Indications / Non-small cell lung cancer · Breast cancer · Pancreatic cancer · Colorectal cancer · Acute myeloid leukemia · Chronic myeloid leukemia · Multiple myeloma · Glioblastoma · Glioma · Neuroblastoma · Medulloblastoma · Diffuse midline glioma (DIPG) · Ependymoma · Atypical teratoid rhabdoid tumour (AT/RT) · Childhood acute lymphoblastic leukemia · Ewing sarcoma · Osteosarcoma · Rhabdomyosarcoma
Medulloblastoma
WNT, SHH, Group 3 and Group 4 disease, and relapse after craniospinal radiotherapy.
Live from ClinicalTrials.gov, fetched 2026-11-12 12:49 UTC. Every row links to its source record.
Drug trials updated in the last 16 months
80
Phase 1 to 3, interventional
For relapsed or refractory disease
51%
41 trial titles name relapsed, refractory or recurrent disease
Led by industry
10%
8 trials with a company as lead sponsor
9 of the 80 medulloblastoma trials are Phase 3; 7 were stopped early
Drug trials in this cancer with a registry update in the last 16 months, by phase. Each bar is split by where the trial stands today.
Running or not yet startedCompletedStopped early (terminated, withdrawn or suspended)
A trial that spans two phases counts as the later one. Stopped early is the registry status, whatever the reason given. Source: ClinicalTrials.gov, fetched live.
Programs: each drug in each segment
A program is one drug in one segment of medulloblastoma (biomarker, line of therapy, setting), so the same drug can be Phase 3 in one segment and Phase 1 in another. Segments are read from each trial's title and conditions by published rules; 18 of 80 trials name no segment and are listed as Untagged.
Segment:All1L1L · Relapsed or refractoryAdvanced or metastaticGroup 3 · Relapsed or refractoryLocally advancedMaintenanceRelapsed or refractorySHHSHH · Relapsed or refractoryWNT · 1LUntagged
Modality:AllAntibodyAntibody-drug conjugateBispecific antibodyCell therapyOther or not recognisedRadioligandSmall moleculeVaccine
| Drug | Segment | Modality | Highest phase | Trials |
|---|---|---|---|---|
| Temozolomide Theodore S. Johnson | 1L · Relapsed or refractory | Small molecule | Ph2 | NCT04049669, NCT06639607, NCT05096481 |
| Cyclophosphamide Theodore S. Johnson | 1L · Relapsed or refractory | Small molecule | Ph2 | NCT04049669 |
Assets over time
80 trials updated in the last 16 months. Each row is an asset; each dot is one of its trials at first posting, sized by phase. Orange is Phase 3.
| Asset | Lead sponsor | |
|---|---|---|
| Cyclophosphamide | Children's Oncology Group | |
| Induction Cycles A1-A3 | Nationwide Children's Hospital | |
| Metformin hydrochloride (HCl) | Donald Mabbott | |
| Metformin | The Hospital for Sick Children | |
| Vinblastin Maintenance | Universitätsklinikum Hamburg-Eppendorf | |
| Indoximod | Theodore S. Johnson | |
| Ensartinib | National Cancer Institute (NCI) | |
| Nivolumab | National Cancer Institute (NCI) | |
| Palbociclib | Pfizer | |
| PEP-CMV | Nationwide Children's Hospital | |
| PLX038 | National Cancer Institute (NCI) | |
| Digoxin | H. Lee Moffitt Cancer Center and Research Institute | |
| dasatinib | City of Hope Medical Center | |
| PEP-CMV vaccine | Washington University School of Medicine | |
| CX 4945 | Pediatric Brain Tumor Consortium | |
| [18F]DPA714 | St. Jude Children's Research Hospital | |
| Tipifarnib | National Cancer Institute (NCI) | |
| Low-Risk Therapy | St. Jude Children's Research Hospital | |
| Erdafitinib | National Cancer Institute (NCI) | |
| Bevacizumab | Medical University of Vienna | |
| Selpercatinib | National Cancer Institute (NCI) | |
| Arsenic Trioxide | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | |
| Liothyronine | Sabine Mueller, MD, PhD | |
| Entrectinib | Hoffmann-La Roche | |
| Ulixertinib | National Cancer Institute (NCI) | |
| Methotrexate | St. Jude Children's Research Hospital | |
| Gemcitabine (Gemzar®), Oxaliplatin | Gustave Roussy, Cancer Campus, Grand Paris | |
| TTRNA-xALT | University of Florida | |
| Difluoromethylornithine | Giselle Sholler | |
| Irinotecan | Pediatric Brain Tumor Consortium |
All trials
Site countries come from each record's listed locations. Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered.
