Disease · MONDO_0020333
aggressive systemic mastocytosis
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. aggressive systemic mastocytosis. Diseases, record MONDO_0020333. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0020333
On this page
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.
Drugs1
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 |
|---|---|---|---|---|---|---|---|
| MIDOSTAURIN | CHEMBL608533 | Small molecule | Phase 2 | ClinicalTrials.gov | 3 | 3 | 3 |
Source Drugs and targets
Targets16
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 |
|---|---|---|---|---|---|---|
| FLT3 | Receptor-type tyrosine-protein kinase FLT3 | 1 | Tyrosine-protein kinase receptor FLT3 inhibitor | Phase 2 | https://conductscience.org/library/genes/2322 | Drugs and targets |
| KDR | Vascular endothelial growth factor receptor 2 | 1 | Vascular endothelial growth factor receptor 2 inhibitor | Phase 2 | https://conductscience.org/library/genes/3791 | Drugs and targets |
| KIT | Mast/stem cell growth factor receptor Kit | 1 | Stem cell growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/3815 | Drugs and targets |
| PDGFRA | Platelet-derived growth factor receptor | 1 | Platelet-derived growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/5156 | Drugs and targets |
| PDGFRB | Platelet-derived growth factor receptor | 1 | Platelet-derived growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/5159 | Drugs and targets |
| PRKCA | Protein kinase C (PKC) | 1 | Protein kinase C (PKC) inhibitor | Phase 2 | https://conductscience.org/library/genes/5578 | Drugs and targets |
| PRKCB | Protein kinase C (PKC) | 1 | Protein kinase C (PKC) inhibitor | Phase 2 | https://conductscience.org/library/genes/5579 | Drugs and targets |
| PRKCD | Protein kinase C (PKC) | 1 | Protein kinase C (PKC) inhibitor | Phase 2 | https://conductscience.org/library/genes/5580 | Drugs and targets |
| PRKCE | Protein kinase C (PKC) | 1 | Protein kinase C (PKC) inhibitor | Phase 2 | https://conductscience.org/library/genes/5581 | Drugs and targets |
| PRKCG | Protein kinase C (PKC) | 1 | Protein kinase C (PKC) inhibitor | Phase 2 | https://conductscience.org/library/genes/5582 | Drugs and targets |
1 to 10 of 16
See all 16 targetsTrials3
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 |
|---|---|---|---|---|---|---|---|---|
| NCT00782067 | Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia | Phase 2 | 116 | 2017-06-06 | Leukemia | MIDOSTAURIN | Its record names the disease | ClinicalTrials.gov |
| NCT00233454 | Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia | Phase 2 | 26 | 2015-07-09 | Systemic Mastocytosis, Aggressive (ASM), Leukemia, Mast Cell, Hematological Non-mast Cell Lineage Disease (AHNMD) | MIDOSTAURIN | Its record names the disease | ClinicalTrials.gov |
| NCT05219266 | Managed Access Programs for PKC412, Midostaurin | FMS-Like Tyrosine Kinase 3 (FLT3)-Mutated Acute Myeloid Leukemia, Acute Myeloid Leukemia, Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Systemic Mastocytosis With an Associated Hematologic Neoplasm | MIDOSTAURIN | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials0
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.
None in this release.
Papers from its trials4
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
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_0020333
GET https://conductscience.org/api/diseases/v1/MONDO_0020333/drugs