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Disease
Rhabdoid Tumor
Late-stage therapeutic developmentEmerging research
1
Publications
19
Clinical trials
2023
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Children's Oncology Group's 2023 blueprint for research: Renal tumors.
Research2023-07-21Pediatric blood & cancer
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 4 clinical trials expected to report results, the earliest in Q1 2029.
Upcoming Milestones
- Q1 2029A Multicenter, Open-label, Uncontrolled Phase II Study to Investigate Efficacy and Safety of ONO4538 in Patients With Rhabdoid Tumor
- Q1 2030A Phase 1/2 Study of Tiragolumab (NSC# 827799) and Atezolizumab (NSC# 783608) in Patients With Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
- Q2 2033A Phase I/II Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and Sarcomas
- Q4 2035A Platform Study of Novel Therapies for Children, Adolescents and Young Adults With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)
Major developments
Upcoming trial readoutHigh impact
Results expected Q2 20332026-07-01
Upcoming trial readoutHigh impact
Results expected Q4 20352026-06-23
Clinical MilestonesViewHide
Results expected
- 2026-07-01A Phase I/II Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and SarcomasResults expected Q2 2033
- 2026-06-23A Platform Study of Novel Therapies for Children, Adolescents and Young Adults With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)Results expected Q4 2035
- 2026-05-13A Phase 1/2 Study of Tiragolumab (NSC# 827799) and Atezolizumab (NSC# 783608) in Patients With Relapsed or Refractory SMARCB1 or SMARCA4 Deficient TumorsResults expected Q1 2030
- 2026-03-12A Multicenter, Open-label, Uncontrolled Phase II Study to Investigate Efficacy and Safety of ONO4538 in Patients With Rhabdoid TumorResults expected Q1 2029
Recently completed
Activity timeline6
- 2032-08-01ClinicaliSTAR: Phase 1b Trial of Idasanutlin and Selinexor Therapy For Children With Progressive/Relapsed Atypical Teratoid Rhabdoid Tumors, Extra-CNS Malignant Rhabdoid Tumors Or Synchronous/Metachronous Rhabdoid TumorsWithdrawn
- 2026-07-01ClinicalA Phase I/II Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and SarcomasResults expected Q2 2033
- 2026-06-23ClinicalA Platform Study of Novel Therapies for Children, Adolescents and Young Adults With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)Results expected Q4 2035
- 2026-05-13ClinicalA Phase 1/2 Study of Tiragolumab (NSC# 827799) and Atezolizumab (NSC# 783608) in Patients With Relapsed or Refractory SMARCB1 or SMARCA4 Deficient TumorsResults expected Q1 2030
- 2026-03-12ClinicalA Multicenter, Open-label, Uncontrolled Phase II Study to Investigate Efficacy and Safety of ONO4538 in Patients With Rhabdoid TumorResults expected Q1 2029
- 2025-09-01ClinicalPhase 2 Study of Alisertib as a Single Agent in Recurrent or Progressive Central Nervous System (CNS) Atypical Teratoid Rhabdoid Tumors (AT/RT) and Extra-CNS Malignant Rhabdoid Tumors (MRT) and in Combination Therapy in Newly Diagnosed AT/RTPrimary completion
Clinical trials
The current development programme across all trial phases.
Clinical programme
19
6
1
6
Late-stage studies
Recruiting
Recently completed
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Recent publications
Major themes5
- Carcinoma, Renal Cell1
- Kidney Neoplasms1
- Rhabdoid Tumor1
- Sarcoma, Clear Cell1
- Wilms Tumor1
Leading journals1
- Pediatric blood & cancer1
Leading researchers8
- Aldrink JH1
- Cost NG1
- Evageliou N1
- Geller JI1
- Hong AL1
- Mullen EA1
- Renfro LA1
- Treece AL1
Affiliations (unnormalised)6
- Aflac Cancer and Blood Disorders Center1
- Children's Hospital of Philadelphia1
- Cincinnati Children's Hospital Medical Center1
- Cook Children's Medical Center1
- Dana-Farber/Boston Children's Blood Disorders and Cancer Center1
- Department of Pathology and Laboratory Medicine1
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A rare but highly lethal childhood tumor found almost exclusively in infants. Histopathologically, it resembles RHABDOMYOSARCOMA but the tumor cells are not of myogenic origin. Although it arises primarily in the kidney, it may be found in other parts of the body. The rhabdoid cytomorphology is believed to be the expression of a very primitive malignant cell. (From Holland et al., Cancer Medicine, 3d ed, p2210)
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.