| Trial | Phase | Sponsor | Status | Posted | Primary completion |
|---|---|---|---|---|---|
| NCT07291102 Comparison of Neurocognitive Outcome in Two Standard Regimen for Treatment of Low-risk Medulloblastoma Single-country: United States (2 sites) Decision brief → | Ph3 | Nationwide Children's Hospital | not yet recruiting | 2025-12-18 | 2034-10 |
| NCT05382338 A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss 2 countries, 95% of sites in the US Decision brief → | Ph3 | Children's Oncology Group | recruiting | 2022-05-19 | 2029-12-31 |
| Met Med Can · NCT05230758 Effect of Metformin on Behaviour and the Brain in Children Treated for a Brain Tumour 2 countries, 0% of sites in the US Decision brief → | Ph3 | Donald Mabbott | recruiting | 2022-02-09 | 2027-11-30 |
| NCT02066220 International Society of Paediatric Oncology (SIOP) PNET 5 Medulloblastoma 16 countries, 0% of sites in the US Decision brief → | Ph2/Ph3 | Universitätsklinikum Hamburg-Eppendorf | active not recruiting | 2014-02-19 | 2026-12 |
| NCT02040376 Metformin for Brain Repair in Children With Cranial-Spinal Radiation for Medulloblastoma Single-country: Canada (1 site) Decision brief → | Ph3 | The Hospital for Sick Children | completed | 2014-01-20 | 2017-12-15 |
| SIOP-PNET-4 · NCT01351870 Hyperfractionated Versus Conventionally Fractionated Radiotherapy in Standard Risk Medulloblastoma (PNET4) Single-country: France (1 site) Decision brief → | Ph3 | Institut Curie | completed | 2011-05-11 | 2008-12 |
| NCT00392327 Chemotherapy and Radiation Therapy in Treating Young Patients With Newly Diagnosed, Previously Untreated, High-Risk Medulloblastoma/PNET 5 countries, 92% of sites in the US Decision brief → | Ph3 | Children's Oncology Group | completed | 2006-10-26 | 2019-06-30 |
| NCT00336024 Combination Chemotherapy Followed By Peripheral Stem Cell Transplant in Treating Young Patients With Newly Diagnosed Supratentorial Primitive Neuroectodermal Tumors or High-Risk Medulloblastoma 4 countries, 89% of sites in the US Decision brief → | Ph3 | Children's Oncology Group | completed | 2006-06-12 | 2016-12-31 |
| NCT00085735 Comparison of Radiation Therapy Regimens in Combination With Chemotherapy in Treating Young Patients With Newly Diagnosed Standard-Risk Medulloblastoma 7 countries, 88% of sites in the US Decision brief → | Ph3 | Children's Oncology Group | completed | 2004-06-16 | 2016-03-31 |
| NCT07856069 Arsenic Trioxide Combined With Chemotherapy for Relapsed SHH-Activated and/or TP53-Mutant Medulloblastoma Single-country: China (1 site) Decision brief → | Ph2 | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | not yet recruiting | 2026-10-02 | 2030-10 |
| PNOC044 · NCT07346157 Liothyronine in Combination With BIT Regimen for Medulloblastoma With or Without Minimal Residual Disease Single-country: United States (3 sites) Decision brief → | Ph1/Ph2 | Sabine Mueller, MD, PhD | recruiting | 2026-01-16 | 2031-03-31 |
| EViDENCE-BT · NCT06804655 Pharmacoscopy for Patients With Refractory Primary Brain Tumors Single-country: Switzerland (3 sites) Decision brief → | Ph2 | University of Zurich | recruiting | 2025-02-03 | 2028-09-15 |
| NCT06701812 Digoxin Medulloblastoma Study Single-country: United States (18 sites) Decision brief → | Ph2 | H. Lee Moffitt Cancer Center and Research Institute | recruiting | 2024-11-22 | 2027-02 |
| PRiME II · NCT06639607 PEP-CMV + Nivolumab for Newly Diagnosed Diffuse Midline Glioma/High-grade Glioma and Recurrent Diffuse Midline Glioma/High-grade Glioma, Medulloblastoma, and Ependymoma Single-country: United States (3 sites) Decision brief → | Ph1/Ph2 | Washington University School of Medicine | suspended | 2024-10-15 | 2031-08-31 |
