NEUROFIBROMATOSIS 1

This study tests a new treatment plan for children with juvenile myelomonocytic leukemia (JMML), a rare blood cancer. Lower-risk patients receive two drugs (trametinib and azacitidine), while higher-risk patients get those plus chemotherapy. The goal is to see if the combinations…

This study tests a new way to prepare donor stem cells for transplant in people with blood cancers like leukemia. By removing certain immune cells from the donated cells, researchers hope to lower the risk of graft-versus-host disease, a serious side effect. The study involves 70…

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 and see if the drug can shrink or stabilize these tumors. Participants take the drug twice daily for…

This study tests whether giving selumetinib early to children aged 1 to 8 with NF1 can prevent plexiform neurofibromas from growing and causing serious issues like pain or blindness. Half the children will receive the drug, while the other half will be observed. The goal is to se…

This study looks at whether Acceptance and Commitment Therapy (ACT) can help caregivers of children with RASopathies—a group of genetic conditions—manage parenting stress. About 70 adult caregivers will take part fully online, watching short videos and having coaching sessions ov…

This study looks at whether an online platform can help adults with neurofibromatosis 1 (NF1) who have low health literacy better understand their symptoms and care. Participants get personalized care letters and either educational videos or a call with a peer navigator. The goal…

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 a higher risk of cancer. Researchers will collect blood, tissue, and medical information from up to 1,000 participant…

This study tests two different educational programs to see which one helps adults with Neurofibromatosis 1 (NF1) and parents of children with NF1 get the recommended health screenings during their regular check-ups. Participants live in the U.S., do not visit a specialized NF cli…

This study looks at whether a blood test (liquid biopsy) can find a rare cancer called MPNST earlier than current methods in adults with Neurofibromatosis Type 1 (NF1). About 1,000 participants will give blood samples and complete surveys every six months for five years. The goal…

This study is working to improve questionnaires that measure pain, daily living, and physical function for people with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas. Researchers will talk with patients and parents to refine existing tools, then test the updated …

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…