Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults
Status:
Active, not recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in
Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with
Neurofibromatosis Type 1 (NF1) and have a type of tumor called a plexiform neurofibroma (PN).
Neurofibromas are tumors that develop from the cells and tissues that cover the nerves.
Plexiform neurofibromas can be disfiguring, painful, and life-threatening. These types of
tumors typically do not respond well to most treatment approaches such as chemotherapy,
radiation, and surgery because of their slow growth and location near vital structures of the
body such as nerves, blood vessels, and the airway.
The primary objective is to determine the response rate of NFI patients with plexiform
neurofibromas treated with Cabozantinib therapy using MRI scans. The objective response rate
to cabozantinib is defined as ≥ 20% reduction in tumor volume at the end of 12 cycles.