NF PROTOCOL 105, A Phase II Study of Cabozantinib (XL184) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type 1 in Children and Adults
Study ID: STU-2018-0014
neurofibromatosis type 1 (nF1), is a common human genetic disorder that affects approximately 100,000 people in the united States alone. individuals afflicted with nF1 develop a wide range of malignant and non-malignant manifestations. Plexiform neurofibromas are benign tumors of the nerve sheath that occur in approximately 40% of patients with nF1. They are a significant cause of morbidity in nF1 by causing pain, disfigurement or impairing function. Cabozantanib has emerged as a drug with significant impact on tumor volume, tumor apoptosis, and mast cell infiltration and as such has been selected for further clinical development.
Primary aim: To estimate the objective response rate (oRR) as defined by 20% volumetric MRi response of the target lesion to cabozantinib at 12 months in children and adults with nF1 plexiform neurofibromas by volumetric MRi imaging.