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Phuturdex › Diseases › Ganglioneuroblastoma

Drugs in development · updated 30 October 2026

Ganglioneuroblastoma: drugs in development

Rare diseasePrevalence <1 / 1 000 000 according to Orphanet · ORPHA:251877

As of 30 October 2026 there are 7 active clinical trials (recruiting or ongoing, phases I–III) on 20 drugs for ganglioneuroblastoma. 0 are experimental, that is not yet approved in the United States.

7Active 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
CyclophosphamideCyclophosphamid, CytoxanIII5NCT06172296
DinutuximabDinituximab, Ch14.18III4NCT06172296
CarboplatinCarboplatine, ParaplatinIII3NCT06172296
DoxorubicinAdriamycin, DoxorubincinIII3NCT06172296
EtoposideAvopef®, Etopophos Preservative Free®III3NCT06172296
IsotretinoinAbsorica Ld®, Absorica®III3NCT06172296
SargramostimrhuGM-CSF, leukineIII3NCT03126916
TopotecanHycamtin®, Topotecan Hospira®III3NCT06172296
CisplatinCis Platinum, CisplatineIII2NCT06172296
IrinotecanOnivyde, CPT-11III2NCT06172296
MelphalanEvomela®, Hepzato®III2NCT06172296
TemozolomideTemodal, TemodarIII2NCT06172296
ThiotepaTepylute®, Tepadina And Sodium Chloride®III2NCT06172296
VincristineVincristin, Oncovin [vincristine]III2NCT06172296
BusulfanBusulphan, Busulfex®III1NCT03126916

Sorted by most advanced phase and number of active trials. Showing 15 of 20 drugs.

All 20 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: D018305 “Ganglioneuroblastoma”. Prevalence: Orphanet.

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