Table 3.
DMG/DIPG trials still recruiting.
| Number of Trial | Study Name | Phase | Countries | Start Date | Enrollment Size | Primary Outcome | Secondary Outcome |
|---|---|---|---|---|---|---|---|
| DIPG/DMG | |||||||
| NCT04250064 | A Study of Low-Dose Bevacizumab with Conventional Radiotherapy Alone in Diffuse Intrinsic Pontine Glioma | II | India | February 2020 | 40 patients | OS | PFS, AE, steroid use, pattern of relapse, QoL |
| (LoBULarDIPG) | |||||||
| NCT04532229 | Nimotuzumab in Combined with Concurrent Radiochemotherapy in the Treatment of Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) in Children | III | China | April 2021 | 48 patients | OR | 1-year OS, PFS |
| NCT04771897 | A Study of BXQ-350 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG) (KONQUER) | I | USA | May 2021 | 22 patients | AE, MTD | OS, QoL |
| NCT04943848 | rHSC-DIPGVax Plus Checkpoint Blockade for the Treatment of Newly Diagnosed DIPG and DMG | I | USA | January 2022 | 36 patients | MTD, DLT | 1-year OS, TTP |
| NCT02992015 | Gemcitabine in Newly Diagnosed DIPG | Early I | USA | September 2016 | 10 patients | PK testing level | - |
| NCT05077735 | Stereotactic Biopsy Split-Course Radiation Therapy in DMG (SPORT-DMG Study) | II | USA | October 2021 | 18 patients | TTP | QoL; PFS; OS; SAE |
| NCT04749641 | Neoantigen Vaccine Therapy Against H3.3-K27M Diffuse Intrinsic Pontine Glioma (ENACTING) | I | China | March 2021 | 30 patients | Safety, 1 year-OS | MTD, median PFS and OS |
| NCT04771897 | A Study of BXQ-350 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG) (KONQUER) | I/II | USA | February 2021 | 22 patients | MTD; SAE, PK | OR; OS; QoL |
| NCT03126266 | Re-Irradiation of Progressive or Recurrent DIPG | NA | Canada | April 2017 | 27 patients | Second PFS | OS |
| NCT03396575 | Brain Stem Gliomas Treated with Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-Intensified Temozolomide (Phase I-BRAVO) | I | USA | May 2018 | 21 patients | Safety and feasibility, DLT | PFS, OS |
| NCT03620032 | Study of Re-irradiation at Relapse Versus RT and Multiple Elective rt Courses (DIPG) | II | Italy | November 2015 | 54 patients | PFS | PFS, OS, RT toxicity, QoL |
| NCT05009992 | Combination Therapy for DMG | II | USA | October 2021 | 216 patients | PFS, OS | |
| NCT04264143 | CED of MTX110 Newly Diagnosed Diffuse Midline Gliomas | I | USA | March 2020 | 9 patients | AE, MTD | PFS, OS |
| NCT05063357 | 131I-omburtamab Delivered by CED in DIPG Patients | I | USA | October 2021 | 36 patients | Safety | PFS |
| NCT04804709 | Non-Invasive FUS With Oral Panobinostat in Children with Progressive DMG | I | USA | March 2021 | 3 patients | SAE | 6-month PFS; 6-month OS; |
| NCT04196413 | GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG) | I | USA | September 2020 | 54 patients | Safety, feasibility, DLT | OS; PFS |
| NCT05478837 | Genetically Modified Cells (KIND T Cells) for the Treatment of HLA-A*0201-Positive Patients With H3.3K27M-Mutated Glioma (PNOC018) | I | USA | July 2022 | 12 patients | MTD; safety | Manufacturing feasibility |
| NCT05476939 | Biological Medicine for DIPG Eradication 2.0 (BIOMEDE 2) | I | France, USA | July 2022 | 368 patients | MTD, safety of infusions | Manufacturing feasibility |
| DMG and other tumors | |||||||
| NCT02960230 | H3.3K27M Peptide Vaccine with Nivolumab for Children With Newly Diagnosed DIPG and Other Gliomas | I/II | USA, Switzerland | November 2016 | 50 patients | AE; OS | - |
| NCT03696355 | Study of GDC-0084 in Pediatric Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma or Diffuse Midline Gliomas | I | USA | October 2018 | 27 patients | MTD; SAE; | RR; DoR; OS; PFS |
