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Phuturdex › Diseases › Familial ependymoma

Drugs in development · updated 30 October 2026

Familial ependymoma: drugs in development

As of 30 October 2026 there are 3 active clinical trials (recruiting or ongoing, phases I–III) on 4 drugs for familial ependymoma. 0 are experimental, that is not yet approved in the United States.

3Active trials
0Experimental 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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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
CisplatinCis Platinum, CisplatineII–III1NCT02265770
Valproic AcidValproate, Depacon®II–III1NCT02265770
ErdafitinibBalversa®II1NCT03210714
PembrolizumabKEYTRUDA, MK-3475I1NCT02359565

Sorted by most advanced phase and number of active trials.

All 4 drugs, with the details

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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: NLM MeSH; NLM MeSH term: C531673 “Familial ependymoma”.

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