The Muscular Dystrophy Association (MDA) wants to collect information about individuals with neuromuscular disease to better understand the disease progression and ultimately improve the medical care, quality of life, and survival of those with neuromuscular disease. To collect this information, MDA has created a data registry called the Neuromuscular Observational Research Data Hub (referred to as the “MOVR Data Hub”).

The purpose of the study is to test new biomarkers of amyotrophic lateral sclerosis (ALS) and primary lateral sclerosis (PLS) using MRI scans at 3 tesla (3T). Identifying biomarkers of a disease can lead to a better understanding of the disease as well as improved treatments.

This research is being completed to examine the cells, brain imaging, and speech in individuals with REM Sleep Behavior Disorder who are taking serotonergic medications such as Citalopram, Escitalopram, Fluoxetine, Fluvoxamine, Paroxetine and Sertraline. The purpose of examining these is to try and see if we can predict signs of Dementia with Lewy bodies (a progressive form of dementia with an increase in decline of thinking, reasoning, and other functions). This may benefit others by enabling us to diagnose Dementia with Lewy Bodies sooner rather than later.

We are studying a new drug, INCB054707, used to treat people who have hidradenitis suppurativa which is a chronic skin condition characterized by lumps or boils in places such as the armpits or groin. The skin lesions develop because of inflammation of the follicle. We are studying two doses of the drug and we will compare the effectiveness and side effects that occur. We will also have a group that receives an inactive medication (placebo). After the first 12 weeks of taking the drug or placebo, all participants will receive the active drug. The study will last for about 62 weeks.

This study will be measuring brain activity using EEG and assessing motor skills and speech in children following cancerous brain tumor resection. No direct cancer treatments or objectives are being targeted.

This is a Phase 1/2 multicenter, open-label study to evaluate palbociclib in combination with either irinotecan (IRN) and temozolomide (TMZ) or topotecan (TOPO) and cyclophosphamide (CTX) chemotherapy in children, adolescents and young adults with recurrent or refractory solid tumors. The study consists of a non- randomized Phase 1 portion for recurrent or refractory solid tumors followed by potential non- randomized tumor specific cohort(s) and a randomized, Phase 2 portion for recurrent or refractory EWS.

This study is being done to see how safe an investigational drug is and how well it will work to help people with obesity, or overweight with weight-related conditions like hypertension, dyslipidemia, obstructive sleep apnea, non-alcoholic fatty liver disease, or diabetes. If you qualify to be in the study, you will be given frequent lifestyle and behavioral counseling for the first 12 weeks of the study. The counseling will consist of advice on physical activities and dietary advice on healthy eating. During the treatment period, you will receive either tirzepatide or placebo. Placebo is a solution that looks like the study drug but has no medicine. The chance that you will get the study drug is 2 in 3. This phase will last about 72 weeks.

This study will provide the most accurate and reliable estimates to date on disease progression and clinical events in evolving chronic pancreatitis. We also hope to develop from the results of this study some lab tests that will help us with early diagnosis of chronic pancreatitis and also to discover any genetic factors that may affect your chances of developing chronic pancreatitis.

Levosimendan has not been approved by the FDA to treat people who have PH-HFpEF or approved to be taken by mouth (orally). In this study, we will measure the amount of levosimendan in blood at various times and evaluate the change in participants 6-Minute Walk Distance.

This is a study of highly effective CFTR modulators and their impact in children with CF on endocrine growth factors, the gut microbiome, respiratory microbiome, liver and pancreatic function, lung function, sweat chloride, and inflammatory markers.

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.

Currently, more than half of all women who are able to quit smoking cigarettes during pregnancy start smoking again within six months after they give birth. We want to find out if the drug bupropion (a commercially-available medicine) can help women who quit smoking during pregnancy to continue not smoking after they give birth. All study visits can be completed either in-person or virtually.

The purpose of this study is to test whether the investigational drug, tegoprubart, in combination with the same standard immunosuppressive medicines (anti-thymocyte globulin, corticosteroids, and mycophenolate) is safe, tolerable and effective compared to tacrolimus. The study will specifically look at the function of the implanted kidney in the tegoprubart group compared to the tacrolimus group and will also assess how well tegoprubart prevents diabetes and prevents rejection.

In this study we will be looking at the safety and effectiveness of the medication GLP-1 (Semaglutide) in patients who are overweight and have been diagnosed with Cystic Fibrosis Related Diabetes (CFRD).

This study aims to test the use of electrophysiology and TMS (transcranial magnetic stimulation) in discovering biomarkers for cognitive impairment in chronic pain and depression. Total time anticipated for the study is around 15 hours.

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.

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.

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.

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.

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.

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.