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ependymoma - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

ependymoma

MONDO:0016698

Also known as:benign ependymomaepithelial ependymomaWHO grade II ependymal neoplasm

Find Clinical TrialsTrials

Overview

A WHO grade II, slow growing tumor of children and young adults, usually located intraventricularly. It is the most common ependymal neoplasm. It often causes clinical symptoms by blocking cerebrospinal fluid pathways. Key histological features include perivascular pseudorosettes and ependymal rosettes. (WHO)

Treatments1 FDA approved

Clinical Trials59 registered · 21 recruiting

Diagnosis

Biomarker and diagnostic research for ependymoma 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.

1 FDA-approved treatment is available for ependymoma, including CARMUSTINE (CARMUSTINE, approved 1977).

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

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.

59 clinical trials registered, 21 recruiting. Interventions under study include drug therapy, procedural interventions, other interventions, and biologic therapy. Pipeline includes 1 PHASE3, 19 PHASE2, 27 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT05278208](https://clinicaltrials.gov/study/NCT05278208)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 9:00 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:251636

GARD

Genetic and Rare Diseases Info Center

GARD:6353

Questions for Your Care Team

→What are the benefits and potential side effects of CARMUSTINE for managing ependymoma?
→Are any of the 59 active clinical trials a good fit for my situation?
→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
Research summaries	38	37%
Laboratory research	25	25%
Patient case studies	10	10%
Testing and diagnosis research	8	8%
Clinical study results	8	8%
Disease patterns and progression	8	8%
New treatment approaches	4	4%
Other research	1	1%

Kisho

ependymoma

Overview

Diagnosis

Treatment

Approved Therapies

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

Prognostic and Neurological Outcomes Following Surgery for Spinal NF2-Associated Ependymomas With Cysts.

ependymoma

Overview

Diagnosis

Treatment

Approved Therapies

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

Prognostic and Neurological Outcomes Following Surgery for Spinal NF2-Associated Ependymomas With Cysts.