To evaluate if daily oral administration of elafibranor 120 mg improves the time of event free survival compared to placebo in adults with Primary Sclerosing Cholangitis (PSC).
This study is being offered for patients with metastatic pancreatic adenocarcinoma who have tried available approved treatments and do not have satisfactory treatment options or are unable to participate in a clinical trial. The purpose of this Expanded Access Program (EAP) is to provide access to daraxonrasib, an investigational drug that has not yet been approved by the FDA.
Daraxonrasib is an oral tablet. Clinic visit are every 28 days to receive a new supply of daraxonrasib. Participation is expected to last until whichever of the following occurs first - 1: you are no longer benefiting from daraxonrasib; 2: daraxonrasib becomes commercially available (FDA approved); 3: Revolution Medicines, the Sponsor of the EAP, the IRB, or the FDA decides to stop the EAP.
Risks include skin rash, diarrhea, nausea and vomiting, mouth sores, and swelling. This EAP is not a research study. Its main goal is to provide access to daraxonrasib to patients who may benefit based on what information is known about the drug so far. There is no guarantee that your cancer will get better as a result of receiving daraxonrasib. It is possible that you will show no improvement or that your cancer could get worse.
This is a 3 month study for pediatric patients between the ages of 6 months and 18 years that have a current diagnosis of heart failure. This study will compare the use of the medication finerenone to placebo. During the study, participants will continue to receive their standard of care therapy according to pediatric heart failure guidelines. At the end of the 3 month study period, participants will have the option to enroll in a 9 month open label extension.
This study is for patients that have been diagnosed with advanced solid tumors. This study is testing an investigational drug called At0365-P1-01. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to evaluate how safe the drug is, how well participants can handle the drug, how it moves through the body, how it affects the body, and if the drug helps treat the cancer of AT03-65. AT03-65 is administered via intravenous (IV) infusion. Participants can continue to receive this study drug until it no longer gives them benefit. The estimated study duration is approximately 30-35 months for subjects screening, treatment and follow-up.
This is a study to test the safety of ALN-6400 which is considered to be "an investigational drug", meaning it is not currently approved by regulatory authorities, including the United States Food and Drug Administration (FDA), for the treatment of any disease and find out what effects, if any, it has on people with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB). ALN-6400 works by lowering protein in the blood breakdown blood clots and reduce bleeding in women ages 16-45. This study is a 24 week study with an optional extension period of 84 weeks.
Iron deficiency is the most common nutritional deficiency worldwide and is a leading cause of anemia, which can result in symptoms such as fatigue, weakness, shortness of breath, and reduced quality of life. This study aims to better understand why some people develop iron deficiency by examining how the body absorbs iron and how this may relate to diet and proteins involved in iron absorption. Participants will complete questionnaires, provide blood, urine, and stool samples, undergo an oral iron absorption test, and complete a short food diary. If an upper endoscopy is already being performed as part of routine medical care, one additional biopsy sample will be collected during the procedure for research purposes. The information gathered may help improve the diagnosis and evaluation of iron deficiency anemia in the future. Participation involves two study visits over approximately one month. There is no direct medical benefit to participants, but the knowledge gained may benefit future patients with iron deficiency anemia.
Select Late will be looking at treatment outcomes in patients presenting with severe blood clot blocking a blood vessel in your brain (ischemic stroke) specifically in one of the large arteries in the brain effecting the front of the brain presenting between 24 and 72 hours after stroke start. The study will be looking at patient outcomes when treated with the standard of care medical management compared to medical management plus endovascular thrombectomy (a surgical procedure to remove a blood clot).
This study will enroll patients with different types of cancer, as well as healthy patients with no history of cancer, into four cohorts to collect urine and/or blood samples only. The study will collect biospecimen samples to support further development of a urine-based ctDNA test. The study will enroll a total of 920 patients over 4 years.
This study is looking for patients who have been diagnosed with advanced non–small cell lung cancer (NSCLC) that have certain biomarkers (negative for PD-L1, and positive for KRAS p.G12C). This study is being conducted to determine how well participants respond to treatment with chemotherapy (carboplatin and pemetrexed) combined with either sotorasib or pembrolizumab. The study will determine whether sotorasib and chemotherapy reduces the risk of this type of cancer getting worse and improves participants survival (i.e., increases the chance of you living longer) compared to the current standard treatment with pembrolizumab and chemotherapy. It is anticipated that participants will be enrolled in this study for approximately 5 and a half years. There will be a total of approximately 15 visits to the study site during the study depending on how well participants tolerate or respond to the study drug. The visits will take several hours each and will take place approximately every 3 weeks.
We will gather feedback from healthcare providers and pregnant or up to two years postpartum adult women to better understand their needs and experiences related to traumatic stress and alcohol use during pregnancy. We will conduct focus groups with maternal health and mental health professionals, as well as conduct 60-minute interviews along with a survey/questionnaire with pregnant women who are experiencing both alcohol-related challenges and trauma-related symptoms. What we learn from these conversations will help us tailor services to address the unique needs of women during pregnancy and the postpartum period.