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
Atypical teratoid rhabdoid tumour (AT/RT)
A SMARCB1-deficient brain tumour of infants and young children: EZH2 inhibition and intensive chemotherapy.
Live from ClinicalTrials.gov, fetched 2026-11-11 23:47 UTC. Every row links to its source record.
Drug trials updated in the last 16 months
53
Phase 1 to 3, interventional
For relapsed or refractory disease
53%
28 trial titles name relapsed, refractory or recurrent disease
Led by industry
9%
5 trials with a company as lead sponsor
2 of the 53 atypical teratoid rhabdoid tumour (at/rt) trials are Phase 3; 1 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Running or not yet startedCompletedStopped early (terminated, withdrawn or suspended)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 atypical teratoid rhabdoid tumour (at/rt) (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; 15 of 53 trials name no segment and are listed as Untagged.
Segment:AllLocally advancedMaintenanceRelapsed or refractorySMARCB1SMARCB1 · Relapsed or refractoryUntagged
| Drug | Segment | Modality | Highest phase | Trials |
|---|---|---|---|---|
| Cabozantinib National Cancer Institute (NCI) | Locally advanced | Small molecule | Ph3 | NCT03793166 |
| Cyclophosphamide Stanford University | Relapsed or refractory | Small molecule | Ph2 | NCT07087002, NCT03709680, NCT07390539, NCT02574728 |
| Palbociclib Pfizer | Relapsed or refractory | Small molecule | Ph2 | NCT03709680, NCT03155620, NCT03526250 |
| Temozolomide Pfizer | Relapsed or refractory | Small molecule | Ph2 | NCT03709680, NCT04901702, NCT04238819 |
| cyclophosphamide St. Jude Children's Research Hospital | Untagged | Small molecule | Ph2 | NCT02114229, NCT04897321 |
| Ensartinib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03213652, NCT03155620 |
| Erdafitinib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03210714, NCT03155620 |
| Ivosidenib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03155620, NCT04195555 |
| Larotrectinib Sulfate National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03155620, NCT03213704 |
| Samotolisib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03155620, NCT03213678 |
| Selpercatinib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT04320888, NCT03155620 |
| Tipifarnib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT04284774, NCT03155620 |
| Ulixertinib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03698994, NCT03155620 |
| Abemaciclib Eli Lilly and Company | Relapsed or refractory | Small molecule | Ph1/Ph2 | NCT04238819 |
| alisertib St. Jude Children's Research Hospital | Untagged | Small molecule | Ph2 | NCT02114229 |
| Cabozantinib Nationwide Children's Hospital | Maintenance | Small molecule | Ph2 | NCT05135975 |
| Celecoxib Emory University | Relapsed or refractory | Small molecule | Ph2 | NCT02574728 |
| dasatinib City of Hope Medical Center | Relapsed or refractory | Small molecule | Ph1/Ph2 | NCT00788125 |
| ifosfamide City of Hope Medical Center | Relapsed or refractory | Small molecule | Ph1/Ph2 | NCT00788125 |
| Olaparib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03155620 |
| Paxalisib Sabine Mueller, MD, PhD | Relapsed or refractory | Small molecule | Ph2 | NCT07447076 |
| Selumetinib Sulfate National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03155620 |
| Talazoparib St. Jude Children's Research Hospital | Relapsed or refractory | Small molecule | Ph1/Ph2 | NCT04901702 |
| Tazemetostat National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03155620 |
| Tazemetostat Susan Chi, MD | SMARCB1 | Small molecule | Ph1/Ph2 | NCT05407441 |
| Tazemetostat National Cancer Institute (NCI) | SMARCB1 · Relapsed or refractory | Small molecule | Ph2 | NCT03213665 |
| Vemurafenib National Cancer Institute (NCI) | Relapsed or refractory | Small molecule | Ph2 | NCT03155620 |
Assets over time
53 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 | |
|---|---|---|
| Cabozantinib | National Cancer Institute (NCI) | |
| Dactinomycin | Children's Oncology Group | |
| Ensartinib | National Cancer Institute (NCI) | |
| Palbociclib | Pfizer | |
| Tazemetostat | Susan Chi, MD | |
| Paxalisib | Sabine Mueller, MD, PhD | |
| dasatinib | City of Hope Medical Center | |
| Selinexor | Memorial Sloan Kettering Cancer Center | |
| Nivolumab | Dana-Farber Cancer Institute | |
| Tipifarnib | National Cancer Institute (NCI) | |
| Low-Risk Therapy | St. Jude Children's Research Hospital | |
| Erdafitinib | National Cancer Institute (NCI) | |
| Selpercatinib | National Cancer Institute (NCI) | |
| Ulixertinib | National Cancer Institute (NCI) | |
| Eflornithine | Milton S. Hershey Medical Center | |
| alisertib | St. Jude Children's Research Hospital | |
| ONO-4538 | Ono Pharmaceutical Co., Ltd. | |
| Atezolizumab | National Cancer Institute (NCI) | |
| Ivosidenib | National Cancer Institute (NCI) | |
| Zoledronic acid | University of Florida | |
| Larotrectinib Sulfate | National Cancer Institute (NCI) | |
| Onivyde | St. Jude Children's Research Hospital | |
| Samotolisib | National Cancer Institute (NCI) | |
| Sirolimus | Emory University | |
| Abemaciclib | Eli Lilly and Company | |
| 21.15.GPC3-CAR T Cells | Baylor College of Medicine |
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 |
|---|---|---|---|---|---|
| NCT03793166 Immunotherapy With Nivolumab and Ipilimumab Followed by Nivolumab or Nivolumab With Cabozantinib for Patients With Advanced Kidney Cancer, The PDIGREE Study 2 countries, 100% of sites in the US Decision brief → | Ph3 | National Cancer Institute (NCI) | active not recruiting | 2019-01-04 | 2028-03-01 |
| NCT00945009 Combination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor 6 countries, 86% of sites in the US Decision brief → | Ph3 | Children's Oncology Group | completed | 2009-07-23 | 2016-12-31 |
