A Phase I Study of Vorasidenib with Pembrolizumab Immunotherapy for Recurrent or Persistent IDH-1 Mutant Astrocytomas


Full Title

A Phase 1, Safety Lead-In and Randomized, Open-label, Perioperative Study of Vorasidenib in Combination with Pembrolizumab in Subjects with Recurrent or Progressive Enhancing IDH-1 Mutant Glioma


The purpose of this study is to find the highest dose of vorasidenib that can be given safely in combination with pembrolizumab immunotherapy in people with astrocytoma that came back or continued to grow despite treatment and contains a mutation in the IDH-1 protein.

Vorasidenib blocks mutated IDH-1 proteins in cancer cells and may stop or slow cancer growth. It has been specifically developed for penetrating brain tissue. Pembrolizumab takes the brakes off the immune response, helping immune cells to find and destroy cancer cells. Vorasidenib is taken orally (by mouth) and pembrolizumab is given intravenously (by vein).

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have Grade 2 or Grade 3 astrocytoma that has come back or continued to grow despite chemotherapy and/or radiation treatment and contains a mutated IDH-1 protein.
  • Patients must have a measurable lesion of at least 1 cm in diameter that can be seen on MRI.
  • Patients must be physically well enough that they are able to be mobile, take care of themselves, and engage in all but physically strenuous activities. For example, they must be well enough that they could carry out office work or light housework.
  • This study is for patients age 18 and older.


For more information about this study and to ask about eligibility, please call 212-639-6767.




Phase I (phase 1)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID