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Drugs in development · updated 30 October 2026

Hepatoblastoma: drugs in development

Rare diseasePrevalence <1 / 1 000 000 according to Orphanet · ORPHA:449

As of 30 October 2026 there are 22 active clinical trials (recruiting or ongoing, phases I–III) on 41 drugs for hepatoblastoma. 7 are experimental, that is not yet approved in the United States.

22Active trials
7Experimental 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
TipifarnibII2NCT04284774
UlixertinibII2NCT03698994
CobolimabTSR-022I–II1NCT06521567
SamotolisibLY3023414II1NCT03155620
TegavivintI–II1NCT04851119
CodrituzumabI1NCT04928677
VAL-413I1NCT04337177

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
DoxorubicinAdriamycin, DoxorubincinIII3NCT03533582
IrinotecanOnivyde, CPT-11III3NCT03533582
VincristineVincristin, Oncovin [vincristine]III3NCT03533582
CarboplatinCarboplatine, ParaplatinIII2NCT03533582
CisplatinCis Platinum, CisplatineIII2NCT03533582
EtoposideAvopef®, Etopophos Preservative Free®III2NCT03533582
Fluorouracil5-Fluorouracil, AdrucilIII2NCT03533582
GemcitabineGemcitabin, GemzarIII2NCT03533582
OxaliplatinOxaliplatine, OxaliplainIII2NCT03533582
SorafenibNexavar®, Sorafenib Accord®III2NCT03533582
Sodium Thiosulfatetiosulfate, Pedmark®III1NCT04478292
CyclophosphamideCyclophosphamid, CytoxanII2NCT01804634
EnsartinibX-396, EnsatinibII2NCT03213652
ErdafitinibBalversa®II2NCT03210714
FludarabineFludara, FLUDARABINII2NCT01804634

Sorted by most advanced phase and number of active trials. Showing 15 of 34 drugs.

All 41 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: Wikidata; NLM MeSH term: D018197 “Hepatoblastoma”. Prevalence: Orphanet.

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