NEUROFIBROMATOSIS TYPE 1

This study tests a drug called selumetinib in children and young adults (ages 3-18) 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 d…

This study compares a targeted drug called selumetinib to standard chemotherapy (carboplatin/vincristine) in children aged 2 to 21 with NF1-related low-grade glioma. The goal is to see if selumetinib works as well or better at controlling the tumor and improving vision. About 165…

This study looks at how taking the drug selumetinib with a low-fat meal affects stomach side effects and drug levels in adolescents (ages 12 to under 18) with neurofibromatosis type 1 (NF1) who have inoperable plexiform neurofibromas. The goal is to find the best way to take the …

This study tests a drug called trametinib in children with a rare blood cancer (JMML) that has returned or not responded to treatment. The drug works by blocking certain enzymes that help cancer cells grow. The goal is to see if it can shrink or stop the cancer, and to check for …

This study tests a new granule (sprinkle) form of the drug selumetinib in 36 children aged 1 to under 7 years who have neurofibromatosis type 1 (NF1) with painful, inoperable tumors called plexiform neurofibromas. The goal is to find the right dose, check safety, and see if the d…

This study looks for substances in the blood that could signal the presence of plexiform neurofibroma tumors in people with Neurofibromatosis type 1 (NF1). Researchers will collect blood samples and use whole-body MRI scans from 200 participants to see if certain biomarkers match…

This study follows 259 children and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years. Researchers track tumors, quality of life, and other health changes to better understand how the disease progresses. The goal is to gather information that could help design better …

This study follows 500 people with neurofibromatosis type 1 (NF1) to see how their skin tumors (cutaneous neurofibromas) grow and change over five years. Researchers use 3D cameras to track tumors and study genetic links. The goal is to better understand these tumors to help doct…

This study monitors the safety of selumetinib in children aged 3 to 18 with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable tumors. Researchers will track heart function, bone growth, muscle enzymes, liver health, eye problems, and puberty development over time. T…