| LUPARPED · NCT06607692 Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib for the Treatment of Recurrent or Relapsed Solid Tumours Expressing Somatostatin Receptor (SSTR) (LuPARPed). Single-country: Spain (1 site) Decision brief → | Ph1/Ph2 | Fundación de investigación HM | recruiting | 2024-09-23 | 2029-07 |
| NCT06161519 PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications Single-country: United States (1 site) Decision brief → | Ph1/Ph2 | National Cancer Institute (NCI) | recruiting | 2023-12-08 | 2028-11-14 |
| NCT05535166 Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma Single-country: United States (10 sites) Decision brief → | Ph2 | St. Jude Children's Research Hospital | recruiting | 2022-09-10 | 2035-07 |
| NCT05278208 Lutathera for Treatment of Recurrent or Progressive High-Grade CNS Tumors Single-country: United States (4 sites) Decision brief → | Ph1/Ph2 | Nationwide Children's Hospital | recruiting | 2022-03-14 | 2028-11 |
| NCT05199584 A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations Single-country: United States (16 sites) Decision brief → | Ph2 | Endeavor Biomedicines, Inc. | completed | 2022-01-20 | 2024-02-20 |
| NCT05128903 Quantitative Assessment of Radiation-induced Neuroinflammation - A Proof of Principle Study Single-country: United States (1 site) Decision brief → | Ph2 | St. Jude Children's Research Hospital | active not recruiting | 2021-11-22 | 2035-10-26 |
| NCT05096481 PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent Medulloblastoma Single-country: United States (13 sites) Decision brief → | Ph2 | Nationwide Children's Hospital | recruiting | 2021-10-27 | 2028-06-15 |
| NCT04743661 131I-Omburtamab, in Recurrent Medulloblastoma and Ependymoma Single-country: United States (3 sites) Decision brief → | Ph2 | Pediatric Brain Tumor Consortium | active not recruiting | 2021-02-08 | 2026-04-01 |
| NCT04743310 Fluorescence Detection of Adult Primary Central Nervous System Tumors With Tozuleristide and the Canvas System Single-country: United States (1 site) Decision brief → | Ph2 | John Yu | completed | 2021-02-08 | 2025-03-06 |
| NCT04696029 DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma 2 countries, 96% of sites in the US Decision brief → | Ph2 | Giselle Sholler | recruiting | 2021-01-06 | 2028-03 |
| PersoMed-I · NCT04402073 Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma 8 countries, 0% of sites in the US Decision brief → | Ph2 | European Organisation for Research and Treatment of Cancer - EORTC | terminated | 2020-05-26 | 2025-06-16 |
| NCT04320888 Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial 3 countries, 99% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2020-03-25 | 2024-09-30 |
| NCT04284774 Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial 2 countries, 99% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2020-02-26 | 2024-03-31 |
| NCT04238819 A Study of Abemaciclib (LY2835219) in Combination With Other Anti-Cancer Treatments in Children and Young Adult Participants With Solid Tumors, Including Neuroblastoma 8 countries, 48% of sites in the US Decision brief → | Ph1/Ph2 | Eli Lilly and Company | completed | 2020-01-23 | 2024-03-15 |
| NCT04195555 Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial) 3 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2019-12-12 | 2025-03-31 |
| NCT04049669 Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG Single-country: United States (5 sites) Decision brief → | Ph2 | Theodore S. Johnson | active not recruiting | 2019-08-08 | 2026-09-21 |
| NCT03904862 Testing the Safety and Tolerability of CX-4945 in Patients With Recurrent Medulloblastoma Who May or May Not Have Surgery Single-country: United States (12 sites) Decision brief → | Ph1/Ph2 | Pediatric Brain Tumor Consortium | terminated | 2019-04-05 | 2025-08-11 |
