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Midostaurin

FDA-approved product (capsule, oral), marketed as Midostaurin, Rydapt.

Registered studies
50+
Lab records
0
Pathogens at ≥40%
2 of 4

Chemical structure

Chemical structure of Midostaurin
A drawing shows what the molecule is, not what it does.

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Acute myeloid leukemia
  • Mast-cell leukemia
  • Mastocytosis
  • Neoplasm
  • Systemic mastocytosis

Indications ChEMBL records as approved, from FDA and DailyMed labels. Label ↗

WHO therapeutic group
Other protein kinase inhibitors
FDA pharmacologic class
Kinase Inhibitor FDA label ↗
Anti-infective classification
Not classified as an anti-infective.

Repurposing investigation

This medicine is being investigated here for AI-predicted antimicrobial activity against MRSA and M. tuberculosis. It was surfaced computationally for further investigation; it is not an established treatment for these infections.

AI-predicted activity

AI prediction · nothing measured
  • MRSA50.9% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • E. coli35.5% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae28.8% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis55.2% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.

A model’s estimate of laboratory activity against each pathogen species. It is not clinical effectiveness.

Evidence

Clinical and experimental

Documented evidence

Clinical

50+registered studies name this medicine, for any condition. The registry search keeps the first 50, so there may be more. See them below.

Experimental

No evidence found

No laboratory measurement against the four pathogens in the ChEMBL records loaded here.

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-9.3 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-6.1 kcal/mol

0better fit →-12

Enoyl-ACP reductase (InhA) ↗ (M. tuberculosis)

-8.0 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-9.4 kcal/mol

0better fit →-12

AutoDock Vina, one experimentally solved protein per pathogen. This project’s screening target is -7.0 kcal/mol, its own mark rather than a universal cutoff. A docking score is a structural hypothesis, not proof of binding, and requires experimental validation.

Other medicines to investigate

AI prediction · nothing measured

Other repurposing candidates: approved medicines besides Midostaurin with AI-predicted activity ≥40% against the chosen pathogen, leaving out existing antimicrobials. They are computational candidates for further investigation, not alternatives and not recommendations.

Sorted by AI-predicted activity, high to low. The order is not a ranking of which medicine is better.

Registered studies

A registration describes a study; it does not establish a positive result.

  • A Phase 1b Master Trial to Investigate CPX-351 in Subjects With Previously Untreated Acute Myeloid Leukemia

    NCT04075747 · start 2019-12-02

    Registered record
    Registry ID
    NCT04075747
    Conditions
    Acute Myeloid Leukemia
    Interventions
    CPX-351; Venetoclax; Midostaurin; Enasidenib
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2019-12-02
    Completion
    2023-09-12
    Enrolment
    57

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia
    InterventionCPX-351; Venetoclax; Midostaurin; Enasidenib
    PhasePhase 1
    Registry statusCompleted
  • HDM201 Added to CT in R/R or Newly Diagnosed AML

    NCT03760445 · start 2019-11-15

    Registered record
    Registry ID
    NCT03760445
    Conditions
    Leukemia, Myeloid, Acute
    Interventions
    HDM201; cytarabine; anthracycline; midostaurin; liposomal cytarabine/daunorubicin; posaconazole; midazolam
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Withdrawn
    Start
    2019-11-15
    Completion
    2023-06-13
    Enrolment
    0

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionLeukemia, Myeloid, Acute
    InterventionHDM201; cytarabine; anthracycline; midostaurin +3 more
    PhasePhase 1 / Phase 2
    Registry statusWithdrawn
  • A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML

    NCT03591510 · start 2019-03-13

    Registered record
    Registry ID
    NCT03591510
    Conditions
    FLT3-mutated Acute Myeloid Leukemia
    Interventions
    Midostaurin; Fludarabine; Cytarabine; Daunorubicin or idarubicin; Mitoxantrone; Etoposide
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Active not recruiting
    Start
    2019-03-13
    Completion
    2029-05-18
    Enrolment
    22

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionFLT3-mutated Acute Myeloid Leukemia
    InterventionMidostaurin; Fludarabine; Cytarabine; Daunorubicin or idarubicin +2 more
    PhasePhase 2
    Registry statusActive not recruiting
  • Gentuzumab Ozogamicin and Midostaurin Combination With Standard Cytarabine and Danunorubi Midostaurin as a Novel Approach to Treating Patients With Newly Diagnosed FLT-3 Mutated Acute Myeloid Leukemia

