NEUROFIBROMATOSIS 1

This study tests a new way to prepare stem cells for transplant in children and adults with blood cancers like leukemia. The method removes certain immune cells to lower the risk of graft-versus-host disease, a serious side effect. About 70 participants will receive the treatment…

This study tests two drugs, trametinib and azacitidine, with or without chemotherapy, in children and young adults newly diagnosed with juvenile myelomonocytic leukemia (JMML), a rare blood cancer. Lower-risk patients get the two-drug combo; higher-risk patients also get two addi…

This study tests a drug called abemaciclib for people with NF1 who have atypical neurofibromas—nerve tumors that can cause pain and other problems. The goal is to find a safe dose that can shrink or control these tumors without surgery. About 55 participants aged 12 and older wil…

This study tests whether giving selumetinib early to children with NF1 (ages 1 to 8) can prevent nerve tumors from growing and causing serious problems like blindness or nerve damage. About 200 children with no current symptoms but tumors in risky locations will either receive se…

This study looks at whether Acceptance and Commitment Therapy (ACT) can help reduce stress for caregivers of children with a RASopathy (a group of genetic conditions). About 70 adult caregivers will take part fully online, using a phone app to watch videos and answer surveys. Hal…

This study compares two educational programs designed to help adults with neurofibromatosis 1 (NF1) and parents of children with NF1 get the recommended health screenings during their annual check-ups. Participants live across the U.S. and do not visit a specialized NF clinic. Th…

This study aims to improve questionnaires that measure pain, daily living, and physical function in people with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (pNFs). Participants aged 5 and older will provide feedback through group discussions or interviews to refine…

This study is testing whether a blood test (liquid biopsy) can detect a rare but serious cancer called MPNST earlier than current methods in adults with Neurofibromatosis Type 1 (NF1). Researchers will collect blood samples and surveys from 1,000 participants every six months for…

This study aims to learn more about a group of genetic conditions called RASopathies, which affect development and can cause heart defects, learning problems, and cancer risk. Researchers will collect blood, tissue, and medical information from up to 1,000 participants, including…

This study looks at people with neurofibromatosis type 1 (NF1), a genetic condition that often causes noncancerous nerve tumors. About half of these people may develop tumors that can become cancerous, but doctors don't know which ones will change. The study will follow 225 parti…

This study is observing 200 people in South Korea who are already prescribed Koselugo for neurofibromatosis type 1 (NF1) with plexiform neurofibromas. Researchers will track side effects and how well the drug works in everyday medical practice. The goal is to confirm the drug's s…

This study is testing two ways to help adults with neurofibromatosis 1 (NF1) who have low health literacy. Participants get personalized care letters and either educational videos or a call with a peer navigator. The goal is to see if these tools are helpful and easy to use. Abou…