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A Phase 11 Study of Cabozantinib (XL184) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type 1 age 16 years or greater


The Cabo Study is designed to evaluate whether Cabozantinib (XL184) will shrink target lesion as noted by radiographic response. The trial will also evaluate the effects, good and/or bad, of XL184.)


Cabozantinib (XL184) is a tyrosine kinase inhibitor and has been approved by FDA for use in medullary thyroid cancer. Cabozantinib has been selected for further human testing for NF1 plexiform neurofibromas because of lab tests that show decrease in tumor size.


Patients 16 years or greater with NF1 and growing, symptomatic or inoperative plexiform neurofibromas are eligible. Plexiform neurofibromas must be analyzed by volumetric MRI at screening and throughout the study. A full list of criteria can be obtained by contacting one of the study sites.


Subject will receive Xl184 by mouth once daily on a continuous basis for up to 24 courses. Treatment will be assessed every 4 courses for the first year and then every 6 courses. Treatment will continue beyond 3 courses if MRI shows at least 15% reduction in volume of the target lesion at the end of course 8. At least 20% shrinkage must be noted on MRI for treatment beyond course 12.


  • Chie-Schin Shih, MD, Chair
  • Michael Fisher, MD, Co-Chair
  • Jaishri Blakeley, MD, Co-Chair


Enrollment is complete. The study is in follow-up and data analysis currently.


None at this time.