    NCT03900949 · start 2019-03-13

    Registered record
    Registry ID
    NCT03900949
    Conditions
    Acute Myeloid Leukemia
    Interventions
    Allogeneic Hematopoietic Stem Cell Transplantation; Cytarabine; Daunorubicin Hydrochloride; Gemtuzumab Ozogamicin; Midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2019-03-13
    Completion
    2025-10-23
    Enrolment
    21

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia
    InterventionAllogeneic Hematopoietic Stem Cell Transplantation; Cytarabine; Daunorubicin Hydrochloride; Gemtuzumab Ozogamicin +1 more
    PhasePhase 1
    Registry statusCompleted
  • iCare4: Genomic Signatures With Midostaurin in Acute Myeloid Leukemia (UF-HEM-004)

    NCT03207334 · start 2018-11

    Registered record
    Registry ID
    NCT03207334
    Conditions
    Acute Myeloid Leukemia
    Interventions
    Midostaurin; Cytarabine
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Withdrawn
    Start
    2018-11
    Completion
    2022-01
    Enrolment
    0

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia
    InterventionMidostaurin; Cytarabine
    PhasePhase 2
    Registry statusWithdrawn
  • Frailty Phenotype Assessments to Optimize Treatment Strategies for Older Patients With Hematologic Malignancies

    NCT03680677 · start 2018-09-21

    Registered record
    Registry ID
    NCT03680677
    Conditions
    Leukemia, Acute; MDS
    Interventions
    Bone Marrow or Peripheral Blood Graft (BMT); Cancer-directed Therapy or Best Supportive Care; Frailty Assessment
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Recruiting
    Start
    2018-09-21
    Completion
    2027-11
    Enrolment
    20

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionLeukemia, Acute; MDS
    InterventionBone Marrow or Peripheral Blood Graft (BMT); Cancer-directed Therapy or Best Supportive Care; Frailty Assessment
    PhaseNot recorded
    Registry statusRecruiting
  • Dexamethasone Added to Intensive Chemotherapy in Older Patients with Acute Myeloid Leukemia (AML)

    NCT03609060 · start 2018-08-24

    Registered record
    Registry ID
    NCT03609060
    Conditions
    Acute Myeloid Leukemia
    Interventions
    Dexamethasone
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Active not recruiting
    Start
    2018-08-24
    Completion
    2025-12-31
    Enrolment
    120

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia
    InterventionDexamethasone
    PhasePhase 2
    Registry statusActive not recruiting
  • Study of Crenolanib vs Midostaurin Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed FLT3 Mutated AML

    NCT03258931 · start 2018-08-15

    Registered record
    Registry ID
    NCT03258931
    Conditions
    Newly Diagnosed FLT3 Mutated AML
    Interventions
    Crenolanib; Midostaurin; Cytarabine; Duanorubicin
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2018-08-15
    Completion
    2026-04-06
    Enrolment
    214

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionNewly Diagnosed FLT3 Mutated AML
    InterventionCrenolanib; Midostaurin; Cytarabine; Duanorubicin
    PhasePhase 3
    Registry statusCompleted
  • A Global Study of the Efficacy and Safety of Midostaurin + Chemotherapy in Newly Diagnosed Patients With FLT3 Mutation Negative (FLT3-MN) Acute Myeloid Leukemia (AML)

    NCT03512197 · start 2018-07-20

    Registered record
    Registry ID
    NCT03512197
    Conditions
    Acute Myeloid Leukemia (AML)
    Interventions
    Midostaurin; Placebo; Chemotherapy
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2018-07-20
    Completion
    2021-02-12
    Enrolment
    511

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia (AML)
    InterventionMidostaurin; Placebo; Chemotherapy
    PhasePhase 3
    Registry statusCompleted
  • Midostaurin Associated With Standard Chemotherapy in Patients With Core-binding Factor Leukemia

    NCT03686345 · start 2018-07-01

    Registered record
    Registry ID
    NCT03686345
    Conditions
    Core Binding Factor Acute Myeloid Leukemia (CBF-AML)
    Interventions
    Midostaurin
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2018-07-01
    Completion
    2024-12-31
    Enrolment
    34

