Active clinical trials for "Neurofibroma, Plexiform"

This prospective pilot study is designed to provide preliminary data on the use of Fluorodeoxyglucose Positron Emission Tomography-Magnetic Resonance Imaging (FDG-PET-MRI) in patients with neurofibromatosis-1 (NF1) associated optic glioma and plexiform neurofibroma (PN). Subjects will undergo FDG-PET-MRI scans in place of standard of care imaging at 0 and 12 months, unless more frequent imaging is clinically indicated. Subjects and their family caregivers will also undergo serial interviews and complete questionnaires related to the psychosocial aspects of NF1.

This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit. Approximately 100 patients in the US will be treated as part of this protocol

This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee and Single Patient IND approval.

The purpose of the research study is intended to use specimens (such as tissue) and medical information in the Laboratory of Musculoskeletal Oncology at the Van Andel Research Institute for laboratory research in Grand Rapids, Michigan. Small tissue samples of cutaneous neurofibromas will be collected as part of this research. The samples will help researchers learn more about cutaneous neurofibroma and help them better understand NF1. There are many different types of studies, both now and in the future, that can be done using the specimens they receive. These include using the specimens and information to look for new ways to diagnose and treat Neurofibromatosis Type 1 (NF1). The specimens may be used to study how genes affect health and disease, or how genes affect the way a disease or condition responds to treatment. Some of these studies may lead to new products, such as treatments or tests for diseases. Through this study, we hope to find better ways to understand and treat NF1 in the future.