| NCT03709680 Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors 13 countries, 67% of sites in the US Decision brief → | Ph2 | Pfizer | completed | 2018-10-17 | 2024-08-26 |
| NCT03698994 Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial) 2 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2018-10-09 | 2022-03-31 |
| NCT03526250 Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial) 2 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | completed | 2018-05-16 | 2023-03-31 |
| NCT03213652 Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial) 4 countries, 97% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2017-07-11 | 2025-09-30 |
| NCT03213704 Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial) 2 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2017-07-11 | 2024-09-30 |
| NCT03213678 Samotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial) 2 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | completed | 2017-07-11 | 2023-09-30 |
| NCT03213665 Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial) 2 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | completed | 2017-07-11 | 2022-03-31 |
| NCT03210714 Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial) 2 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2017-07-07 | 2023-09-30 |
| NCT03173950 Immune Checkpoint Inhibitor Nivolumab in People With Recurrent Select Rare CNS Cancers Single-country: United States (3 sites) Decision brief → | Ph2 | National Cancer Institute (NCI) | terminated | 2017-06-02 | 2025-06-23 |
| NCT03155620 Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial) 4 countries, 98% of sites in the US Decision brief → | Ph2 | National Cancer Institute (NCI) | active not recruiting | 2017-05-16 | 2025-03-31 |
| NCT02724579 Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma 5 countries, 89% of sites in the US Decision brief → | Ph2 | Children's Oncology Group | active not recruiting | 2016-03-31 | 2028-03-31 |
| STARTRK-NG · NCT02650401 Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options 9 countries, 58% of sites in the US Decision brief → | Ph1/Ph2 | Hoffmann-La Roche | active not recruiting | 2016-01-08 | 2027-06-30 |
| AflacST1502 · NCT02574728 Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors Single-country: United States (6 sites) Decision brief → | Ph2 | Emory University | active not recruiting | 2015-10-14 | 2026-06 |
| NCT01878617 A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma 4 countries, 61% of sites in the US Decision brief → | Ph2 | St. Jude Children's Research Hospital | active not recruiting | 2013-06-17 | 2028-10-13 |
| MEMMAT · NCT01356290 Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors 8 countries, 18% of sites in the US Decision brief → | Ph2 | Medical University of Vienna | recruiting | 2011-05-19 | 2030-04 |
| Re-MATCH · NCT01326104 Vaccine Immunotherapy for Recurrent Medulloblastoma and Primitive Neuroectodermal Tumor Single-country: United States (3 sites) Decision brief → | Ph2 | University of Florida | completed | 2011-03-30 | 2020-03-28 |
| NCT00840047 Methionine PET/CT Studies In Patients With Cancer Single-country: United States (1 site) Decision brief → | Ph2 | St. Jude Children's Research Hospital | active not recruiting | 2009-02-10 | 2027-07-27 |
| NCT00788125 Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors Single-country: United States (1 site) Decision brief → | Ph1/Ph2 | City of Hope Medical Center | terminated | 2008-11-10 | 2010-04-30 |
| NCT00602667 Risk-Adapted Therapy for Young Children With Embryonal Brain Tumors, Choroid Plexus Carcinoma, High Grade Glioma or Ependymoma 2 countries, 83% of sites in the US Decision brief → | Ph2 | St. Jude Children's Research Hospital | active not recruiting | 2008-01-28 | 2017-09-27 |
