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.

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.

There is evidence that dysfunction of the diaphragm following COVID infection that leads to the shortness of breath and chest tightness. The diaphragm is the main muscle of respiration. This study involves muscle stretching of the diaphragm and associated muscles to improve the quality of respiration. Half of the participants will receive PT twice a week, for 12 weeks, for 1 hour (in person) and a half hour via telehealth. A control group will undergo traditional treatment as recommended by your provider.

We are studying the use of Efzofitimod given IV at two different doses to treat people who have pulmonary sarcoidosis. Participants must be on stable treatment with an oral corticosteroid with or without immunosuppressant therapy. Some people will receive IV saline (placebo) and we will compare groups to see how well the drug works and what side effects occur. The trial will last for about one year.

The goal of this current study is to test whether men with prostate cancer that is getting worse after treatment with hormone therapy and abiraterone respond better to alternating treatment with testosterone and enzalutamide vs. enzalutamide alone. We are testing to see which is better at stopping tumor growth that can be seen on a bone scan or CT scan and the effect of each regimen on lowering Prostate Specific Antigen (PSA values). Participants will be in the study for 6 to 24 months.

Through this research, the study staff hopes to better understand how DBS works and to define the optimal site in the brain for DBS treatment for Parkinson’s Disease. You will be asked to come for one study visit where you will perform some physical and mental tasks while on and temporarily off your medications and DBS treatment. Participation in this research study will take 7-8 hours.

The purpose of this study is to describe current knowledge and opinions about anal cancer screening among men who have sex with men (MSM), as well as their experience receiving guideline-compliant care aimed at anal cancer risk reduction using a large-scale survey disseminated via social media.

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.

The primary objective of this study is to estimate the overall survival (OS) of PIRD patients from the time of diagnosis. Patients may be treated with Best Available Therapy (BAT) or Hematopoietic Cell Transplant (HCT).

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.

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 study is looking at the liver function of people who have bariatric surgery. People who want to be in this trial must be obese, have abnormal liver function tests & prediabetes or type 2 diabetes. The study includes assistance with diet and exercise for a year after surgery.

This is an open-label, multi-center, single arm, Phase 1/2 study to evaluate the safety, PK, PD, and efficacy of quizartinib administered in combination with fludarabine and cytarabine (FLA) (Re-Induction Cycles 1 and 2) chemotherapy for re-induction, with optional consolidation chemotherapy, and as a single agent continuation therapy (after optional, but strongly encouraged, HSCT per standard of care), in pediatric relapsed/refractory AML subjects aged ≥1 month old to <18 years old (and young adults up to 21 years old) with FLT3-ITD mutations.

This is a global, multicenter, prospective, observational, longitudinal registry conducted to characterize the natural history and real-world clinical management of patients diagnosed with AHP. This protocol will not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of registry participation.

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 goals of the current proposal are to systematically approach the development and iterative testing of a novel, culturally-responsive, evidence-informed psychotherapeutic activities (MyGender Dolls) for transgender and gender diverse (TGD) children (ages 5-10) and their caregivers. There are two primary objectives: 1) to develop and refine the MyGender Dolls psychotherapeutic activities based on feedback from community stakeholders, and 2) to assess the initial feasibility, acceptability, and usability of the MyGender Dolls with TGD children and their caregivers.

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.

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.

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.

The study drug FP-001 (Leuprolide mesylate) is being developed for children that are suffering from central (gonadotropin-dependent) precocious puberty (CPP). Leuprolide has been approved in the United States (US) and the European Union (EU) as treatment for prostate cancer already, and other forms of Leuprolide from other companies have been approved for the treatment of CPP. In this clinical study, Leuprolide will be used in the form of a 6-month depot injection.

We are studying how well combination chemotherapy, bevacizumab, and/or atezolizumab work in treating people with deficient deoxyribonucleic acid (DNA) mismatch repair colorectal cancer that has spread from where it first started (primary site) to other places in the body (metastatic). Chemotherapy drugs, such as fluorouracil, oxaliplatin, and leucovorin calcium, 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. Bevacizumab may stop or slow colorectal cancer by blocking the growth of new blood vessels necessary for tumor growth. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving combination chemotherapy, bevacizumab, and atezolizumab may work better in treating patients with colorectal 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®.

This study will investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutations. This study is a parallel-group, two-staged, Phase 2/3, randomized, multi-center study with two cohorts: Cohort A followed by Cohort B. The purpose of Cohort A is to provide early data on efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas, and provide guidance for the correct dose, population, sample size, and endpoint for the confirmatory part of the study (Cohort B). Additional goals for Cohort A are to assess effects of food on drug absorption. The purpose of Cohort B of the study is to assess the efficacy and safety of REC-2282 compared with placebo in participants with progressive NF2 mutated meningiomas.

The main purpose of the study is to see whether farudodstat, taken orally for 12 weeks, can help people with alopecia areata and to find out if farudodstat is safe and tolerable when compared to placebo. The placebo is a pill that looks like farudodstat tablet but has no drug or other active ingredient in it.

The purpose of this research is to understand the effort needed to understand speech if hearing is impaired. Listening effort is higher in people with hearing impairment and has wide-ranging negative consequences across many aspects of a person’s life. The goal of this project is to explore factors that make listening effortful, with special focus on the need to repair perceptual mistakes by relying on context.

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.

This protocol will characterize the effects of deep brain stimulation (DBS) location (both adverse and beneficial) on motor signs in people with Parkinson’s disease (PD). This information can be used to inform future DBS protocols to tailor stimulation to the specific needs of a patient. If targeted dorsal GP stimulation is shown to significantly improve motor features that are typically resistant to dopamine replacement therapy, these experiments will likely have major impact on clinical practice by providing a potential strategy to treat medically intractable symptoms.

The study's purpose is to see if the drug abemaciclib is safe and effective in combination with temozolomide and irinotecan (Part A) and abemaciclib in combination with temozolomide (Part B) in pediatric and young adult participants with relapsed/refractory solid tumor and abemaciclib in Combination with Dinutuximab, GM-CSF, Irinotecan, and Temozolomide in Pediatric and Young Adult Patients with Relapsed/Refractory Neuroblastoma (Part C).

This study proposes to collect lung fluid to identify potential biomarkers associated with pulmonary sarcoidosis.

This study seeks to understand visual perception in people with Visual Snow Syndrome and how this relates to brain function.