NEUROFIBROMATOSIS TYPE 1

This study is testing a new 'sprinkle' granule form of a drug called selumetinib for very young children (ages 1 to 7) with neurofibromatosis type 1 (NF1) who have painful or problematic tumors that cannot be removed by surgery. The main goals are to find the right dose for this …

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 tested an oral drug called selumetinib for children and young adults with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The main goals were to find a safe dose and see if the drug could shrink or stop these tumors from growing.…

This study is testing whether taking the approved drug selumetinib with a low-fat meal, instead of on an empty stomach, can reduce stomach-related side effects like nausea and diarrhea in teenagers with neurofibromatosis type 1 (NF1) and inoperable tumors. Researchers will measur…

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 is for children and young adults (ages 2-21) with a genetic condition called Neurofibromatosis Type 1 (NF1) who have a low-grade brain tumor. It compares a newer oral medication called selumetinib against the standard intravenous chemotherapy (carboplatin/vincristine).…

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…