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    Association details:
    Biomarker:FLT3 mutation
    Cancer:Leukemia
    Drug:Rydapt (midostaurin) (Multi-tyrosine kinase inhibitor)
    Direction:Sensitive
    Evidence:
    Evidence Level:
    Sensitive: B - Late Trials
    Source:
    ASH 2021
    Title:

    Midostaurin in Patients (Pts) with Newly Diagnosed FLT3-Mutation Negative Acute Myeloid Leukemia (AML): Final Results and Measurable Residual Disease (MRD) Analyses from the Unify Trial

    Published date:
    11/04/2021
    Excerpt:
    Final HR for EFS was 1.00 (95% CI, 0.78–1.29); the majority of events in both treatment arms were induction failures (Table). HR for OS was 0.85 (95% CI, 0.57–1.25) in favor of midostaurin. The safety profile of midostaurin was consistent with prior reports (Table)....Results from UNIFY are consistent with the safety/tolerability profile previously reported for midostaurin, but do not show efficacy for midostaurin in FLT3-MN AML; this suggests that the clinical effect of midostaurin in AML is primarily in the FLT3-mutated setting.
    DOI:
    10.1182/blood-2021-148129
    Trial ID:
    UNIFY
    Evidence Level:
    Sensitive: C1 - Off-label
      (Approved for Acute Myelogenous Leukemia)
    New
    Excerpt:
    Rydapt is indicated...in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive...
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    Open-label Multicenter Study of PKC412 in Pts With AML and MDS With Either Wild-type or Mutated FLT3

    Excerpt:
    ...- Patients with AML who are or MDS subtypes RAEB, RAEB-T or CMML - Patients with a wild type or mutated FLT3 documented within 14 days prior to start of study who have - Patients with a WHO performance status of 0 to 2 with a life expectancy of at least 3 months ...
    Trial ID:
    NCT00977782
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    RAD001 in Combination With PKC412 in Patients With Relapsed, Refractory or Poor Prognosis AML or MDS

    Excerpt:
    ...Observe anti-leukemic effects of this combination including a coda of patients with mutant FLT3 AML.`...
    More C2 evidence
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    PKC412 With or Without Itraconazole in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

    Excerpt:
    ...DISEASE CHARACTERISTICS: - One of the following diagnoses: - Acute myeloid leukemia, meeting 1 of the following criteria: - Relapsed or refractory disease after standard therapy and - Myelodysplastic syndromes of 1 the following types: - Refractory anemia with excess blasts (RAEB) - RAEB in transformation - Chronic myelomonocytic leukemia - Documented wild type or mutated FLT3 PATIENT CHARACTERISTICS: Age - 18 and over Performance status - WHO 0-2 Life expectancy - At least 3 months Hematopoietic - - SGOT or SGPT ≤ 3 times upper limit of normal (ULN) - Bilirubin ≤ 2 times ULN Renal - Creatinine ≤ 1.5 times ULN Pulmonary - - - - - - - Negative pregnancy test - Fertile patients must use effective contraception PRIOR CONCURRENT THERAPY: Biologic therapy - At least 2 months since prior allogeneic, syngeneic, or autologous bone marrow or stem cell transplantation Chemotherapy - See Disease Characteristics - Recovered from prior cytotoxic chemotherapy - At least 1 week since prior hydroxyurea Endocrine therapy - - - - At least 4 weeks since prior anticancer therapy - - - ...
    Trial ID:
    NCT00045578
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia

    Excerpt:
    ...or FLT3 mutated Acute Myeloid Leukemia (AML) that does...
    Trial ID:
    NCT00866281
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML

    Excerpt:
    ...- Presence of a FLT3 mutation status as measured/confirmed by a designated lab with results available prior first dose of Midostaurin...
    Trial ID:
    NCT03591510
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    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.

    Excerpt:
    ......
    Trial ID:
    A2408
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    MAP to Provide Access to Midostaurin, for Patients 18 Years of Age and Older With Newly-diagnosed FLT3-mutated AML and Eligible for Induction and Consolidation Chemotherapy

    Excerpt:
    ...Patients must have a documented FLT3-mutation (ITD and/or TKD)....
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    A Study of Midostaurin Efficacy and Safety in Newly Diagnosed Patients With FLT3-mutated AML

    Excerpt:
    ...- Documented presence of an ITD and/or TKD activating mutation in the FLT3 gene, as determined by analysis in a Novartis designated laboratory An exception will be patients who are enrolled into the part 1 in Japan, who may be treated with midostaurin irrespective of AML FLT3 genotype....
    Trial ID:
    NCT03280030
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    Midostaurin and Azacitidine in Treating Elderly Patients With Acute Myelogenous Leukemia

    Excerpt:
    ...- Patients with and without FLT3 mutations will be eligible to participate...
    Trial ID:
    NCT01093573
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    Midostaurin in MRD (Minimal Residual Disease) Positive Acute Myeloid Leukemia After Allogeneic Stem Cell Transplantation

    Excerpt:
    ...number of patients with low MRD (Minimal Residual Disease)`Incidence of acute and chronic graft-versus-host disease (GvHD)`Incidence of adverse events grade 3-5 of midostaurin after allogeneic SCT`Next-generation sequencing analyses of FLT3-mutation`quality of life assessment with certified "EORTC QLQ - C30 questionnaire"...
    Trial ID:
    NCT03951961
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    PKC412, Daunorubicin, and Cytarabine in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

    Excerpt:
    ...CR rate by FLT3 mutation and treatment arm`Overall survival by FLT3 mutation status...
    Trial ID:
    NCT00093600
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    A Safety and Efficacy Study of LGH447 in Patients With Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)

    Excerpt:
    ...AML patients may have either FLT3 wild type or FLT3-ITD/TKD mutant disease, and FLT3 mutation status needs to be defined at study entry....
    Trial ID:
    NCT02078609
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    Midostaurin Access Program for Newly Diagnosed FLT3 (ITD or TKD) Mutated AML Adult Patients Eligible for Standard Induction and Consolidation Chemotherapy

    Excerpt:
    ...- Patients must have a documented FLT3 mutation (ITD or TKD)...
    Trial ID:
    AMLFLT3
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    An Expanded Treatment Protocol (ETP) of Midostaurin (PKC412) in Patients 18 Years of Age or Older With Newly-diagnosed FLT3-mutated Acute Myeloid Leukemia (AML)

    Excerpt:
    ...Patients must have a documented FLT3 mutation (ITD or TKD) 4....
    Trial ID:
    NCT03114228
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    New
    Source:
    clinicaltrials.gov
    Title:

    Midostaurin in Treating Older Patients With Mutated Acute Myeloid Leukemia Post-Transplant

    Excerpt:
    ...- Elderly patients with FLT3-mutated acute myeloid leukemia (AML)...
    Trial ID:
    NCT02723435
    Less C2 evidence
    Evidence Level:
    Sensitive: D – Preclinical
    Source:
    Eur J Haematol
    Title:

    Use of Midostaurin in Mixed Phenotype Acute Leukemia with FLT3 Mutation: A Case Series

    Published date:
    10/15/2021
    Excerpt:
    Here, we report the successful use of midostaurin to treat three FLT3-positive MPAL T/myeloid and B/myeloid patients. hese results suggest that the addition of midostaurin during induction and consolidation therapy may represent a treatment option for FLT3-positive MPAL.
    DOI:
    10.1111/ejh.13717