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Drugs in development · updated 30 October 2026

Ependymoma: drugs in development

Rare diseasePrevalence 1-9 / 1 000 000 according to Orphanet · ORPHA:251636

As of 30 October 2026 there are 37 active clinical trials (recruiting or ongoing, phases I–III) on 56 drugs for ependymoma. 18 are experimental, that is not yet approved in the United States.

37Active trials
18Experimental 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
5-ALAII1NCT06678867
^1^1C-methionineII1NCT02125786
AntiemeticsII1NCT04743661
AntipyreticII1NCT04743661
CobolimabTSR-022I–II1NCT06521567
DNX-2401II1NCT07424092
LuvometinibFCN-159II1NCT07713745
Omburtamab I-131II1NCT04743661
PLX038I–II1NCT06161519
SamotolisibLY3023414II1NCT03155620
SerplulimabHLX10, Hetronifly®II1NCT07713745
ONC206I2NCT04541082
G207I1NCT03911388
IL13Ralpha2-specificI1NCT04661384
Iopofosine I 131CLR 131I1NCT03478462

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, CytoxanII6NCT01356290
EtoposideAvopef®, Etopophos Preservative Free®I–II5NCT06485908
TemozolomideTemodal, TemodarII4NCT01356290
Bevacizumabavastin, MVASIII3NCT04374305
SelumetinibAZD6244, Koselugo®II3NCT07707947
CelecoxibCelebrex Capsule, Elyxyb®II2NCT01356290
EnsartinibX-396, EnsatinibII2NCT03213652
ErdafitinibBalversa®II2NCT03210714
IrinotecanOnivyde, CPT-11II2NCT01356290
IvosidenibAG-120, Tibsovo®II2NCT03155620
LarotrectinibBAY2757556, Vitrakvi®II2NCT03155620
LutatheraLutetium Lu 177 Dotatate, 177Lu-DOTATATEI–II2NCT05278208
OlaparibAZD2281, Lynparza®I–II2NCT06607692
SelpercatinibLOXO-292, Retevmo®II2NCT04320888
18F-FDG^1^8F-fluorodeoxyglucose, Fludeoxyglucose F-18II1NCT02125786

Sorted by most advanced phase and number of active trials. Showing 15 of 38 drugs.

All 56 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: D004806 “Ependymoma”. Prevalence: Orphanet.

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