NEUROFIBROMATOSIS TYPE 1

This study tracks the safety of selumetinib, a drug for children with neurofibromatosis type 1 (NF1) who have painful, inoperable nerve tumors. About 124 children aged 3 to 18 across Europe and Israel will be monitored for side effects like heart changes, bone growth issues, musc…

This study tests whether the targeted drug selumetinib works as well as standard chemotherapy (carboplatin/vincristine) for children and young adults (ages 2-21) with NF1-related low-grade glioma. The goal is to see if selumetinib can stop tumor growth and improve vision better t…

This study tests an experimental drug, selumetinib, in children and young adults with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink the tumors or stop them from growing. Participants take the drug…

This study tests a new granule (sprinkle) form of the drug selumetinib in children aged 1 to 6 years who have neurofibromatosis type 1 (NF1) and painful, inoperable tumors called plexiform neurofibromas. The goal is to find the right dose, check safety, and see if the medicine ca…

This study looks at whether taking the drug selumetinib with a low-fat meal reduces stomach side effects in teenagers with neurofibromatosis type 1 (NF1) who have tumors that cannot be removed by surgery. About 24 teens will take the drug under both fed and fasted conditions to c…

This study tests a drug called trametinib in children and young adults with a rare blood cancer (juvenile myelomonocytic leukemia) that has returned or not responded to standard treatment. The drug works by blocking certain enzymes that help cancer cells grow. The goal is to see …

This study looks for signals in the blood that could show whether a person with Neurofibromatosis type 1 (NF1) has plexiform neurofibroma tumors. Currently, finding these tumors requires whole-body MRI scans. Researchers will collect blood samples and compare them with MRI result…

This study follows 500 people with neurofibromatosis type 1 (NF1) to track how their skin tumors (cutaneous neurofibromas) grow and change over five years. Researchers use a special 3D camera to measure tumors without touching the skin. The goal is to better understand these tumo…

This study follows 259 children and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years to learn how the disease changes over time. Researchers use imaging, blood tests, and quality-of-life surveys to track tumors and other symptoms. The goal is to better understand NF1…