Fibrosis (CF) population: body mass index (BMI) and laboratory values. This study plans to look at more ways to measure nutrition, and body composition, like the percentages of fat, bone and muscle in your body. One of the ways we will measure these items is by using dual energy X-ray absorptiometry (DXA) scan, which is a type of x-ray. This study hopes to provide researchers with more detailed information about nutrition and body composition in adults with CF.

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 phase III trial compares the safety and effect of adding vinorelbine to vincristine, dactinomycin, and cyclophosphamide (VAC) for the treatment of patients with high risk rhabdomyosarcoma (RMS). High risk refers to cancer that is likely to recur (come back) after treatment or spread to other parts of the body. This study will also examine if adding maintenance therapy after VAC therapy, with or without vinorelbine, will help get rid of the cancer and/or lower the chance that the cancer comes back. Vinorelbine and vincristine are in a class of medications called vinca alkaloids. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy. Cyclophosphamide is in a class of medications called alkylating agents. Vinorelbine, vincristine, dactinomycin and cyclophosphamide are chemotherapy medications that work by slowing or stopping the growth of cancer cells in the body. This trial may have the potential to eliminate rhabdomyosarcoma for a long time or for the rest of patient's life.

This phase I/II trial tests the safety, side effects, and best dose of selinexor given in combination with standard radiation therapy in treating children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG) or high-grade glioma (HGG) with a genetic change called H3 K27M mutation. It also tests whether combination of selinexor and standard radiation therapy works to shrink tumors in this patient population. Glioma is a type of cancer that occurs in the brain or spine. Glioma is considered high risk (or high-grade) when it is growing and spreading quickly.

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 conducting this research to: better understand barriers and facilitators of successful at-home care after an emergency laparotomy (EL) surgical procedure, create technology solutions for EL patient recovery tools to support at-home care and evaluate the success of the new at-home care tools. Participation includes a video-recorded or audio-recorded interview, either in person or virtual, with a study team member. The interview questions will ask about various topics related to the post-surgical experience. Prior to the interview, participants will also be asked to complete a 30-minute survey to help the study team prepare for the interview. Additionally, if a participant is having ongoing post surgery complications, they may be invited to opt-in to a second 60-minute interview.

One type of infection that is possible after bone marrow transplant is called an invasive fungal disease (IFD), a type of fungal infection that has the ability to spread throughout the body. In this study, rezafungin will be compared with the currently approved drugs for the prevention of IFD. The currently approved drugs are referred to as the standard antimicrobial regimen (SAR) which is posaconazole or fluconazole. We want to learn if rezafungin is safe and tolerable, if it is effective in preventing IFD compared to the standard treatment and to find out how much rezafungin is in blood over time after study drug has been given.

The purpose of this research is to determine the impact of providing personalized legal and financial services on the financial and emotional health of people with advanced stage colorectal cancer. We have partnered with a local nonprofit company called Cancer Legal Care (CLC) to provide the legal and financial services that people will receive as part of this research.

The main purpose of this study is to look at how safe and well tolerated the study drug is in combination with ribociclib (Group 1) or alpelisib (Group 2), the levels of the study drug and ribociclib or alpelisib in your blood, and how your body and your cancer respond.

There is evidence that the gut microbes interact with the body’s immune system, and people with pulmonary hypertension may have altered composition of gut microbes. We are studying whether transplanting gut microbes from healthy donors using a treatment called microbiota transplant therapy may have beneficial effects on pulmonary arterial hypertension.

There are 2 phases to this clinical research study: Phase 1 (dose escalation) and Phase 2 (dose expansion). The goal of Phase 1 is to find the recommended dose of the study drug IDP-023 that can be given alone (referred to as a “monotherapy”), with or without interleukin-2 (IL-2) and in combination with another anti-cancer drug, either daratumumab in subjects with relapsed/refractory MM or rituximab in subjects with relapsed/refractory NHL. The goal of Phase 2 is to learn if the recommended dose of IDP-023 found in Phase 1 with or without IL-2 can help to control advanced MM or NHL when given in combination with daratumumab or rituximab, respectively.

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.

Amblyopia (sometimes called 'lazy eye') is reduced vision in one eye caused by abnormal visual development early in life. The weaker (or 'lazy') eye often wanders inward and outward. Amblyopia is the leading cause of reduced vision in children and can lead to blindness if not treated. Treatments for amblyopia are glasses, and if needed, further treatment with part-time patching or penalization with atropine eye drops. Patching or atropine are administered to the stronger eye to force the child to use the weaker (amblyopic) eye. In recent years, an alternative type of therapy has emerged. It is called dichoptic treatment and involves stimulating both eyes simultaneously but with different stimuli. When it was first introduced, it was done in an office-based setting. Home-based technologies utilizing games and movies have been developed and studied to a limited extent in younger children with amblyopia. In this study, we will use a system called Luminopia. It uses a virtual reality headset to view web-based videos in which the contrast of the image seen by the stronger eye is reduced by 15% from that of the weaker eye. Luminopia has been available for use in the U.S. since 2022 and has been approved by the FDA for the treatment of amblyopia in this age group. In a previous large randomized trial, home-based dichoptic movies were shown to be superior to glasses alone but treatment effectiveness compared to patching (the gold standard for treating amblyopia) has not yet been established. If dichoptic therapy using the Luminopia system is confirmed to be at least as effective as patching, it would be an appealing alternative for treating amblyopia in young children, as it shows promise of better adherence and an easier treatment experience for the parent and the child. Children in this study would be randomized 1:1 to either the Patching Group or the Luminopia Group and followed for at least 6 months. Children in the Patching Group will have the option to do the Luminopia treatment after 6 months of patching. They will be followed for an additional 6 months. Thus, their participation will last for a total of 1 year.

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

The purpose of this screening study is to collect samples to conduct the testing of specific human leukocyte antigen (HLA). TScan Therapeutics is developing cellular therapies across multiple solid tumors in which the eligibility criteria require that participants have specific HLA types. The results from this screening study will be used to determine if participants meet the eligibility criteria and could potentially be enrolled in a future TScan treatment study.

A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone (fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who are unable to receive additional anthracyclines, or in 2nd relapse.

We are doing this study to check how well semaglutide can help adolescents with excess body weight to lose weight and to maintain weight loss. This study will mainly look at long-term maintenance of weight loss and the long-term safety of semaglutide in children with excess body weight.

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 purpose of this research study is to investigate new magnetic resonance imaging (MRI) methods to better detect and monitor osteonecrosis of the femoral head (ONFH) before and after treatment. ONFH causes injury to the hip joint that can lead to osteoarthritis (the breaking/wearing down of cartilage & tissues within the joint) and the eventual need for a hip replacement. It can be difficult to detect ONFH early on using current medical imaging techniques, which is when treatments may be the most effective. Furthermore, available treatments are not always effective at preventing the progression (spread or growth) of ONFH. This research may benefit others with ONFH by providing more effective medical imaging tools to detect ONFH earlier and inform treatment decisions to increase the chance of stopping or delaying the progression of ONFH and preventing hip osteoarthritis.

This phase III trial tests how well adding dinutuximab to induction chemotherapy along with standard of care surgery radiation and stem cell transplantation works for treating children with newly diagnosed high risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found in greater than normal amounts on some types of cancer cells. This helps cells of the immune system kill the cancer cells. Chemotherapy drugs such as cyclophosphamide, topotecan, cisplatin, etoposide, vincristine, dexrazoxane, doxorubicin, temozolomide, irinotecan and isotretinoin, 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. During induction, chemotherapy and surgery are used to kill and remove as much tumor as possible. During consolidation, very high doses of chemotherapy are given to kill any remaining cancer cells. This chemotherapy also destroys healthy bone marrow, where blood cells are made. A stem cell transplant is a procedure that helps the body make new healthy blood cells to replace the blood cells that may have been harmed by the cancer and/or chemotherapy. Radiation therapy is also given to the site where the cancer originated (primary site) and to any other areas that are still active at the end of induction.

The purpose of CureGN2 is to gather a group of people with glomerular disease to create a source of information and blood and urine samples, so that researchers can easily and effectively study glomerular disease.

The aim of this study is to investigate whether voclosporin, added to standard treatment, is able to reduce activity of lupus nephritis over a study treatment period of 24 weeks, and to determine its safety as well as the best dose for treatment of lupus nephritis in children or adolescents.

Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The study will look at the safety, efficacy, and pharmacokinetic (PK) trial in children ≥1 to <18 years treated for up to 108 weeks with the drug sparsentan.

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 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.

We are looking at a new drug called 'ziltivekimab' to see if it can be used to treat people living with heart failure and inflammation. People will get either ziltivekimab or a placebo (inactive dummy drug). The study drug is an injection given into the fold of the skin on the stomach, thigh or upper arm once every month. People will take the study drug for up to 4 years.

Cyclophosphamide is a chemotherapy (chemo) drug often given after a transplant to prevent graft-versus-host disease (GvHD). We are doing this study to see if a lower dose of cyclophosphamide after transplant is as safe and works just as well. This study does not include any new or untested drugs. The drugs and procedures in this study are standard for people who receive a transplant.

This research is being done to determine whether the investigational drug tabelecleucel (allogeneic Epstein-Barr virus-specific cytotoxic T lymphocytes [EBV-CTLs]) can help people with EBV-associated diseases.

The primary objective of this study is to assess the overall safety of VOR33 in participants with acute myeloid leukemia. VOR33 is a genome-edited hematopoietic stem and progenitor cell therapy product. Other objectives of this study include assessing the safety and tolerability and identifying the maximum tolerated dose of Mylotarg, which is an antibody-drug conjugate already approved by the FDA for adult patients with acute myeloid leukemia.

We are doing this study to learn more about anifrolumab (SAPHNELOTM) in patients with systemic sclerosis and to better understand the studied disease and associated health problems. Systemic sclerosis (scleroderma) affects the skin as well as other organs, such as blood vessels, muscles and joints, digestive tract, kidneys, lungs and heart.