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A Phase 2 Trial of MEK Inhibitor PD-0325901 in Adolescents and Adults with NF1-associated Plexiform Neurofibromas


The MEK Study is designed to evaluate whether PD-0325901 will shrink target lesion as noted by radiographic response. The trial will also evaluate the effects, good and/or bad, of PD-0325901.


PD-0325901 is a new study medication that is also currently being tested in clinical cancer trials. PD-0325901 appears to inhibit neurofibroma growth.


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.


Subjects will receive PD-0325901 by mouth twice daily on a 3 week on/1 week off 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.


  • Brian Weiss, MD, Chair
  • Scott Plotkin, MD, Co-Chair
  • Brigitte Widemann, MD, Co-Chair


Enrollment is complete. The study is in data analysis.