A Phase IB/II Study of Immunotherapy with Nivolumab Alone and with Ipilimumab in Children with High-Grade Brain and Spinal Cord Cancers

Full Title

Phase Ib/II Clinical Trial of Nivolumab Monotherapy and Nivolumab in Combination with Ipilimumab in Pediatric Subjects with High Grade Primary CNS Malignancies


Nivolumab and ipilimumab are immunotherapy drugs that boost the power of the immune system to find and kill cancer cells. They work by inhibiting the actions of proteins that normally put the brakes on the immune response. Both drugs are approved for use, individually and together, to treat melanoma. Nivolumab is also used to treat other cancers.

In this study, researchers are assessing the safety and effectiveness of giving nivolumab alone and in combination with ipilimumab in children with high-grade tumors of the brain or spinal cord. The use of these drugs in children is considered investigational. Both drugs are given intravenously (by vein).


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

  • Patients must be at least 6 months of age and less than 22 years.
  • Patients must have high-grade glioma, diffuse intrinsic pontine glioma (DIPG), medulloblastoma, ependymoma, or other recurrent malignant brain tumor.
  • Patients with DIPG may enroll during radiation therapy or up to 4 weeks after radiation therapy. Patients with other tumor types must have recurrent disease.

For more information about this study and to inquire about eligibility, please contact Dr. Ira Dunkel at 212-639-2153.





Disease Status

Newly Diagnosed