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.

We are conducting a research study for children ages 6-17 with patchy Alopecia Areata (AA). The purpose of this research study is to learn more about the safety, tolerability and efficacy of an investigational drug called Baricitinib. This study will compare the investigational drug to a placebo (inactive substance) to see how well the investigational drug works.

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.

This study is testing different ways to look for neurologic side effects in patients who get CAR-T therapy for their cancer.

The purpose of this research study is to compare the transfusion of cold-stored (refrigerated) platelets to standard room temperature stored platelets. The goal of the trial is to determine whether platelets stored cold are similar or better at stopping bleeding compared to platelets stored at room temperature and, if so, to determine the maximum duration of cold storage that maintains a similar effect on bleeding

The goal of this study is to better understand how drinking alcohol may lead to oral cancers. Acetaldehyde, a chemical formed when the body breaks down alcohol, is believed to play an important role. This study will measure acetaldehyde and DNA damage levels in the mouth of participants after a low dose of alcohol. The levels will be compared between three groups, all having different degrees of risk for developing oral cancer, in order to identify DNA damage that might be crucial to cancer formation.

The purpose of the trial is to evaluate the effect and safety of imlifidase when given to participants with antiGBM disease (also called Goodpasture disease). We will study if the addition of imlifidase to the standard of care treatment results in a better effect without causing unacceptable side effects compared to standard of care alone.

The objective of this study is to create an international registry with long-term follow-up to characterize and evaluate the safety of abatacept in juvenile idiopathic arthritis (JIA). The primary objective of the JIA registry is to describe the long-term safety of abatacept treatment for JIA by quantifying the incidence rates of serious infections, autoimmune disorders, and malignancies.

To assess the safety of Afrezza in a pediatric population when compared to the usual standard of care insulin.

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.

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.

The purpose of this study is to investigate how effective and safe ZYN002, a transdermal gel, is in participants with FXS. The drug product ZYN002 is a pharmaceutically manufactured CBD. It is being developed as a clear gel that can be applied to the skin (called transdermal delivery), to provide consistent, controlled levels of CBD in the blood when it is given twice a day. Participants will be assigned by chance to get one of the following study treatments: Active study drug – ZYN002 or placebo. Assigning study drug by chance is called “randomization,” and it is an important part of testing an experimental study drug. Participants will be randomly assigned to study treatment according to a computer program and will have 1 in 2 chance of receiving the active study drug.

One goal of this study is to find out if enfortumab vedotin is effective and safe as a treatment for people with breast, lung, head and neck, gastric, gastroesophageal junction, or esophageal cancer. Researchers will look at how enfortumab vedotin can act in the body. Enfortumab vedotin is expected to work by attacking cells that have a protein called Nectin-4, commonly found in cancer cells. Another goal of this study is to find out if enfortumab vedotin is effective and safe when combined with another US Food and Drug Administration (FDA) approved medicine, pembrolizumab (brand name KEYTRUDA®), and used as a treatment for people with head and neck cancer who have not received previous chemotherapy treatment other than the chemotherapy that may have been given in combination with radiation therapy or right before or right after surgery in the past.

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.

Black Americans are 60% more likely to be diagnosed with Type 2 Diabetes (T2D) and 2 times more likely to die from T2D than non-Hispanic White Americans. Recent research has shown that sedentary behavior (such as sitting or lying down) have a significant negative impact on health. We are doing a small study with older people who identify as Black or African American and have T2D to look at ways to reduce sedentary behavior. The study will last about 1-2 months for each participant.

PR001A is designed to deliver a normal GBA1 gene copy into the body to increase the activity of GCase, which is low in Type 2 Gaucher Disease (GD2) patients. The new GBA1 gene will remain a child’s body cells for many years and possibly for the rest of their life. A participant will need one surgery during which the study drug will be given and will stay in the hospital for at least 48 hours following the surgery.

This research is being performed to study whether an investigational ultrasound treatment of the spleen can reduce the symptoms of Long COVID by reducing inflammation in the body. Ultrasound is widely used in human medicine because it is designed to be safe, non-invasive, and painless. The same kind of ultrasound that is used for imaging (for example, to visualize babies in utero) may be able to treat Long COVID.

The purpose of this research study is to determine the best dose of FP-045 for Fanconi anemia pediatric and adolescent participants. The study will look at whether the participants have any side effects and if there are any possible changes in something called “biomarkers,” which are blood proteins that will be checked to see if they change when taking FP-045 and that may indicate if FP-045 can delay or prevent disease symptoms. Every participant will receive FP-045.

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.

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.

To compare the effects of treatment with enzalutamide (ENZ) versus darolutamide (DARO) on the cognitive function of men with non-metastatic and metastatic castration-resistant prostate cancer (mCRPC) by comparing the change in the maximally changed cognitive domain from baseline in patients in each study arm by 24 weeks.

There is an urgent public health need to reduce our reliance on opioids for effective long-term pain management, particularly in knee osteoarthritis (KOA). This effectiveness trial will compare recommended treatments to reduce pain and functional limitations in KOA and identify clinical and patient-level factors associated with treatment response. These results will lead to improved patient selection for treatment and inform evidence based guidelines by offering well-tested, effective, non-opioid alternatives.

This phase III trial investigates the best dose of vinblastine in combination with selumetinib and the benefit of adding vinblastine to selumetinib compared to selumetinib alone in treating children and young adults with low-grade glioma (a common type of brain cancer) that has come back after prior treatment (recurrent) or does not respond to therapy (progressive). Selumetinib is a drug that works by blocking a protein that lets tumor cells grow without stopping. Vinblastine blocks cell growth by stopping cell division and may kill cancer cells. Giving selumetinib in combination with vinblastine may work better than selumetinib alone in treating recurrent or progressive low-grade glioma.

Researchers hope that using Fisetin, an antioxidant, will reduce inflammation in the body caused by sepsis, which could help your body and organs have more time to recover. Placebo in this study will be something that looks similar to the study treatment, but does not contain any active drug. Everyone will receive 4 pills, but your group will determine how many of the pills are Fisetin. Researchers will look at the potential effects Fisetin or placebo has on inflammation in the body and how sick participants are at 7 days and 28 days.

This research study will develop an efficient electroencephalographic (EEG) method that uses narrated stories to identify frequency-specific hearing loss.

Idiopathic Pulmonary Fibrosis Prospective Outcomes (IPF-PRO) Registry is a prospective registry that will collect information regarding the natural history, health care interactions, participant reported questionnaire data to assess quality of life of IPF participants, and the methods of treatment of participants with a diagnosis of idiopathic pulmonary fibrosis (IPF) established at the enrolling centers. In addition, blood samples will be collected and banked for future research projects.

This study identifies the bio-behavioral predictors and correlation of responses to Comprehensive Behavioral Intervention to Tics (CBIT) in young people with tic disorder.

TrialNet is an international research group dedicated to the study, prevention, and early treatment of Type 1 diabetes. Type 1 diabetes is now understood as a disease that develops over time in stages. Stage 1 starts with the appearance of having two or more autoantibodies. This is followed by Stage 2, which is the development of abnormal blood glucose levels. Stage 3 is the clinical diagnosis of Type 1 diabetes. This study will help us learn more about how Type 1 diabetes occurs through the screening of diabetes-related autoantibodies and monitoring individuals who have tested positive for these autoantibodies.

Feasibility study to test our overall hypothesis that time restricted eating (TRE) presents a viable alternative to caloric restriction for improving glycemic measures and reducing weight in overweight/obese patients with metformin-only treated Type 2 diabetes (T2DM).