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The purpose of this study is to find out more about an investigational drug called mRNA-3927 (the study drug). An "investigational drug" is a drug that is being tested and is not approved by the U.S. Food and Drug Administration (FDA). We want to see how safe the study drug is and if it works for participants with propionic acidemia (PA).
Food allergies are a major public health issue affecting an estimated 8% of U.S. children, and possibly up to 10% of adults. Thirty-two (32) million patients have food allergies in the US alone and no cure currently exists. This study is a biorepository (large collection) of samples from patients with a physician-diagnosed and/or an oral food challenge-confirmed food allergy. The repository is potentially a rich resource for advancing the field of food allergy. This study aims to collect blood from patients who have had a positive oral food challenge or have been diagnosed as having a food allergy based on a positive skin prick test or elevated levels of allergen specific-IgE antibodies and a convincing history. The biorepository will also include blood from individuals with no history of food allergies.
The purpose of this study is to evaluate how safe and effective the drug Qsymia (Phentermine/Topiramate of Ph/T) is for children, adolescents, and young adults with hypothalamic obesity. We will also evaluate if Ph/T helps with weight loss and changes in hunger in patients with hypothalamic obesity.
If you are determined to be eligible, then your participation will last for approximately 8 months. Participants will be asked to complete up to 5 in-person visits and 5 telephone safety check-ins. During the study you will be randomly assigned to study drug (phentermine/topiramate) or placebo for 28 weeks.
You will complete study procedures including blood tests, ECGs, mobile cardiac outpatient telemetry, body composition scan, and questionnaires. Compensation and reimbursement of limited travel expenses is available.
This study is enrolling people who have developed lung problems (called Bronchiolitis Obliterans or BO) after hematopoietic stem cell transplant (HSCT). BO is a type of lung injury after HSCT due to graft versus host disease. While BO is uncommon after HSCT, when BO occurs it is often diagnosed late in patients, when lung injury is hard to treat and can be irreversible, leading to long-term lung disease or even death.
The purpose of this study is to find out more about a drug called ruxolitinib (the "study drug") as an early treatment for lung injury and BO after HSCT.
This extension study will determine the long-term safety and effectiveness of mRNA-3705, a treatment for Methylmalonic acidemia (MMA), in patients who previously enrolled in and completed a prior mRNA-3705 study.
The purpose of this study is to determine how what you eat and drink (your diet), antibiotics you take, and the bacteria that live in your gut (microbiome) contribute to developing kidney stones. Kidney stone disease, known as nephrolithiasis, is also influenced by products of metabolism (metabolites) that are found in your urine. These factors will be examined in participants at least 4 years of age. Each participant will complete three 24-hour dietary recalls and will provide one stool sample and up to two urine samples. The information from this study will help doctors find new metabolic pathways that can be used for treatment of kidney stones.
This study involves taking a study drug called asciminib. The overall goal of the study is to find out how safe and effective the study drug is in treating people that have been diagnosed with Chronic Myeloid Leukemia (CML) and have not responded to treatment.
The DISCOVERY Study is a new, national effort to learn about the causes of type 2 diabetes in children. DISCOVERY is for children 9-14 years old who are at higher risk of developing type 2 diabetes. We want to figure out why some children get type 2 diabetes and others do not.
The goals of this research study are to find out how having COVID-19 recently affects blood sugar, inflammation, and insulin in people with type 1 and type 2 diabetes. We also want to see if COVID-19 changes blood vessel health or makes blood clot more easily. We will also look at how things like genes, lifestyle, and environment might affect these issues.
This study will evaluate the safety and tolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with a neurocognitive deficit that are at least 4 months old. This study involves administration of the RGX-111, general anesthesia, lumbar puncture, blood draws, MRI, ultrasound, electrocardiogram, echocardiogram, hearing test, and chart review.