| NCT07447076 Study of Novel Therapies for Young People With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT) Single-country: United States (4 sites) Decision brief → | Ph2 | Sabine Mueller, MD, PhD | recruiting | 2026-03-03 | 2035-12-31 |
| NCT06625190 Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors Single-country: United States (1 site) Decision brief → | Ph1/Ph2 | University of Florida | recruiting | 2024-10-03 | 2030-02 |
| NCT06622941 Study to ONO-4538 in Patients With Rhabdoid Tumor Single-country: Japan (14 sites) Decision brief → | Ph2 | Ono Pharmaceutical Co., Ltd. | recruiting | 2024-10-02 | 2029-01-31 |
| NCT06465199 Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas Single-country: United States (14 sites) Decision brief → | Ph1/Ph2 | Milton S. Hershey Medical Center | recruiting | 2024-06-18 | 2033-05 |
| NCT05985161 A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors Single-country: United States (16 sites) Decision brief → | Ph2 | Memorial Sloan Kettering Cancer Center | recruiting | 2023-08-14 | 2029-08-01 |
| NCT05407441 Tazemetostat+Nivo/Ipi in INI1-Neg/SMARCA4-Def Tumors Single-country: United States (3 sites) Decision brief → | Ph1/Ph2 | Susan Chi, MD | active not recruiting | 2022-06-07 | 2027-02-01 |
| NCT05286801 Tiragolumab and Atezolizumab for the Treatment of Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors 3 countries, 88% of sites in the US Decision brief → | Ph1/Ph2 | National Cancer Institute (NCI) | active not recruiting | 2022-03-18 | 2030-03-31 |
| NCT05135975 A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors Single-country: United States (6 sites) Decision brief → | Ph2 | Nationwide Children's Hospital | recruiting | 2021-11-26 | 2026-07 |
| NCT04901702 Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma 2 countries, 70% of sites in the US Decision brief → | Ph1/Ph2 | St. Jude Children's Research Hospital | recruiting | 2021-05-25 | 2026-12-31 |
| NCT04416568 Study of Nivolumab and Ipilimumab in Children and Young Adults With INI1-Negative Cancers Single-country: United States (8 sites) Decision brief → | Ph2 | Dana-Farber Cancer Institute | active not recruiting | 2020-06-04 | 2025-07-17 |
| 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 |
| 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 |
| 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 |
| 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 |
| SJATRT · NCT02114229 Phase 2 Study of Alisertib Therapy for Rhabdoid Tumors Single-country: United States (9 sites) Decision brief → | Ph2 | St. Jude Children's Research Hospital | active not recruiting | 2014-04-15 | 2025-09-30 |
| 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 |
| 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 |
| NCT07513194 GPC3 CAR T Cells With IL-15 and IL-21 for Recurrent ATRT and CNS Rhabdoid Tumors (RADIANT) Single-country: United States (1 site) Decision brief → | Ph1 | Baylor College of Medicine | not yet recruiting | 2026-04-07 | 2029-10-31 |
| 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 |
| GABRIEL · NCT07331064 Gallium Maltolate for the Treatment of Pediatric Patients With Relapsed or Refractory Pediatric High-Grade Glioma and Atypical Teratoid Rhabdoid Tumor No sites listed Decision brief → | Ph1 | Sarah Rumler | not yet recruiting | 2026-01-09 | 2030-11-15 |
| IMPACT · NCT07148050 Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells Single-country: United States (1 site) Decision brief → | Ph1 | Seattle Children's Hospital | recruiting | 2025-08-29 | 2029-04-22 |
| 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 |
| NCT06198296 Immunotherapy For Adults With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells Single-country: United States (1 site) Decision brief → | Ph1 | Baylor College of Medicine | recruiting | 2024-01-10 | 2028-02-01 |
| 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 |
| NCT05103631 Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in Autologous T Cells for Solid Tumors Single-country: United States (1 site) Decision brief → | Ph1 | Baylor College of Medicine | active not recruiting | 2021-11-02 | 2025-01-13 |
| NCT04897321 B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR) Single-country: United States (1 site) Decision brief → | Ph1 | St. Jude Children's Research Hospital | recruiting | 2021-05-21 | 2027-03-01 |
| NCT04715191 Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors Single-country: United States (1 site) Decision brief → | Ph1 | Baylor College of Medicine | recruiting | 2021-01-20 | 2027-08-01 |
| 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 |
| NCT04483778 B7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults Single-country: United States (1 site) Decision brief → | Ph1 | Seattle Children's Hospital | active not recruiting | 2020-07-23 | 2040-12 |
| NCT04377932 Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors Single-country: United States (1 site) Decision brief → | Ph1 | Baylor College of Medicine | active not recruiting | 2020-05-07 | 2024-08-17 |
| 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 |
| NCT04099797 C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B) Single-country: United States (1 site) Decision brief → | Ph1 | Baylor College of Medicine | recruiting | 2019-09-23 | 2027-02 |
| 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 |
| NCT03618381 EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults Single-country: United States (1 site) Decision brief → | Ph1 | Seattle Children's Hospital | active not recruiting | 2018-08-07 | 2040-06 |
| 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 |
| NCT01625351 A Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas Single-country: United States (1 site) Decision brief → | Ph1 | St. Jude Children's Research Hospital | completed | 2012-06-21 | 2020-02-10 |