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rhabdoid tumor - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

rhabdoid tumor

MONDO:0002728

Also known as:malignant rhabdoid tumormalignant rhabdoid tumourrhabdoid sarcoma

Find Clinical TrialsTrials

Overview

An aggressive malignant embryonal neoplasm usually occurring during childhood. It is characterized by the presence of large cells with abundant cytoplasm, large eccentric nucleus, and a prominent nucleolus and it is associated with abnormalities of chromosome 22. It can arise from the central nervous system, kidney, and the soft tissues. The prognosis is poor.

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials48 registered · 20 recruiting

Diagnosis

Biomarker and diagnostic research for rhabdoid tumor has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for rhabdoid tumor. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for rhabdoid tumor, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for rhabdoid tumor. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: Unknown (Unknown prevalence).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

48 clinical trials registered, 20 recruiting. Interventions under study include drug therapy, procedural interventions, biologic therapy, and other interventions. Pipeline includes 2 PHASE3, 17 PHASE2, 24 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT04715191](https://clinicaltrials.gov/study/NCT04715191)

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 3:00 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:69077

GARD

Genetic and Rare Diseases Info Center

GARD:7572

Questions for Your Care Team

→Are any of the 48 active clinical trials a good fit for my situation?
→What is the current development status of N-hydroxy-N-(methylacylfulvene)urea, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Research Type	Count	% of Total
Patient case studies	73	32%
Laboratory research	48	21%
Research summaries	37	16%
Disease patterns and progression	21	9%
New treatment approaches	21	9%
Clinical study results	16	7%
Testing and diagnosis research	11	5%
Other research	1	0%

Kisho

rhabdoid tumor

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Outcomes and Surgical Management of Malignant Rhabdoid Tumor of the Kidney: A Report From the Pediatric Surgical Oncology Research Collaborative.

Impact of Radiation Therapy on Malignant Rhabdoid Tumor of the Kidney: A National Cancer Database Analysis.

rhabdoid tumor

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Outcomes and Surgical Management of Malignant Rhabdoid Tumor of the Kidney: A Report From the Pediatric Surgical Oncology Research Collaborative.

Impact of Radiation Therapy on Malignant Rhabdoid Tumor of the Kidney: A National Cancer Database Analysis.