NEUROFIBROMATOSIS TYPE 1

This study follows children who previously took dabrafenib and/or trametinib for certain brain tumors. It aims to see how safe these drugs are over the long term by tracking side effects and monitoring growth. About 163 children are enrolled, and the study is no longer recruiting…

This study tests an experimental drug called HLX-1502 in people aged 12 and older with neurofibromatosis type 1 (NF1) who have growing or symptomatic plexiform neurofibromas (nerve tumors). The main goal is to see if the drug can shrink these tumors by at least 20%. Researchers w…

This study tests whether combining whole-body MRI with artificial intelligence can reliably detect early signs of cancer in 15 children (ages 5-17) with neurofibromatosis type 1 (NF1). Participants will have three MRI scans over a year, and the AI will compare images to see if it…

This study is testing two questionnaires to see if they can capture how people with neurofibromatosis type 1 (NF1) feel about the appearance of their tumors. About 110 participants — including children, adults, and caregivers — will join focus groups or interviews to share their …

This research study aims to discover new genes that may cause cancer to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had childhood cancers. Participants will not receive direct treatment o…

This study is a disease registry that will collect information from 200 people in Egypt with neurofibromatosis type 1 (NF1), a genetic condition that causes tumors on nerves. The goal is to understand how common NF1 is, how it progresses, and how it is managed in Egypt. No new tr…

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 is testing whether a device that measures pupil changes can help assess pain and other unusual sensations in people with neurofibromatosis type 1 (NF1). Researchers will place a small electrode on a finger or toe to send mild, painless signals while a camera records th…

This study aims to understand how 9 rare skin diseases affect people's lives beyond just physical symptoms. Researchers will survey 900 patients and their families to measure the emotional, social, and financial burden. The goal is to create a tool that helps doctors and caregive…