Table 1.

Clinical trials involving immune checkpoint-targeting immunotherapy in medulloblastoma.

Trial ID	Title	Phase	Treatment	Target	Indications	Age	N	Status
NCT 04730349	A Study of Bempegaldesleukin (BEMPEG: NKTR-214) in Combination with Nivolumab in Children, Adolescents and Young Adults with Recurrent or Treatment-resistant Cancer (PIVOT IO 020)	1/2	i.v. nivolumab with bempegaldesleukin (BEMPEG: NKTR-214)	PD1, CD122	Ependymoma Ewing sarcoma High-grade glioma Leukemia and lymphoma Medulloblastoma Miscellaneous brain tumors Miscellaneous solid tumors Neuroblastoma Relapsed, refractory malignant neoplasms Rhabdomyosarcoma	<18 and 18–30 years	228	Not yet recruiting
NCT 03130959	An Investigational Immuno-therapy Study of Nivolumab Monotherapy and Nivolumab in Combination with Ipilimumab in Pediatric Patients with High Grade Primary CNS Malignancies (CheckMate 908)	2	Nivolumab, ipilimumab	PD1, CTLA-4	Various Advanced Cancer (including MB)	6 months–21 years	166	Active, not recruiting
NCT 03173950	Immune Checkpoint Inhibitor Nivolumab in People with Recurrent Select Rare CNS Cancers	2	i.v. nivolumab	PD1	Medulloblastoma Ependymoma Pineal region tumors Choroid plexus tumors Atypical/malignant meningioma	>18 years	180	Recruiting
NCT 02359565	Pembrolizumab in Treating Younger Patients with Recurrent, Progressive, or Refractory High-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma or Medulloblastoma	1	i.v. pembrolizumab	PD1	Constitutional Mismatch repair Deficiency syndrome Lynch syndrome Malignant glioma Recurrent brain neoplasm Recurrent childhood ependymoma Recurrent diffuse intrinsic pontine glioma Recurrent medulloblastoma Refractory brain neoplasm Refractory diffuse intrinsic pontine glioma Refractory ependymoma Refractory medulloblastoma	1–29 years	110	Recruiting
NCT 03838042	INFORM2 Study Uses Nivolumab and Entinostat in Children and Adolescents with High-risk Refractory Malignancies (INFORM2 NivEnt)	1/2	Nivolumab and entinostat	PD1	CNS Tumor Solid Tumor	6–21 Years	128	Recruiting
NCT 02502708	Study of the IDO Pathway Inhibitor, Indoximod, and Temozolomide for Pediatric Patients with Progressive Primary Malignant Brain Tumors	1	Oral indoximod with radiation therapy, temozolomide, or cyclophosphamide and etoposide	IDO	Glioblastoma multiforme Glioma Gliosarcoma Malignant brain tumor Ependymoma Medulloblastoma Diffuse intrinsic pontine glioma Primary CNS tumor	3–21 years	81	Completed
NCT 04049669	Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG	2	Oral indoximod with combinations of temozolomide, cyclophosphamide, etoposide, lomustine and radiation therapy.	IDO	Glioblastoma Medulloblastoma Ependymoma Diffuse intrinsic pontine glioma	3–21 years	140	Recruiting
NCT 03389802	Phase I Study of APX005M in Pediatric CNS Tumors	1	i.v. APX005M	CD40	Glioblastoma Multiforme High-grade astrocytoma, NOS CNS primary tumor, NOS Ependymoma, NOS Diffuse intrinsic pontine gliomas Medulloblastoma	1–21 years	45	Recruiting
NCT 04167618	177Lu-DTPA-Omburtamab Radio-immunotherapy for Recurrent or Refractory Medulloblastoma	1/2	177Lu-DTPA-omburtamab radio-immunotherapy	B7-H3	Pediatric medulloblastoma	3–19 years	40	Not yet recruiting
NCT 04743661	131I-Omburtamab, in Recurrent Medulloblastoma and Ependymoma	2	cRIT 131I-omburtamab radio-immunotherapy with Irinotecan, temozolomide, and bevacizumab.	B7-H3	Recurrent medulloblastoma Recurrent ependymoma	<22 years	62	Not yet recruiting

Abbreviations: i.v., intravenous; IDO, indoleamine-pyrrole 2,3-dioxygenase; PD1, programmed cell death protein 1. All data concerning the clinical trials were obtained from ClinicalTrials.gov (accessed on 25 May 2021).