Aggressive Systemic Mastocytosis (DBCOND0062505)

Identifiers

Synonyms

Aggressive Systemic Mastocytosis (ASM) / Aggressive systemic mastocytosis / Advanced systemic mastocytosis / Aggressive systemic mastocytosis (disorder) / Aggressive systemic mastocytosis (morphologic abnormality) / Aggressive lymphadenopathic mastocytosis with eosinophilia (disorder)

Associated Data

Indicated Drugs and Targets

Drug	Description	Targets
Avapritinib	A selective tyrosine kinase inhibitor being investigated for the treatment of multidrug resistant gastrointestinal tumors.	Mast/stem cell growth factor receptor Kittarget ATP-dependent translocase ABCB1transporter Broad substrate specificity ATP-binding cassette transporter ABCG2transporter Cytochrome P450 3A4enzyme Cytochrome P450 2C9enzyme Bile salt export pumptransporter Multidrug and toxin extrusion protein 1transporter Multidrug and toxin extrusion protein 2transporter Platelet-derived growth factor receptor alphatarget
Imatinib	A tyrosine kinase inhibitor used to treat a number of leukemias, myelodysplastic/myeloproliferative disease, systemic mastocytosis, hypereosinophilic syndrome, dermatofibrosarcoma protuberans, and gastrointestinal stromal tumors.	Platelet-derived growth factor receptor betatarget Tyrosine-protein kinase ABL1target Mast/stem cell growth factor receptor Kittarget Proto-oncogene tyrosine-protein kinase receptor Rettarget High affinity nerve growth factor receptortarget Macrophage colony-stimulating factor 1 receptortarget Platelet-derived growth factor receptor alphatarget Epithelial discoidin domain-containing receptor 1target Cytochrome P450 3A4enzyme Breakpoint cluster region proteintarget Cytochrome P450 1A2enzyme Cytochrome P450 2D6enzyme Cytochrome P450 2C9enzyme Cytochrome P450 2C19enzyme Cytochrome P450 3A5enzyme Cytochrome P450 3A7enzyme Solute carrier family 22 member 1transporter ATP-dependent translocase ABCB1transporter Solute carrier family 22 member 2transporter Broad substrate specificity ATP-binding cassette transporter ABCG2transporter Phospholipid-transporting ATPase ABCA3transporter Albumincarrier Alpha-1-acid glycoprotein 1carrier Bile salt export pumptransporter Cytochrome P450 2C8enzyme ATP-binding cassette sub-family C member 2transporter Multidrug resistance-associated protein 1transporter Organic cation/carnitine transporter 2transporter Solute carrier organic anion transporter family member 1B3transporter ATP-binding cassette sub-family C member 4transporter Solute carrier organic anion transporter family member 1A2transporter Discoidin domain-containing receptor 2target Induced myeloid leukemia cell differentiation protein Mcl-1target
Midostaurin	An antineoplastic agent used to treat high-risk acute myeloid leukemia (AML) with specific mutations, aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell leukemia (MCL).	Protein kinase C alpha typetarget Vascular endothelial growth factor receptor 2target Mast/stem cell growth factor receptor Kittarget Platelet-derived growth factor receptor alphatarget Platelet-derived growth factor receptor betatarget Receptor-type tyrosine-protein kinase FLT3target Cytochrome P450 3A Subfamilyenzyme Cytochrome P450 1A2enzyme Cytochrome P450 2B6enzyme Cytochrome P450 2C8enzyme Cytochrome P450 2C9enzyme Cytochrome P450 2C19enzyme Cytochrome P450 2D6enzyme Cytochrome P450 2E1enzyme Cytochrome P450 3A4enzyme Cytochrome P450 3A5enzyme Cytochrome P450 3A7enzyme Cytochrome P450 3A43enzyme Protein kinase C gamma typetarget

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT05362773	A Study of MGD024 in Patients With Relapsed or Refractory Hematologic Malignancies	No drug interventions	treatment	1	recruiting
NCT02561988	(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies	Avapritinib	treatment	1	completed
NCT05219266	Managed Access Programs for PKC412, Midostaurin	Midostaurin	Not Available	Not Available	no_longer_available
NCT04695431	Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSM	No drug interventions	Not Available	Not Available	completed
NCT01807598	Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia	Brentuximab vedotin	treatment	2	completed
NCT03580655	(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis	Avapritinib	treatment	2	active_not_recruiting
NCT02415608	Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis	Ibrutinib	treatment	2	terminated
NCT04996875	(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis	Bezuclastinib	treatment	2	recruiting
NCT00233454	Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia	Midostaurin	treatment	2	completed
NCT02380222	Patient-Reported Outcome Questionnaire for Systemic Mastocytosis	No drug interventions	Not Available	Not Available	completed