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Drugs in development · updated 30 October 2026

Mast cell leukemia: drugs in development

Rare diseasePrevalence <1 / 1 000 000 according to Orphanet · ORPHA:98851

As of 30 October 2026 there are 3 active clinical trials (recruiting or ongoing, phases I–III) on 7 drugs for mast cell leukemia. One is experimental, that is not yet approved in the United States.

3Active trials
1Experimental 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
Bezuclastinib TabletsCGT9486II1NCT04996875

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
CyclophosphamideCyclophosphamid, CytoxanII2NCT03779854
FludarabineFludara, FLUDARABINII2NCT03779854
BusulfanBusulphan, Busulfex®II1NCT03779854
MethotrexateJylamvo®, Reditrex®II1NCT03779854
TacrolimusEnvarsus XR, Prograf®II1NCT03779854
ThiotepaTepylute®, Tepadina And Sodium Chloride®II1NCT03779854

Sorted by most advanced phase and number of active trials.

All 7 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: D007946 “Leukemia, Mast-Cell”. Prevalence: Orphanet.

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