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We are doing this study to find out if an investigational drug called HM43239 (the study drug) is a safe and effective option for people who have AML that came back after remission or did not respond to standard treatments. We also want to find out what dose of the study drug works best for most patients.
We are doing this study to learn more about children with atrioventricular septal defect. We hope to improve care for patients with this condition in the future.
Before joining the main part of the study, you will have some tests to see if you qualify. These tests include check-ups for your body, skin, eyes, and nerves, as well as heart tests, an MRI scan, and bloodwork. If you qualify, you will have more tests every few cycles. A cycle is 28 days. These tests include check-ups, eye exams, blood tests, a stool sample, and MRI scans. After the treatment part of the study, you will be in a follow-up study called PNOC COMP. This part will look at how your thinking, daily life, and feelings change over time. You will get a different form to learn more about that part of the study.
In this study, you will be placed by chance into one of three groups. One group will get a medicine called daraxonrasib. Another group will get daraxonrasib plus two other medicines called gemcitabine and nab paclitaxel. The third group will get gemcitabine and nab paclitaxel without daraxonrasib. You will keep taking the study medicines as long as they are safe for you. The study is planned to last three years. Within thirty days after your last dose, you will have a follow up visit. After that, the study team will contact you every three months to check on your health until the study ends.
We are doing this study to find out if breathing exercises can improve lung function for kids who have asthma.
Participants will receive the study drug ASP2020 in one eye during an outpatient surgery and will not need to stay in the hospital. Participation will last about 13 to 15 months and includes 12 study visits for eye exams, tests, and other study procedures.
This study is a research study that watches how a vision problem changes over time. The condition is called Stargardt disease or Stargardt type macular dystrophy, which affects how well a person can see. Researchers will follow people with this condition and collect information to learn how vision changes. The goal is to better understand the disease so doctors can improve care and treatment in the future.