| NCT00407433 Clinical Studies of Gemcitabine-Oxaliplatin Single-country: France (1 site) Decision brief → | Ph2 | Gustave Roussy, Cancer Campus, Grand Paris | completed | 2006-12-05 | – |
| MEDWAY · NCT07865130 Study of CDK8 Inhibitor RVU120 in Combination With Everolimus in Children With Recurrent or Progressive Group 3 or 4 Medulloblastoma Single-country: Poland (1 site) Decision brief → | Ph1 | Bożenna Dembowska Bagińska | not yet recruiting | 2026-10-08 | 2033-06 |
| NCT07698899 A Study of 177Lu-DTPA-Omburtamab in Children and Adolescents With Brain Cancer or Cancer That Has Spread to the Central Nervous System (CNS) Single-country: United States (7 sites) Decision brief → | Ph1 | Memorial Sloan Kettering Cancer Center | recruiting | 2026-07-13 | 2030-06-02 |
| NCT07664176 NgFUS NIVO: NeuroNavigation-Guided Focused Ultrasound With Nivolumab in Relapsed and Progressive DMG and Other High Grade Brain Tumors Single-country: United States (1 site) Decision brief → | Ph1 | Children's National Research Institute | recruiting | 2026-06-24 | 2030-12 |
| MRG003 · NCT07584499 Phase I Study of Becotatug Vedotin for Safety and Efficacy in EGFR-Positive Pediatric Relapsed/Refractory or Metastatic Solid Tumors Single-country: China (1 site) Decision brief → | Ph1 | Sun Yat-sen University | recruiting | 2026-05-13 | 2027-05-30 |
| PNOC020 rMB · NCT07492316 RNA-lipid Particle (RNA-LP) Vaccines for Recurrent/Progressive Medulloblastoma (MB) Single-country: United States (1 site) Decision brief → | Ph1 | University of Florida | active not recruiting | 2026-03-25 | 2030-09-01 |
| NCT07390539 B7-H3.CD28Z.CART in CNS Neoplasms Single-country: United States (2 sites) Decision brief → | Ph1 | Robbie Majzner | recruiting | 2026-02-05 | 2030-08-31 |
| NCT07087002 GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults Single-country: United States (1 site) Decision brief → | Ph1 | Stanford University | recruiting | 2025-07-25 | 2027-08 |
| AflacBT2303 · NCT06624371 Atovaquone Combined With Radiation in Children With Malignant Brain Tumors Single-country: United States (2 sites) Decision brief → | Ph1 | Emory University | recruiting | 2024-10-03 | 2027-10 |
| MATCHPOINT · NCT06514898 Adoptive T Cell Therapy, DC Vaccines, and Hematopoietic Stem Cells Combined With Immune checkPOINT Blockade in Patients With Medulloblastoma Single-country: United States (2 sites) Decision brief → | Ph1 | University of Florida | recruiting | 2024-07-23 | 2027-09-01 |
| NCT06466798 Fourth Ventricular Administration of Immune Checkpoint Inhibitor (Nivolumab) and Methotrexate or 5-Azacytidine for Recurrent Medulloblastoma, Ependymoma, and Other CNS Malignancies No sites listed Decision brief → | Ph1 | Jeffrey Treiber | withdrawn | 2024-06-20 | 2027-07-01 |
| IMPACT · NCT06193759 Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT) Single-country: United States (1 site) Decision brief → | Ph1 | Children's National Research Institute | recruiting | 2024-01-05 | 2030-12-29 |
| NCT05835687 Loc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors Single-country: United States (1 site) Decision brief → | Ph1 | St. Jude Children's Research Hospital | active not recruiting | 2023-04-28 | 2026-06-04 |
| NCT05429502 Study of Efficacy and Safety of Ribociclib (LEE011) in Combination With Topotecan and Temozolomide (TOTEM) in Pediatric Patients With Relapsed or Refractory Neuroblastoma and Other Solid Tumors 4 countries, 50% of sites in the US Decision brief → | Ph1 | Novartis Pharmaceuticals | terminated | 2022-06-23 | 2025-02-26 |
| NCT05298995 GD2-CAR T Cells for Pediatric Brain Tumours Single-country: Italy (1 site) Decision brief → | Ph1 | Bambino Gesù Hospital and Research Institute | recruiting | 2022-03-28 | 2027-11 |
