human stem cell derived neurons

REGN2810 in Pediatric Patients With Relapsed, Refractory Solid, or CNS Tumors and Safety and Efficacy of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed or Recurrent Glioma (PNOC013)


This phase I/II  clinical trial will evaluate REGN2810 (also called cemiplimab) in combination with radiation therapy as a treatment for pediatric patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG), newly diagnosed or recurrent high-grade glioma (HGG), and relapsed/refractory solid or central nervous system (CNS) tumors.  

REGN2810 (or cemiplimab) is an antibody-based immunotherapy, administered intravenously, that also targets the immune system and activates it to stop cancer growth and/or kill cancer cells. More specifically, REGN2810 works by binding and inhibiting PD-1, a protein that normally protects the body from attacking itself. Normally, PD-1 works by detecting a molecular signal (called PDL-1) made by various cells across the body. Some cancer cells take advantage of this protection mechanism making the protective signal themselves. Blocking PD-1 with REGN2810 allows the immune system to activate its T-cells and recognize these tumors as cells to be attacked.

In this study, REGN2810 will be evaluated in combination with radiation therapy. Preclinical data suggest that radiation “primes” the immune system, which may increase the efficacy of the REGN2810 when combined. 

This phase I/II study will evaluate REGN2810 in combination with radiation therapy as a treatment for children and young adults with newly diagnosed DIPG, newly diagnosed or recurrent HGG, and relapsed/refractory solid or CNS tumors. Eligible participants will be assigned to the following groups, based on study phase and diagnosis

  • Experimental Group (Phase I, Solid Tumor and CNS Tumor Cohorts): REGN2810
  • Experimental Group (Phase II, Newly Diagnosed DIPG): REGN2810 + radiation therapy
  • Experimental Group (Phase II, Newly Diagnosed HGG): REGN2810 + radiation therapy
  • Experimental Group (Phase II, Recurrent HGG): REGN2810 + re-irradiation


Inclusion Criteria
  1. Age 0 to <18 years of age (Phase 1)
  2. Age ≥3 and ≤25 years of age (Efficacy Phase)
  3. Karnofsky ≥50 for patients >16 years of age and Lansky ≥50 for patients ≤ 16 years of age
  4. Patients who are receiving corticosteroids must be on a stable or decreasing dose for at least 7 days prior to enrollment
  5. Adequate Bone Marrow Function
  6. Adequate Renal Function
  7. Adequate Liver Function
  8. Adequate Neurologic Function

Other protocol Inclusion/Exclusion criteria apply. For the most up-to-date information, please visit

Exclusion Criteria
  1. Patients with bulky, metastatic disease of the CNS causing Uncal herniation or symptomatic midline shift, significant, symptomatic mass effect, or uncontrolled neurological symptoms such as seizures or altered mental status
  2. Patients with metastatic spine disease and gliomatosis as documented by diffuse involvement of greater than 2 lobes
  3. Patients who are receiving any other investigational agents
  4. Patients on greater than dexamethasone 0.1 mg/kg/day (maximum 4 mg/day) or equivalent dose in alternate corticosteroid or actively undergoing corticosteroid dose escalation
  5. Patients with a history of allogeneic stem cell transplant
  6. Prior treatment with an agent that blocks the PD-1/PD-L1/PD-L2 pathway
  7. Prior treatment with idelalisib

Other protocol Inclusion/Exclusion criteria apply. For the most up-to-date information, please visit


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