NEUROFIBROMATOSIS TYPE 1

This study is testing a new, easier-to-swallow granule form of the drug selumetinib in very young children (ages 1-7) with neurofibromatosis type 1 (NF1) who have inoperable, painful nerve tumors. The main goals are to find the right dose for this age group and to check the drug'…

This study is testing whether a pill called selumetinib works as well as or better than standard intravenous chemotherapy (carboplatin/vincristine) for children and young adults with low-grade brain tumors linked to Neurofibromatosis Type 1 (NF1). The main goals are to see which …

This study tested an oral drug called selumetinib in children and young adults with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas—nerve tumors that cannot be safely removed by surgery. The goal was to see if the drug could shrink these tumors or stop them from g…

This study is testing whether taking the drug selumetinib with a low-fat meal is easier on the stomach than taking it on an empty stomach for teenagers with neurofibromatosis type 1 (NF1) and inoperable tumors. Researchers want to see if eating first reduces common side effects l…

This study follows children and teens who are already taking the medications dabrafenib and/or trametinib for various types of brain tumors. The main goal is to monitor their long-term health, safety, and development while they continue treatment. Researchers will track side effe…

This study is testing a drug called trametinib in children whose juvenile myelomonocytic leukemia (JMML) has come back or hasn't responded to other treatments. The goal is to see if the drug can control the cancer by blocking signals that help the cancer cells grow. Researchers w…

This study is monitoring the long-term safety of selumetinib, a medication already approved for children with neurofibromatosis type 1 who have inoperable tumors. Researchers will follow 124 children aged 3-18 years across Europe and Israel for up to 6 years to track potential si…

This study aims to understand how Neurofibromatosis Type 1 (NF1) changes over many years in children and adults. Researchers will follow about 260 participants for up to 10 years, performing regular check-ups, scans, and tests to track tumors, pain, and quality of life. The goal …

This study aims to understand how and why benign skin tumors grow in people with Neurofibromatosis Type 1 (NF1). Researchers at Johns Hopkins will follow 500 participants for five years, using annual 3D whole-body imaging to track changes in these tumors over time. The goal is to…

This study aims to find signals in the blood that can tell doctors about tumor growth in people with Neurofibromatosis type 1 (NF1). Researchers will study 200 people with NF1, using blood tests and full-body MRI scans to see if specific blood markers match the size and number of…