A Phase II Study of Targeted Therapies for Meningiomas with Specific Genetic Mutations


Full Title

Phase II Trial of SMO/AKT/NF2 Inhibitors in Progressive Meningiomas with SMO/AKT/NF2 Mutations (A071401) (CIRB)


This study is assessing the safety and effectiveness of two drugs against meningiomas that contain certain genetic mutations. One mutation is in the SMO gene and the other is in the NF2 gene. Patients whose tumors contain an SMO mutation will receive the drug vismodegib, which blocks the receptor for the SMO protein. Those whose tumors contain NF2 mutations will receive GSK2256098, which seems to work better against tumors with this mutation. Both drugs are taken orally (by mouth).

Some patients may have mutations in both genes. In this case, they’ll receive one drug first, and if the tumor continues growing, they’ll receive the other drug instead.

GSK2256098 is an investigational drug. Vismodegib is approved for treating basal cell skin cancer, but its use in this study is considered investigational.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a meningioma that has gotten bigger or grew back after treatment.
  • Patients’ tumors must contain an SMO mutation and/or an NF2 mutation.
  • Prior therapy is allowed. At least 4 weeks must pass between the completion of chemotherapy or brain surgery and 6 months since radiation therapy and entry into the study.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is open to patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Thomas Kaley at 212-639-5122 or the Brain Tumor Hotline at 212-639-6767.





Disease Status

Newly Diagnosed & Relapsed/Refractory