Neurofibromatosis

This phase 2 trial is testing whether the combination of avutometinib and defactinib can stop or shrink malignant peripheral nerve sheath tumors (MPNST) that have returned or cannot be removed with surgery. The study enrolls people over 12 years old with relapsed, refractory, met…

This trial tests several drugs in people with NF2-related schwannomatosis, a condition that causes tumors on nerves and in the brain. The study uses a flexible 'platform' design to evaluate treatments for four tumor types: vestibular schwannomas, non-vestibular schwannomas, menin…

This trial tests the drug selumetinib in people with NF2-related schwannomatosis, a genetic condition that causes non-cancerous tumors on nerves, often leading to hearing loss. The study includes both adults and children whose tumors cannot be surgically removed or are growing. R…

This trial tests whether combining two drugs—luvometinib (a targeted therapy) and serplulimab (an immunotherapy)—can shrink tumors and improve hearing in people with NF2-related schwannomatosis. Participants have progressive vestibular schwannomas or meningiomas that are not suit…

This early-phase trial tests a combination of three drugs—selumetinib, ZEN-3694, and durvalumab—in people with advanced sarcomas, including malignant peripheral nerve sheath tumors (MPNST) linked to NF1. The study first checks safety and dosing, then looks at whether the treatmen…

This phase 2 trial tests the drug mirdametinib, alone or with radiation, in people with NF1-related high-grade gliomas (aggressive brain tumors). It includes three groups: those with recurrent sporadic glioblastoma, newly diagnosed NF1-associated glioblastoma, and other NF1-relat…

This early-stage trial tests whether two cancer drugs, cabozantinib and selumetinib, can safely shrink plexiform neurofibromas in people aged 16 and older with neurofibromatosis type 1 (NF1). About 30 participants will receive the combination daily. The study first finds the safe…

This early-phase trial tests an oral drug called Trineumin (PRG-N-01) in 25 adults with neurofibromatosis type II (NF2), a genetic condition that causes non-cancerous tumors on nerves. The study has two parts: first finding a safe dose, then checking if that dose can shrink tumor…

This trial tests whether pain reprocessing therapy (PRT) — a brief psychological treatment that combines education, cognitive restructuring, and body-focused techniques — can reduce chronic pain. Adults with persistent pain lasting over three months are randomly assigned to full …

This study tests whether a new online program can help families understand their inherited cancer risk and encourage relatives to get low-cost genetic testing. Researchers will enroll 400 adults who carry a cancer-related gene change and their family members. The goal is to see i…

This study looks at how young children with neurofibromatosis type 1 (NF1) walk, to see if simple walking tests can help doctors spot and predict movement problems early. Researchers will measure walking speed and other skills in 56 children under 6 years old. The goal is to crea…

This study will collect blood and tumor tissue from up to 50,000 adults with hereditary or genetic-linked cancers. Researchers will use these samples to create lab-grown tumor models, like mini-tumors, to study how these cancers work and test new treatments. The goal is to better…