Disease · MONDO_0016586

systemic mastocytosis

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. systemic mastocytosis. Diseases, record MONDO_0016586. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0016586

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: systemic mastocytosis; Open Targets
Open Targets ID: MONDO_0016586; Open Targets
Other names: systemic tissue Mast cell disease; Open Targets
Disease Ontology: DOID:349; Disease Ontology
NCI Thesaurus: C9235; NCI Thesaurus
Orphanet: ORPHA:2467; Orphanet
Highest stage of its drugs: Approval, for 1 of its 24 drugs, from FDA and TTD; Open Targets

Drugs24

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
MIDOSTAURIN	CHEMBL608533	Small molecule	Approval	FDA and TTD	4	1	1	Drugs and targets
MASITINIB	CHEMBL1908391	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
ELENESTINIB	CHEMBL5314552	Small molecule	Phase 2/3	ClinicalTrials.gov	2	2	2	Drugs and targets
CLADRIBINE	CHEMBL1619	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
HYDROXYCHLOROQUINE	CHEMBL1535	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
INTERFERON ALFA-2A	CHEMBL2108508	Protein	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
AVAPRITINIB	CHEMBL4204794	Small molecule	Phase 2	ClinicalTrials.gov	5	4	4	Drugs and targets
BEZUCLASTINIB	CHEMBL5095229	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
IMATINIB	CHEMBL941	Small molecule	Phase 2	ClinicalTrials.gov	2	1	1	Drugs and targets
BRENTUXIMAB VEDOTIN	CHEMBL1742994	Antibody drug conjugate	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 24

See all 24 drugs

Targets74

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
KIT	Mast/stem cell growth factor receptor Kit	6	Stem cell growth factor receptor inhibitor	Approval	https://conductscience.org/library/genes/3815	Drugs and targets
PDGFRA	Platelet-derived growth factor receptor	4	Platelet-derived growth factor receptor alpha inhibitor; Platelet-derived growth factor receptor inhibitor	Approval	https://conductscience.org/library/genes/5156	Drugs and targets
PDGFRB	Platelet-derived growth factor receptor	4	Platelet-derived growth factor receptor beta inhibitor; Platelet-derived growth factor receptor inhibitor	Approval	https://conductscience.org/library/genes/5159	Drugs and targets
ABL1	Bcr/Abl fusion protein	3	Bcr/Abl fusion protein inhibitor; Tyrosine-protein kinase ABL inhibitor	Phase 2	https://conductscience.org/library/genes/25	Drugs and targets
BCR	Bcr/Abl fusion protein	3	Bcr/Abl fusion protein inhibitor	Phase 2	https://conductscience.org/library/genes/613	Drugs and targets
DNMT1	DNA (cytosine-5)-methyltransferase 1	2	DNA (cytosine-5)-methyltransferase 1 inhibitor	Phase 1	https://conductscience.org/library/genes/1786	Drugs and targets
DNMT3A	DNA (cytosine-5)-methyltransferase 3A	2	DNA (cytosine-5)-methyltransferase 3A inhibitor	Phase 1	https://conductscience.org/library/genes/1788	Drugs and targets
BCL2	Apoptosis regulator Bcl-2	1	Apoptosis regulator Bcl-2 inhibitor	Phase 2	https://conductscience.org/library/genes/596	Drugs and targets
BCL2A1	Bcl-2-related protein A1	1	Bcl-2-related protein A1 inhibitor	Phase 2	https://conductscience.org/library/genes/597	Drugs and targets
BCL2L1	Bcl-2-like protein 1	1	Apoptosis regulator Bcl-X inhibitor	Phase 2	https://conductscience.org/library/genes/598	Drugs and targets

1 to 10 of 74

See all 74 targets

Trials27

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00109707	A Study of Oral AMN107 in Adults With Chronic Myelogenous Leukemia (CML) or Other Hematologic Malignancies	Phase 1/2	507	2021-06-29	Chronic Myelogenous Leukemia, Acute Lymphoblastic Leukemia (Philadelphia Chromosome Positive), Hypereosinophilic Syndrome, Systemic Mastocytosis	NILOTINIB	Its record names the disease	ClinicalTrials.gov
NCT03770273	Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis	Phase 2	21	2024-12-17	Indolent Systemic Mastocytosis	SARILUMAB	Open Targets' reading of its record	ClinicalTrials.gov
NCT00449748	Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis	Phase 2	10	2011-04-29	Systemic Mastocytosis	EVEROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT01807598	Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia	Phase 2	10	2019-01-29	Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Systemic Mastocytosis	BRENTUXIMAB VEDOTIN	Its record names the disease	ClinicalTrials.gov
NCT02415608	Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis	Phase 2	4	2018-09-20	Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Systemic Mastocytosis	IBRUTINIB	Its record names the disease	ClinicalTrials.gov
NCT00918931	Obatoclax for Systemic Mastocytosis	Phase 2	3	2011-04-25	Leukemia, Systemic Mastocytosis	OBATOCLAX MESYLATE	Its record names the disease	ClinicalTrials.gov
NCT04910685	(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis	Phase 2/3	534		Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis	ELENESTINIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT03731260	(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis	Phase 2	251		Indolent Systemic Mastocytosis	AVAPRITINIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT05186753	(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis	Phase 2	237		SSM, Mastocytosis, Indolent, Mastocytosis, Systemic, Mastocytosis, ISM, BMM, Smoldering Systemic Mastocytosis, Bone Marrow Mastocytosis	BEZUCLASTINIB	Its record names the disease	ClinicalTrials.gov
NCT04333108	Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment	Phase 3	140		Indolent Systemic Mastocytosis	MASITINIB	Its record names the disease	ClinicalTrials.gov

1 to 10 of 27

See all 27 trials

Comparisons in its trials7

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
CML-CP With Prior Imatinib Only vs CML-AP With Prior Imatinib Only: Participants With (MMR) Major Molecular Response (Phase II)	106 of 299	18 of 122	421	NCT00109707	Trial comparisons
Drug: Sarilumab vs Placebo: deaths from any cause	0 of 8	0 of 8	16	NCT03770273	Trial comparisons
Drug: Sarilumab vs Placebo: serious adverse events	1 of 8	0 of 8	16	NCT03770273	Trial comparisons
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: Progression-free Survival (PFS)	0 of 3	0 of 1	4	NCT02415608	Trial comparisons
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: Overall Response Rate (ORR)	0 of 3	0 of 1	4	NCT02415608	Trial comparisons
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: deaths from any cause	1 of 3	0 of 1	4	NCT02415608	Trial comparisons
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: serious adverse events	1 of 3	0 of 1	4	NCT02415608	Trial comparisons

Papers from its trials22

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
28069279	RESULT	1	PubMed
23432617	RESULT	1	PubMed
21108325	RESULT	1	PubMed
41690487	DERIVED	1	PubMed
41604606	DERIVED	1	PubMed
41244098	DERIVED	1	PubMed
40529483	DERIVED	1	PubMed
38320129	DERIVED	1	PubMed
36048877	DERIVED	1	PubMed
35790816	DERIVED	2	PubMed

1 to 10 of 22

See all 22 papers from its trials

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0016586

GET https://conductscience.org/api/diseases/v1/MONDO_0016586/drugs?page=2

API reference For AI assistants