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Phuturdex › Diseases › Juvenile myelomonocytic leukemia

Drugs in development · updated 30 October 2026

Juvenile myelomonocytic leukemia: drugs in development

Rare diseasePrevalence 1-9 / 1 000 000 according to Orphanet · ORPHA:86834

As of 30 October 2026 there are 11 active clinical trials (recruiting or ongoing, phases I–III) on 28 drugs for juvenile myelomonocytic leukemia. 4 are experimental, that is not yet approved in the United States.

11Active trials
4Experimental 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
AlphaII1NCT05735717
CD19II1NCT03849651
CPX-351II1NCT05656248
PMD-026I1NCT07379125

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
FludarabineFludara, FLUDARABINI–II7NCT05849662
CyclophosphamideCyclophosphamid, CytoxanII5NCT03849651
MelphalanEvomela®, Hepzato®II4NCT03849651
AzacitidineAzacytidine, 5-azacytidineI–II2NCT05849662
BlinatumomabBlincyto®II2NCT03849651
BusulfanBusulphan, Busulfex®II2NCT00544115
CytarabineAra-C, Cytosine ArabinosideI–II2NCT05849662
MesnaUromitexan, Mesnex®II2NCT03849651
MethotrexateJylamvo®, Reditrex®II2NCT00544115
RituximabRuxience, RituxanII2NCT05735717
SirolimusRapamycin, Scomara®II2NCT00544115
TacrolimusEnvarsus XR, Prograf®II2NCT00544115
ThiotepaTepylute®, Tepadina And Sodium Chloride®II2NCT03849651
TrametinibMekinist®, Spexotras®I–II2NCT05849662
Anti-Thymocyte GlobulinII1NCT02790515

Sorted by most advanced phase and number of active trials. Showing 15 of 24 drugs.

All 28 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: D054429 “Leukemia, Myelomonocytic, Juvenile”. Prevalence: Orphanet.

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