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Home › Conditions › Medulloblastoma

Medulloblastoma

Part of Glioma

4 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Medulloblastoma

18 trials tracked for Medulloblastoma: 2 in phase 3 or 4 and 1 with published results. The most-cited published study has 204 citations.

18Trials tracked
2Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 3 2 Phase 2 11 Phase 1 2 Other / NA 3
  1. Phase 3 Comparison of Radiation Therapy Regimens in Combination With Chemotherapy in Treating Young Patients With Newly Diagnosed Standard-Risk Medulloblastoma Completed · 204 cited
  2. Phase 3 Combination Chemotherapy Followed By Peripheral Stem Cell Transplant in Treating Young Patients With Newly Diagnosed Supratentorial Primitive Neuroectodermal Tumors or High-Risk Medulloblastoma Completed
  3. Phase 2 A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB Completed
  4. Phase 2 Vaccine Immunotherapy for Recurrent Medulloblastoma and Primitive Neuroectodermal Tumor Completed
  5. Phase 2 A Phase II Study of Oral LDE225 in Patients With Hedge-Hog (Hh)-Pathway Activated Relapsed Medulloblastoma (MB) Completed
  6. Phase 2 Combination of Irinotecan and Temozolomide in Children With Brain Tumors. Completed
Show 12 more trials
  1. Phase 2 Dosimetry-Guided, Peptide Receptor Radiotherapy (PRRT) With 90Y-DOTA- tyr3-Octreotide (90Y-DOTATOC) Completed
  2. Phase 2 Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma Completed
  3. Phase 2 A Study of Pemetrexed in Children With Recurrent Cancer Completed
  4. Phase 2 Impact of Ga-68 DOTATOC PET-CT Imaging in Management of Neuroendocrine Tumors Completed
  5. Phase 2 Safety of 68Ga-DOTA-tyr3-Octreotide PET in Diagnosis of Solid Tumors Completed
  6. Phase 2 A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors Completed
  7. Phase 2 Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma Completed
  8. Phase 1 TPI 287 in Patients With Refractory or Recurrent Neuroblastoma or Medulloblastoma Completed
  9. Phase 1 Phase 1b Study PVSRIPO for Recurrent Malignant Glioma in Children Completed
  10. N/A Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer Completed
  11. N/A Molecular-Guided Therapy for Childhood Cancer Completed
  12. N/A Proton Beam Radiotherapy for Medulloblastoma and Pineoblastoma Completed

Showing the 18 most-cited and recently-updated of 18 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Medulloblastoma: what the trials found

Cisplatin administration in medulloblastoma patients is associated with specific post-treatment outcomes, including Grade 3+ hearing loss and neurocognitive function measured by Estimated Full-scale IQ (FSIQ) at both 4-15 months and 27-48 months post-diagnosis 1.

Carboplatin has been evaluated in Phase 3 trials; however, results showed no statistically significant differences in complete response rates (p=0.35), event-free survival (p=0.28), or patterns of failure (p=1.00) 2. In a separate Phase 2 trial, Carboplatin demonstrated response rates of 92.0% at 189 days and 88.0% at 273 days 9.

LDE225 showed varying objective response rates (ORR) in Phase 2 trials, with results ranging from 3.3% to 37.5% depending on the specific cohort; notably, a pediatric ORR of 66.7% was observed in patients with Hedgehog (Hh) signaling pathway status 11.

Imaging modalities including 68Ga-DOTATOC PET/CT have been established in Phase 2 trials for this condition [6, 7].

Recent results — preliminary, needs further review

  • Proton beam radiation was evaluated for neurocognitive effects, showing a statistically significant difference (p=0.01024) between groups; acute toxicity rates were reported at 90% for Grade 1 and 84% for Grade 2 16.

For the clinician treating this condition

  • Cisplatin treatment is associated with measurable impacts on hearing and neurocognitive function (FSIQ) in the months following diagnosis 1.
  • Carboplatin shows high response rates at 189 and 273 days in some cohorts, though larger Phase 3 trials did not show statistically significant differences in EFS or CR rates [2, 9].
  • LDE225 efficacy appears to correlate with Hedgehog (Hh) signaling pathway status in pediatric patients 11.

AI synthesis of 5 cited trials, updated Jun 27, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
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