| NCT01922076 | Adavosertib and Local Radiation Therapy in Treating Children with Newly Diagnosed DIPG | I | USA | April 2013 | 46 patients | MTD; SAE | PK; RR; PFS; OS; |
| NCT04758533 | Clinical Trial to Assess the Safety and Efficacy of AloCELYVIR with Newly DIPG in Combination With Radiotherapy or Medulloblastoma in Monotherapy (AloCELYVIR) | I/II | Spain | April 2021 | 12 patients | DLT | OS, AE |
| NCT03605550 | A Phase 1B Study of PTC596 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High-Grade Glioma | I | USA | August 2018 | 54 patients | MTD, AE, PK | PFS, OS |
| NCT03652545 | Multi-antigen T Cell Infusion Against Neuro-Oncologic Disease (REMIND) | I | USA | December 2018 | 32 patients | Aes | OR |
| NCT04049669 | Pediatric Trial of Indoximod with Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG | II | USA | October 2019 | 140 patients | 8 months PFS, | OS, PFS TTP |
| 12 months OS | |||||||
| NCT04911621 | Adjuvant Dendritic Cell Immunotherapy for Pediatric Patients with HGG or DIPG (ADDICT-pedGLIO) | I/II | Belgium | September 2021 | 10 patients | Safety and feasibility | OS, PFS, TTP |
| NCT04837547 | PEACH TRIAL Precision Medicine and Adoptive Cellular Therapy (PEACH) for Neuroblastoma and DIPG | I | USA | September 2021 | 24 patients | DLT | AE, safety, feasibility; OS, PFS, ORR |
| NCT02644460 | Abemaciclib in Children with DIPG or Recurrent/Refractory Solid Tumors (AflacST1501) | I | USA | February 2016 | 60 patients | DLT, MTD, PK | AE, hematological toxicities |
| NCT03416530 | ONC201 in Pediatric H3 K27M Gliomas | I | USA | January 2018 | 130 patients | RP2D | - |
| NCT02525692 | Oral ONC201 in Recurrent GBM, H3 K27M Glioma, and Midline Glioma | II | USA | August 2015 | 89 patients | PFS | - |
| NCT04541082 | Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms | I | USA | September 2020 | 102 patients | MTD | - |
| NCT04732065 | ONC206 for the Treatment of Newly Diagnosed or Recurrent DMG and Other Recurrent Malignant CNS Tumors (PNOC 023) | I | USA, Switzerland | August 2021 | 250 patients | DLT, MTD | PK parameters |
| NCT04185038 | Study of B7-H3-Specific CAR T Cell Locoregional Immunotherapy for DIPG/DMG and Recurrent or Refractory Pediatric Central Nervous System Tumors | I | USA | December 2019 | 90 patients | Safety and feasibility | Distribution of CNS-CART cells, RR |
| NCT02359565 | Pembrolizumab in Treating Younger Patients with Recurrent, Progressive, Refractory HGG, DIPG, Hyper-Mutated tumors, Ependymoma, or Medulloblastoma | I | USA | May 2015 | 110 patients | AE, OR | PFS, EFS, OS, radiological response |
| NCT05009992 | Combination Therapy for the Treatment of DMG | II | USA | August 2021 | 216 patients | 6-months PFS; 7-months OS | |
| NCT03893487 | Fimepinostat in Treating Brain Tumors in Children and Young Adults (PNOC016) | I | USA | August 2019 | 30 patients | BBB penetration | - |
| NCT03243461 | International Cooperative Phase III Trial of the HIT-HGG Study Group (HIT-HGG-2013) | III | Germany | July 2018 | 167 patients | EFS | - |
| NCT03598244 | Volitinib in Treating Patients with Recurrent or Refractory Primary CNS Tumors | I | USA | October 2018 | 50 patients | MTD, RP2D | CR, PR, PK |
| NCT03690869 | REGN2810 in Pediatric Patients With Relapsed, Refractory Solid, or Central Nervous System (CNS) Tumors and Safety and Efficacy of REGN2810 in Combination With Radiotherapy in Pediatric Patients With Newly Diagnosed or Recurrent Glioma | I/II | USA | October 2018 | 130 patients | AE, SAE, DLT, PK, OR, PFS | OR |
