ORPHA:251636
Ependymoma
Also known as: Classic ependymoma
Clinical definition (Orphanet)
Ependymoma is the most frequent intramedullary tumor in adults (but accounts for only 10-12% of pediatric central nervous system tumors), and can be benign or anaplastic. Ependymoma arise from the ependymal cells of the cerebral ventricles, corticle rests and central canal of the spinal cord, and manifest with variable symptoms such headache, vomiting, , focal neurological signs and loss of vision and can cause obstructive hydrocephalus in some cases.
How rare: 1-9 / 1 000 000 — roughly one to nine people per million. In a city the size of Kolkata, perhaps a few dozen.
Is anyone studying this?
20,137
20,137 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=183) is 38.
20,137 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 38 (publications denominator n=183).
10,921 in the last 10 years · medium confidence · 99.2th percentile (publications denominator)
Is a treatment being tested?
149
trials for this specific condition
149 interventional trials matched this specific condition name; 25 currently recruiting in our sample.
Data as of 26 July 2026
149 interventional trials — more than 59.2% of diseases in the trials denominator have none at all (151 of 255; this disease is at the 97.8th percentile).
medium confidence · 97.8th percentile (trials denominator)
Do we know what causes it?
Not yet — the cause hasn't been pinned down in GenCC.
No strong gene–disease assertion joined for this Orphanet entity.
Who's working on it?
1,486
Distinct author names in 200 sampled papers — named people below.
Who's working on it?
People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.
- 01Ichimura K6 papers · 2026
Department of Pathology, Faculty of Medicine, Kyorin University, Tokyo, Japan.
Papers in Europe PMC - 02Liu Y6 papers · 2026
Department of Radiology, Beijing Tiantan Hospital, Capital Medical University, Beijing 100070, China.
Papers in Europe PMC - 03Wang Y6 papers · 2026
Department of Radiology, The First Affiliated Hospital of Xinjiang Medical University, Urumqi, Xinjiang Uygur Autonomous Region, China. doctorwang1005@163.com.
Papers in Europe PMC - 04Hirato J5 papers · 2026
Department of Pathology, Public Tomioka General Hospital, Tomioka, Japan.
Papers in Europe PMC - 05Merchant TE5 papers · 2026
Chair, Department of Radiation Oncology, St. Jude Children's Research Hospital, Memphis, TN, United States. Electronic address: thomas.merchant@stjude.org.
Papers in Europe PMC - 06Tabori U5 papers · 2026
Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, 555 University Avenue, Toronto, ON M5G 1X8, Canada.
Papers in Europe PMC - 07Wang C5 papers · 2026
Department of Neurosurgery, Beijing Tiantan Hospital, Capital Medical University, Beijing, China; Department of Neurosurgery, Beijing Neurosurgical Institute, Capital Medical University, Beijing, China.
Papers in Europe PMC - 08Ellison DW4 papers · 2026
Department of Pathology, St. Jude Children's Research Hospital, Memphis, Tennessee.
Papers in Europe PMC - 09Mack SC4 papers · 2026
Department of Developmental Neurobiology, St. Jude Children's Research Hospital, Memphis, Tennessee.
Papers in Europe PMC - 10Pfister SM4 papers · 2026
Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Papers in Europe PMC
Recruiting interventional trials
Trials testing a treatment from the matched ClinicalTrials.gov set
149 interventional trials matched after quoted-phrase search and title/condition post-filter.
- NCT06161519·PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
- NCT05106296·Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer
- NCT01356290·Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors
- 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).
- NCT04541082·Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms
- NCT04732065·ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors
- NCT07017816·A Phase 0/1 Study of cDNA for TP53, Checkpoint Inhibition and Radiation in Children With Recurrent, Progressive or Refractory CNS Malignancies.
- NCT06193759·Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)
- NCT07226453·Target Validation and Efficacy of Metformin in Patients With Posterior Fossa Group A (PFA) Ependymoma
- NCT07707947·Selumetinib for NF2-Related Schwannomatosis
- NCT04374305·Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)
- NCT07664176·NgFUS NIVO: NeuroNavigation-Guided Focused Ultrasound With Nivolumab in Relapsed and Progressive DMG and Other High Grade Brain Tumors
- NCT06804655·Pharmacoscopy for Patients With Refractory Primary Brain Tumors
- NCT05278208·Lutathera for Treatment of Recurrent or Progressive High-Grade CNS Tumors
- NCT07424092·Intratumoral DNX-2401 for High Grade Pediatric Brain Tumors
Observational and natural-history studies
20 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.
Recruiting or not-yet-recruiting
- NCT05259605·Observational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Using cIMPACT-NOW and 2021 WHO Classification
- NCT07703605·AI-Assisted MRI Molecular Subtyping in Pediatric Brain Tumors
- NCT07589361·Safety and Efficacy of Vertebral Body-Sparing Craniospinal Irradiation With Proton Therapy in Pediatric Tumors
- NCT06323408·Integrated Analysis of Therapy Response and Resistence in Embryonal Tumors and Gliomas
- NCT05672043·Genetic and Molecular Risk Profiles of Pediatric Malignant Brain Tumors in China
- NCT06499636·In Vitro Models From Pediatric Brain Tumors
- NCT05151718·Multiomic Approach to Radioresistance of Ependymomas in Children and Adolescents
- NCT04648462·Proton Therapy Research Infrastructure- ProTRAIT- Neuro-oncology
- NCT06779487·Systemic Biomarkers to Predict Radiation-Induced Neurocognitive Decline
Where to find support
We do not yet link condition-specific patient organisations. These umbrella groups support undiagnosed and ultra-rare families:
India — NPRD
Last verified 2026-07-26This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.
Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.
How we counted this
Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored below but are not added to the query string.
"Ependymoma" OR "Classic ependymoma" OR "benign ependymoma"
MeSH descriptor terms unioned into the query: Ependymoma
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Ependymoma" OR "Classic ependymoma" OR "benign ependymoma"
Interventional trials matched via: both (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).
Study-type breakdown: 149 interventional · 20 observational · 0 expanded access. Only interventional studies enter the trial headline.
Cross-references (from Mondo): MESH:D004806 UMLS:C0014474 NCIT:C3017
Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh
Run this search on ClinicalTrials.gov
Confidence reasoning
- Preferred label is short or not clearly distinctive
- No synonyms dropped by stoplist
- No label/synonym collisions with other diseases in this corpus