NEUROFIBROMATOSIS 1

This study is testing a drug called abemaciclib for people with Neurofibromatosis type 1 (NF1) who have nerve tumors called atypical neurofibromas. The main goals are to find a safe dose of the drug and to see if it can shrink these tumors, which can sometimes turn into cancer. P…

This study is observing patients in Korea who are already taking Koselugo for neurofibromatosis type 1 (NF1) and its associated nerve tumors. Its main purpose is to monitor the drug's safety and see how well it works in everyday medical practice, outside of a controlled clinical …

This study aims to see if a 'liquid biopsy' blood test can find a dangerous cancer called MPNST earlier in adults with Neurofibromatosis Type 1 (NF1). Researchers will compare the blood test results to the current standard methods of doctor exams and imaging scans over five years…

This study is testing if starting a drug called selumetinib early can prevent dangerous tumors from growing and causing serious problems in young children with neurofibromatosis type 1 (NF1). It will enroll 200 children aged 1 to 8 years who have these tumors in high-risk areas b…

This study is testing if a type of therapy called Acceptance and Commitment Therapy (ACT) can help parents and guardians better manage the stress of caring for a child with a rare genetic condition known as a RASopathy. The 8-week program is done completely from home using a smar…

This study is comparing two different educational programs to see which one better helps people with Neurofibromatosis 1 (NF1) get the recommended health screenings during their regular doctor visits. The research is conducted entirely online with adults who have NF1 or parents o…

This study is testing two different online approaches to help adults with Neurofibromatosis 1 (NF1) better understand their condition and recommended care. Researchers will provide personalized care letters and then randomly assign participants to either watch educational videos …

This study aims to create and test better questionnaires for people with neurofibromatosis 1 (NF1) who have painful plexiform tumors. Researchers are working with patients and parents to improve how pain and its impact on daily life are measured. The goal is to develop reliable t…

This study aims to learn how to better predict which benign nerve tumors in people with Neurofibromatosis type 1 (NF1) might become cancerous. Researchers will observe 225 participants, aged 3 and up, for 10 years, using scans and check-ups to track their tumors. Participants wil…

This study aims to better understand a group of rare genetic conditions called RASopathies, which include Noonan Syndrome and Neurofibromatosis. Researchers will collect blood, tissue, and health information from up to 1,000 participants who have or are suspected of having one of…