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Drugs in development · updated 30 October 2026

Wilms Tumor: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:654

As of 30 October 2026 there are 24 active clinical trials (recruiting or ongoing, phases I–III) on 37 drugs for Wilms Tumor. 6 are experimental, that is not yet approved in the United States.

24Active trials
6Experimental 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
SamotolisibLY3023414II1NCT03155620
TegavivintI–II1NCT04851119
Becotatug VedotinMRG003I1NCT07584499
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
IrinotecanOnivyde, CPT-11I–II3NCT04901702
CabozantinibResniben®, Cabometyx®II2NCT02867592
CyclophosphamideCyclophosphamid, CytoxanII2NCT04322318
EnsartinibX-396, EnsatinibII2NCT03213652
ErdafitinibBalversa®II2NCT03210714
EtoposideAvopef®, Etopophos Preservative Free®I–II2NCT05384821
IvosidenibAG-120, Tibsovo®II2NCT03155620
LarotrectinibBAY2757556, Vitrakvi®II2NCT03155620
SelpercatinibLOXO-292, Retevmo®II2NCT04320888
TemozolomideTemodal, TemodarI–II2NCT04901702
VincristineVincristin, Oncovin [vincristine]I–II2NCT05384821
AtezolizumabTecentriq, MPDL3280AI–II1NCT05765084
Cabozantinib S-malateCabometyx®, Cometriq®II1NCT02867592
CarboplatinCarboplatine, ParaplatinII1NCT04322318
DoxorubicinAdriamycin, DoxorubincinII1NCT04322318

Sorted by most advanced phase and number of active trials. Showing 15 of 31 drugs.

All 37 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: D009396 “Wilms Tumor”. Prevalence: Orphanet.

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