A Phase 2 Trial of the MEK Inhibitor PD-0325901 in Adolescents and Adults with NF1- Associated Morbid Plexiform Neurofibromas
Up to 50% of patients with neurofibromatosis type 1 have plexiform neurofibromas: tumors of the nerve, muscle and soft tissue that are slowly progressive and can cause neurologic difficulty and possibly, progress to a type of cancer called sarcoma. Drugs that target the Ras pathway such as MEK inhibitors may stop the growth of these tumors. PD-0325901 has been shown to work in stopping these tumors in mice with neurofibromas. This study will test whether this drug works similarly in patients with neurofibromas.
-patients with Neurofibromatosis Type 1 with plexiform neurofibroma(s)that are progressive or are causing significant morbidity-plexiform neurofibromas amenable to volumetric MRI analysis-prior surgery is allowed if incompletely resected and evaluable by volumetric analysis-prior therapies allowed if appropriate wash-out periods are met
The MEK inhibitor PD-0325901 will be taken orally twice daily on a 3 week on/1 week off schedule. Each course will we 4 weeks in total. Radiographic response of the plexiform neurofibroma(S), as measured by MRI volumetric analysis, is the primary endpoint driving continued study participation.
04/24/2015 04:01 AM