29 Medulloblastoma Clinical Trials Recruiting Now (May 2026): Pediatric Brain Tumor, Molecular Subgroup SHH Group 3 MYC, CAR-T B7-H3 GPC2, MEMMAT
Last updated: May 28, 2026
Current Clinical Trial Landscape
About medulloblastoma:
Medulloblastoma is the most common malignant brain tumor in children. It arises in the cerebellum and can spread through cerebrospinal fluid. Molecular subgroups (WNT, SHH, Group 3, Group 4) guide treatment and prognosis.
Active research areas in 2026:
- CAR-T cell therapy (B7-H3, GPC2, GD2 targets)
- Oncolytic virus therapy (HSV G207)
- PRRT (Lutathera 177Lu-DOTATATE ± olaparib) for somatostatin-receptor-positive tumors
- MYC/MYCN-targeted approaches for Group 3 medulloblastoma (PLX038)
- Reduced-intensity radiation strategies (focal RT in WNT, FOR-WNT2)
- Maintenance therapy (DFMO; RNA-LP vaccines PNOC020 rMB)
- Cognitive recovery / supportive care (sodium thiosulfate otoprotection; metformin for cognition)
Standard of care: Surgery + craniospinal radiation + chemotherapy (cisplatin, vincristine, cyclophosphamide). Reduced radiation for younger children and low-risk groups.
Recruiting Trials by Treatment Setting
Newly Diagnosed
For patients at initial diagnosis:
- NCT05382338 - Sodium thiosulfate to reduce hearing loss (otoprotection, Children's Oncology Group, Phase 3)
- NCT04474964 - Focal radiotherapy + low dose craniospinal irradiation in WNT-activated medulloblastoma (FOR-WNT2)
- NCT05535166 - Molecular and clinical risk-directed therapy for infants and young children (St. Jude, Phase 2)
Relapsed/Refractory
After progression on initial therapy:
- NCT05057702 - Individualized treatment plan in relapsed medulloblastoma/ependymoma (PNOC027)
- NCT04501718 - Apatinib + temozolomide + etoposide (Phase 2)
- NCT01356290 - Antiangiogenic therapy for recurrent medulloblastoma/ependymoma/ATRT (MEMMAT, Phase 2)
- NCT06701812 - Digoxin for relapsed Non-WNT / Non-SHH medulloblastoma (Moffitt, Phase 2)
Cognitive Recovery (Supportive Care)
For brain-tumor survivors recovering cognitive function:
- NCT05230758 - Metformin for cognitive recovery and white-matter growth in children/adolescents treated for brain tumor (Met Med Can, Phase 3)
Trials by Treatment Approach
CAR-T Cell Therapy
Engineered T cells targeting tumor antigens:
Immunotherapy
- NCT05096481 - PEP-CMV vaccine for newly diagnosed
- NCT06193759 - Adoptive cellular therapy for malignant pediatric brain tumors (IMPACT, Phase 1)
- NCT05106296 - Ibrutinib + indoximod chemo-immunotherapy
Oncolytic Virus Therapy
PRRT (Peptide Receptor Radionuclide Therapy)
- NCT05278208 - Lutathera (177Lu-DOTATATE) for recurrent/progressive high-grade CNS tumors with DOTATATE-PET uptake (CONNECT2007, Phase 1/2)
- NCT06607692 - 177Lu-DOTATATE + olaparib (PARP inhibitor) in pediatric DOTATATE-positive tumors (LUPARPED, Phase 1/2)
Group 3 / MYC-Amplified Targeted
Targeting MYC/MYCN amplification, common in Group 3 medulloblastoma:
- NCT06161519 - PLX038 (PEGylated SN-38 / topoisomerase-I inhibitor) in primary CNS tumors with MYC or MYCN amplifications (NCI, Phase 1/2)
Maintenance Therapy
- NCT04696029 - DFMO maintenance for molecular high/very high risk and relapsed medulloblastoma (Phase 2)
- NCT07492316 - RNA-lipid particle (RNA-LP) vaccines for recurrent/progressive medulloblastoma (PNOC020 rMB, Phase 1)
View all 29 medulloblastoma trials on ClinicalTrials.gov →
Frequently Asked Questions
How do I find medulloblastoma clinical trials?
Paste a medical summary into ClinTrialFinder to get AI-matched medulloblastoma trials in minutes. The tool considers molecular subgroup, age, and treatment history for both pediatric and adult patients.
What medulloblastoma trials are currently recruiting?
There are 29 recruiting interventional trials for medulloblastoma including targeted therapies, immunotherapy, and novel agents for pediatric and adult patients across all molecular subgroups.
Find Medulloblastoma Trials Matched to Your Situation
Use ClinTrialFinder's AI-powered matching to find trials based on your specific molecular subgroup and treatment history.
Find Matching Trials
