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Drugs in development · updated 30 October 2026

Medulloblastoma: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:616

As of 30 October 2026 there are 49 active clinical trials (recruiting or ongoing, phases I–III) on 77 drugs for medulloblastoma. 22 are experimental, that is not yet approved in the United States.

49Active trials
22Experimental drugs
0Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
IndoximodII2NCT04049669
TipifarnibII2NCT04284774
UlixertinibII2NCT03698994
[18F]DPA714II1NCT05128903
AntiemeticsII1NCT04743661
AntipyreticII1NCT04743661
ApatinibRivoceranibII1NCT04501718
Omburtamab I-131II1NCT04743661
PLX038I–II1NCT06161519
SamotolisibLY3023414II1NCT03155620
Becotatug VedotinMRG003I1NCT07584499
G207I1NCT03911388
GM-CSFGranulocyte-Macrophage Colony-Stimulating FactorI1NCT06514898
IL13Ralpha2-specificI1NCT04661384
Iopofosine I 131CLR 131I1NCT03478462
ONC206I1NCT04541082
Pp65I1NCT07492316
RVU120I1NCT07865130
SavolitinibAZD6094I1NCT03598244
VAL-413I1NCT04337177
CDK4Early I1NCT06959979
FimepinostatEarly I1NCT03893487

Sorted by most advanced phase and number of active trials.

Approved drugs in new trials

Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.

DrugMost advanced phaseActive trialsReference trial
CyclophosphamideCyclophosphamid, CytoxanIII12NCT05382338
CisplatinCis Platinum, CisplatineIII6NCT05382338
VincristineVincristin, Oncovin [vincristine]III5NCT05382338
LomustineCCNU, Gleostine®III3NCT05382338
MetforminRiomet Er®, Glumetza®III1NCT05230758
Sodium Thiosulfatetiosulfate, Pedmark®III1NCT05382338
VinblastineVinblastin, Velban®II–III1NCT02066220
EtoposideAvopef®, Etopophos Preservative Free®I–II9NCT06485908
TemozolomideTemodal, TemodarI–II9NCT07346157
Bevacizumabavastin, MVASII–II3NCT07346157
CelecoxibCelebrex Capsule, Elyxyb®II3NCT01356290
IrinotecanOnivyde, CPT-11I–II3NCT07346157
CarboplatinCarboplatine, ParaplatinII2NCT05535166
CytarabineAra-C, Cytosine ArabinosideII2NCT01356290
DexamethasoneDexametasone, DextenzaII2NCT07856069

Sorted by most advanced phase and number of active trials. Showing 15 of 55 drugs.

All 77 drugs, with the details

In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.

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Broader categories

Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D008527 “Medulloblastoma”. Prevalence: Orphanet.

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