Medulloblastoma Clinical Trials (2026): 27 Recruiting Interventional Studies

Last updated: March 10, 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) for somatostatin receptor-positive tumors
Reduced-intensity radiation strategies
Maintenance therapy (DFMO)

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, Phase 3)
NCT04474964 - Focal radiotherapy + low dose craniospinal irradiation
NCT05535166 - Risk-directed therapy for infants and young children

Relapsed/Refractory

After progression on initial therapy:

NCT05057702 - Individualized treatment plan for relapsed medulloblastoma
NCT04501718 - Apatinib + temozolomide + etoposide
NCT01356290 - Antiangiogenic therapy for recurrent medulloblastoma

Trials by Treatment Approach

CAR-T Cell Therapy

Engineered T cells targeting tumor antigens:

NCT05835687 - B7-H3 CAR-T (locoregional delivery)
NCT07087002 - GPC2 CAR-T for relapsed/refractory
NCT05298995 - GD2 CAR-T for pediatric brain tumors
NCT04185038 - B7-H3 CAR-T locoregional immunotherapy

Immunotherapy

NCT05096481 - PEP-CMV vaccine for newly diagnosed
NCT06193759 - Adoptive cellular therapy
NCT05106296 - Ibrutinib + indoximod chemo-immunotherapy

Oncolytic Virus Therapy

NCT03911388 - HSV G207 for recurrent cerebellar tumors

PRRT (Peptide Receptor Radionuclide Therapy)

NCT05278208 - Lutathera for recurrent high-grade CNS tumors
NCT06607692 - 177Lu-DOTATATE + standard therapy

Maintenance Therapy

NCT04696029 - DFMO maintenance for high/very high risk

View all 27 medulloblastoma trials on ClinicalTrials.gov →

Trial listings from ClinicalTrials.gov. Page summaries generated by AI and may contain errors. Always verify with your healthcare provider.

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