| NCT05106296 Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer Single-country: United States (1 site) Decision brief → | Ph1 | Theodore S. Johnson | recruiting | 2021-11-03 | 2028-03-30 |
| NCT04978727 A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma 2 countries, 93% of sites in the US Decision brief → | Ph1 | Children's Oncology Group | active not recruiting | 2021-07-27 | 2027-12-31 |
| NCT04661384 Brain Tumor-Specific Immune Cells (IL13Ralpha2-CAR T Cells) for the Treatment of Leptomeningeal Glioblastoma, Ependymoma, or Medulloblastoma Single-country: United States (1 site) Decision brief → | Ph1 | City of Hope Medical Center | active not recruiting | 2020-12-10 | 2027-01-12 |
| PNOC020 · NCT04573140 A Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM) Single-country: United States (2 sites) Decision brief → | Ph1 | University of Florida | recruiting | 2020-10-05 | 2027-07-01 |
| NCT04541082 Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms Single-country: United States (1 site) Decision brief → | Ph1 | Jazz Pharmaceuticals | active not recruiting | 2020-09-09 | 2027-03-31 |
| NCT04337177 Flavored, Oral Irinotecan VAL-413 (Orotecan®) Given With Temozolomide for Treatment of Recurrent Pediatric Solid Tumors Single-country: United States (11 sites) Decision brief → | Ph1 | Valent Technologies, LLC | active not recruiting | 2020-04-07 | 2026-12 |
| NCT04185038 Study of B7-H3-Specific CAR T Cell Locoregional Immunotherapy for Diffuse Intrinsic Pontine Glioma/Diffuse Midline Glioma and Recurrent or Refractory Pediatric Central Nervous System Tumors Single-country: United States (1 site) Decision brief → | Ph1 | Seattle Children's Hospital | recruiting | 2019-12-04 | 2027-05 |
| NCT03911388 HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors Single-country: United States (3 sites) Decision brief → | Ph1 | M.D. Anderson Cancer Center | recruiting | 2019-04-11 | 2026-09-01 |
| NCT03638167 EGFR806-specific CAR T Cell Locoregional Immunotherapy for EGFR-positive Recurrent or Refractory Pediatric CNS Tumors Single-country: United States (1 site) Decision brief → | Ph1 | Seattle Children's Hospital | completed | 2018-08-20 | 2023-12-22 |
| NCT03598244 Savolitinib in Treating Patients With Recurrent or Refractory Primary CNS Tumors 2 countries, 92% of sites in the US Decision brief → | Ph1 | National Cancer Institute (NCI) | active not recruiting | 2018-07-26 | 2026-04-22 |
| NCT03500991 HER2-specific CAR T Cell Locoregional Immunotherapy for HER2-positive Recurrent/Refractory Pediatric CNS Tumors Single-country: United States (1 site) Decision brief → | Ph1 | Seattle Children's Hospital | active not recruiting | 2018-04-18 | 2024-01-17 |
| CLOVER-2 · NCT03478462 Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas 3 countries, 75% of sites in the US Decision brief → | Ph1 | Cellectar Biosciences, Inc. | active not recruiting | 2018-03-27 | 2027-06-30 |
| NCT03389802 Phase I Study of APX005M in Pediatric Central Nervous System Tumors Single-country: United States (11 sites) Decision brief → | Ph1 | Pediatric Brain Tumor Consortium | completed | 2018-01-04 | 2023-09-30 |
| PRiME · NCT03299309 PEP-CMV in Recurrent MEdulloblastoma/Malignant Glioma Single-country: United States (1 site) Decision brief → | Ph1 | Daniel Landi | active not recruiting | 2017-10-03 | 2023-04-27 |
| NCT02359565 Pembrolizumab in Treating Younger Patients With Recurrent, Progressive, or Refractory High-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma or Medulloblastoma 2 countries, 92% of sites in the US Decision brief → | Ph1 | National Cancer Institute (NCI) | active not recruiting | 2015-02-10 | 2026-01-05 |