Ependymoma
Recent clinical, regulatory, research and industry developments relating to this disease.
Adolescents and Young Adults With Cancer: CNS Tumors.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 2 clinical trials expected to report results, the earliest in Q2 2030.
- Q2 2030A Phase II Study of Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent/Progressive Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors
- Q3 2031Phase 1/2 Trial of PEP-CMV + Nivolumab for Newly Diagnosed Diffuse Midline Glioma/High-grade Glioma and Recurrent Diffuse Midline Glioma/High-grade Glioma, Medulloblastoma, and Ependymoma (PRiME II)
Clinical MilestonesViewHide
- 2026-07-01NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 MutationsResults posted
- 2026-04-30Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients With Newly Diagnosed Ependymoma Ages 1 to 21 YearsResults posted
- 2026-02-24A Phase II Trial of Surgery and Fractionated Re-Irradiation for Recurrent EpendymomaResults posted
- 2026-06-17Phase 1/2 Trial of PEP-CMV + Nivolumab for Newly Diagnosed Diffuse Midline Glioma/High-grade Glioma and Recurrent Diffuse Midline Glioma/High-grade Glioma, Medulloblastoma, and Ependymoma (PRiME II)Results expected Q3 2031
- 2026-02-23A Phase II Study of Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent/Progressive Medulloblastoma, Ependymoma, ATRT and Rare CNS TumorsResults expected Q2 2030
- 2026-07-01ClinicalNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 MutationsResults posted
- 2026-06-17ClinicalPhase 1/2 Trial of PEP-CMV + Nivolumab for Newly Diagnosed Diffuse Midline Glioma/High-grade Glioma and Recurrent Diffuse Midline Glioma/High-grade Glioma, Medulloblastoma, and Ependymoma (PRiME II)Results expected Q3 2031
- 2026-04-30ClinicalPhase III Randomized Trial of Post-Radiation Chemotherapy in Patients With Newly Diagnosed Ependymoma Ages 1 to 21 YearsResults posted
- 2026-03-31ClinicalPhase 2 Study of Intraventricular Omburtamab-based Radioimmunotherapy for Pediatric Patients With Recurrent Medulloblastoma and EpendymomaPrimary completion
- 2026-02-24ClinicalA Phase II Trial of Surgery and Fractionated Re-Irradiation for Recurrent EpendymomaResults posted
- 2026-02-23ClinicalA Phase II Study of Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent/Progressive Medulloblastoma, Ependymoma, ATRT and Rare CNS TumorsResults expected Q2 2030
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — Carmustine is indicated n adults in the following malignant neoplasms as a single ag… (2018)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes6
- Central Nervous System Neoplasms1
- Cerebellar Neoplasms1
- Ependymoma1
- Glioma1
- Medulloblastoma1
- Neoplasms1
Leading journals1
- Journal of clinical oncology : official journal of the American Society of Clinical Oncology1
Leading researchers3
- Gajjar A1
- Heidelberg RE1
- Wu J1
Affiliations (unnormalised)2
- Center for Cancer Research1
- St Jude Children's Research Hospital1
Reference
Authoritative identity, definition & identifiers.
Glioma derived from EPENDYMOGLIAL CELLS that tend to present as malignant intracranial tumors in children and as benign intraspinal neoplasms in adults. It may arise from any level of the ventricular system or central canal of the spinal cord. Intracranial ependymomas most frequently originate in the FOURTH VENTRICLE and histologically are densely cellular tumors which may contain ependymal tubules and perivascular pseudorosettes. Spinal ependymomas are usually benign papillary or myxopapillary tumors. (From DeVita et al., Principles and Practice of Oncology, 5th ed, p2018; Escourolle et al., Manual of Basic Neuropathology, 2nd ed, pp28-9)
- 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.