| NCT04099797 | C7R-GD2.CAR T Cells for Patients with GD2-Expressing Brain Tumors (GAIL-B) | I | USA | February 2020 | 34 patients | DLT | RR |
| NCT01837862 | A Phase I Study of Mebendazole for the Treatment of Pediatric Gliomas | I | USA | October 2013 | 36 patients | MTD | EFS, OS, PR o CRR |
| NCT04239092 | 9-ING-41 in Pediatric Patients with Refractory Malignancies | I | USA | June 2020 | 68 patients | AE | - |
| NCT03478462 | Dose Escalation Study of CLR 131 in Children, Adolescents, and Young Adults with Relapsed or Refractory Malignant Tumors Including, But Not Limited to, Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma, and Osteosarcoma (CLOVER-2) | I | USA | April 2019 | 30 patients | DLT | EFS, OS, dosimetry |
| NCT03389802 | Phase I Study of APX005M in Pediatric CNS Tumors | I | USA | February 2018 | 45 patients | AE, MTD, DLT, PK | ORR, PFS, OS |
| NCT04295759 | INCB7839 in Treating Children with Recurrent/Progressive HGG | I | USA | May 2020 | 28 patients | AE, MTD, CMAX | PFS, OS, TTP |
| NCT01884740 | Intraarterial Infusion of Erbitux and Bevacizumab for Relapsed/Refractory Intracranial Glioma In Patients Under 22 | I/II | USA | June 2013 | 30 patients | ORR | AE, PFS, OS |
| NCT03709680 | Study Of Palbociclib Combined with Chemotherapy in Recurrent/Refractory Solid Tumors | I/II | USA | May 2019 | 167 patients | EFS, DLT, AE | AE, CR or PR, DoR, PFS, OS, PK, Tmax |
| NCT04870944 | CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma | I/II | USA | January 2022 | 38 patients | DLT, anti-tumor effect | AE, min-max SC, clearance, IR, OS, PFS |
| NCT05135975 | A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors | II | USA | October 2021 | 100 patients | 1-year PFS | 1–2–5 year OS, 2–5 year PFS, DoR, AE |
| NCT04730349 | 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) | I/II | USA | June 2021 | 234 patients | DLT, AE, SAE, PK, ORR | PFS, OS |
| NCT04238819 | A Study of Abemaciclib (LY2835219) in Combination with Temozolomide, Irinotecan, and Abemaciclib in Combination with Temozolomide in Children and Young Adult Participants with Solid Tumors | I | USA, Europe, Asia | November 2020 | 60 patients | DLT, PK | ORR, DoR, CBR, DCR |
| NCT05298995 | GD2-CAR T Cells for Pediatric Brain Tumors | I | Italy | May 2022 | 54 patients | Safety and MTD | Expansion infiltration, TTP, EFS, OS |
| NCT05099003 | A Study of the Drug Selinexor with Radiation Therapy in Patients with Newly Diagnosed DIPG H3K27M-Mutant HGG | I/II | USA | October 2021 | 36 patients | MTD, | - |
| EFS | |||||||
| OS, OR | |||||||
| NCT05123534 | A Phase 1/2 Study of Sonodynamic Therapy Using SONALA-001 and Exablate 4000 Type 2 in DIPG Patients | II | USA | November 2021 | 18 patients | Safety; | OR, TTP, OS |
| MTD | |||||||
| NCT05169944 | Magrolimab in Children and Adults with Recurrent or Progressive Malignant Brain Tumors (PNOC025) | I | USA | December 2021 | 24 patients | Definition of phase II-MTD; | - |
| SAE | |||||||
| NCT05096481 | PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent Medulloblastoma | II | USA | October 2021 | 120 patients | 4-months PFS; 1-year PFS, 1 year-OS | 1-year PFS in rMB, 1-year OS in rHHG |
| NCT05278208 | Lutathera for Treatment of Recurrent or Progressive High-Grade CNS Tumors or Meningiomas Expressing SST2A | I/II | USA | March 2022 | 65 patients | MTD, SAE | OR |
| PFS |
SAE: severe adverse event; OR: objective response; SC: serum concentration; CED: convection-enhanced delivery; FUS: focus ultrasound; EFS: event-free survival; PK: pharmacocynethic; RR: response rate; DoR: duration of response; CBR: clinical benefit rate; DCR: disease control rate; RP2D: recommended phase II dose.