Immunotherapy in children with highly malignant primary CNS tumors

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

In this study, children and adolescents aged 6 months to 22 years with high-grade brain tumors (diffuse intrinsic pons glion (DIPG), medullablastoma, ependynoma, some rare tumors (pineoblastoma, atypical teratoid rhabdoid tumors (AT / RT) and embryonic CNS tumors are treated. The study aims to examine the efficacy, safety and tolerability of investigational medicinal products.

Nivolumab is a monoclonal antibody designed to help the body's immune system fight against the tumor cells. It is already being used to treat various cancers in adults (lung cancer, melanoma, renal cell carcinoma). The antibody nivolumab binds to the PD-1 receptor on T cells and inhibits the interaction with ligands PD-L1 and PD-L2 on cancer cells. This stimulates the immune system. The medicine is administered as an intravenous infusion.

Ipilimumab is an active substance in the group of monoclonal antibodies with indirect cytotoxic and antitumoral properties. It enhances the T-cell mediated immune response by binding to CTLA-4 (cancer immunotherapy). The medicine is administered as an intravenous infusion. The combination therapy of nivolumab and ipilimumab has been approved in the EU for adult patients with advanced melanoma since 2016.

Recruitment is closed.

  • Age ≥ 6 months and < 22 years
  • Cohort 1: newly diagnosed DIPG after irradiation therapy
  • Cohort 2: histologically diagnosed, recurrent or progressive HGG (highgrade glioma) after sugery and irradiation
  • Cohort 3: histologically diagnosed, recurrent or first line therapy resistant medulloblastoma (incl. irradiation)
  • Cohort 4: histologically diagnosed, recurrent or first line therapy resistant epnendymoma (incl. surgery and irradiation)
  • Cohort 5: first line therapy resistant or progressive tumor


For more information, see the Study Registry: The study is registered with the U.S. National Institutes of Health (
See NCT03130959.

EudraCT-number: 2016004441-82 (see EU clinical trial register).