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Drugs in development · updated 30 October 2026

Rhabdomyosarcoma: drugs in development

Rare diseasePrevalence 1-9 / 1 000 000 according to Orphanet · ORPHA:780

As of 30 October 2026 there are 47 active clinical trials (recruiting or ongoing, phases I–III) on 68 drugs for rhabdomyosarcoma. 18 are experimental, that is not yet approved in the United States.

47Active 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
AnlotinibAL3818, AnrotinibII2NCT07444918
PEEL-224I–II2NCT06721689
TipifarnibII2NCT04284774
UlixertinibII2NCT03698994
^1^1C-methionineII1NCT01871766
CobolimabTSR-022I–II1NCT06521567
IvocizumabII1NCT06683846
PucotenlimabHX008I–II1NCT06456892
SamotolisibLY3023414II1NCT03155620
SilmitasertibI–II1NCT06541262
177Lu-DOTATOCLutetium Lu 177-EdotreotideI1NCT06441331
Becotatug VedotinMRG003I1NCT07584499
Copper GluconateI1NCT05210374
EntinostatEnitinostatI1NCT07888114
Iopofosine I 131CLR 131I1NCT03478462
Irinotecan SucrosofateMM-398I1NCT02013336
MocetinostatI1NCT04299113
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
VincristineVincristin, Oncovin [vincristine]III14NCT02567435
CyclophosphamideCyclophosphamid, CytoxanIII13NCT02567435
IrinotecanOnivyde, CPT-11III9NCT02567435
DactinomycinActinomycin D, ActinomycinIII7NCT02567435
VinorelbineNavelbine®III7NCT02567435
TemsirolimusTorisel®III1NCT02567435
TemozolomideTemodal, TemodarI–II10NCT06721689
DoxorubicinAdriamycin, DoxorubincinI–II3NCT04625907
EtoposideAvopef®, Etopophos Preservative Free®II3NCT01871766
FilgrastimRecombinant Human Granulocyte Colony-Stimulating Factor, NeupogenI–II3NCT06709495
FludarabineFludara, FLUDARABINII3NCT01804634
IfosfamideI–II3NCT04625907
CabozantinibResniben®, Cabometyx®II2NCT02867592
DexrazoxaneTotect®, Zinecard®II2NCT01871766
EnsartinibX-396, EnsatinibII2NCT03213652

Sorted by most advanced phase and number of active trials. Showing 15 of 50 drugs.

All 68 drugs, with the details

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Specific forms

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: D012208 “Rhabdomyosarcoma”. Prevalence: Orphanet.

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