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionCore Binding Factor Acute Myeloid Leukemia (CBF-AML)
    InterventionMidostaurin
    PhasePhase 2
    Registry statusTerminated
  • A Study of Midostaurin Efficacy and Safety in Newly Diagnosed Patients With FLT3-mutated AML

    NCT03280030 · start 2018-04-06

    Registered record
    Registry ID
    NCT03280030
    Conditions
    Acute Myeloid Leukemia
    Interventions
    Midostaurin; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2018-04-06
    Completion
    2022-11-14
    Enrolment
    67

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia
    InterventionMidostaurin; Placebo
    PhasePhase 2
    Registry statusCompleted
  • Study to Assess the Safety and Efficacy of Midostaurin (PKC412) in Combination With Standard Chemotherapy During Induction and Consolidation Followed by 12 Months of Maintenance Monotherapy in Patients With Newly-diagnosed FMS-like Tyrosine 3 (FLT3) Kinase Receptor-mutated Acute Myeloid Leukemia.

    NCT03379727 · start 2018-02-13

    Registered record
    Registry ID
    NCT03379727
    Conditions
    Acute Myeloid Leukemia
    Interventions
    Midostaurin; Cytarabine; anthracycline ((daunorubicin or idarubicin): Induction Phase only
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2018-02-13
    Completion
    2021-07-09
    Enrolment
    301

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia
    InterventionMidostaurin; Cytarabine; anthracycline ((daunorubicin or idarubicin): Induction Phase only
    PhasePhase 3
    Registry statusCompleted
  • Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

    NCT03092674 · start 2018-02-02

    Registered record
    Registry ID
    NCT03092674
    Conditions
    Acute Myeloid Leukemia; Myelodysplastic Syndrome; Myelodysplastic Syndrome/Acute Myeloid Leukemia
    Interventions
    Azacitidine; Cytarabine; Decitabine; Laboratory Biomarker Analysis; Midostaurin; Nivolumab
    Study type
    INTERVENTIONAL
    Phase
    Phase 2 / Phase 3
    Registry status
    Completed
    Start
    2018-02-02
    Completion
    2024-06-26
    Enrolment
    76

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia; Myelodysplastic Syndrome; Myelodysplastic Syndrome/Acute Myeloid Leukemia
    InterventionAzacitidine; Cytarabine; Decitabine; Laboratory Biomarker Analysis +2 more
    PhasePhase 2 / Phase 3
    Registry statusCompleted
  • Geriatric Assessment & Genetic Profiling to Personalize Therapy in Older Adults With Acute Myeloid Leukemia

    NCT03226418 · start 2017-07-07

    Registered record
    Registry ID
    NCT03226418
    Conditions
    Adult Acute Myeloid Leukemia; Secondary Acute Myeloid Leukemia; Therapy-Related Acute Myeloid Leukemia
    Interventions
    Cytarabine; Decitabine; Idarubicin; Laboratory Biomarker Analysis; Liposome-encapsulated Daunorubicin-Cytarabine; Quality-of-Life Assessment; Questionnaire Administration; Azacitidine; Venetoclax; glasdegib
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2017-07-07
    Completion
    2024-10-01
    Enrolment
    75

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAdult Acute Myeloid Leukemia; Secondary Acute Myeloid Leukemia; Therapy-Related Acute Myeloid Leukemia
    InterventionCytarabine; Decitabine; Idarubicin; Laboratory Biomarker Analysis +6 more
    PhasePhase 2
    Registry statusCompleted
  • Improving Risk Assessment of AML With a Precision Genomic Strategy to Assess Mutation Clearance

    NCT02756962 · start 2016-07-06

    Registered record
    Registry ID
    NCT02756962
    Conditions
    Acute Myeloid Leukemia
    Interventions
    Cytarabine; Allogeneic stem cell transplant; Bone marrow aspiration; Punch skin biopsy; ClinSeq
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Active not recruiting
    Start
    2016-07-06
    Completion
    2029-07-31
    Enrolment
    107

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcute Myeloid Leukemia
    InterventionCytarabine; Allogeneic stem cell transplant; Bone marrow aspiration; Punch skin biopsy +1 more
    PhasePhase 2
    Registry statusActive not recruiting