NCT03686345: AML FLT3

Midostaurin Associated With Standard Chemotherapy in Patients With Core-binding Factor Leukemia

Terminated Phase 2 Last updated 25 February 2025

What this trial tests

Phase 2 trial testing Midostaurin in Core Binding Factor Acute Myeloid Leukemia (CBF-AML) in 34 participants. Terminated before completion.

Timeline

1 July 2018

Primary endpoint
30 June 2023

31 December 2024

Quick facts

Lead sponsor	Niguarda Hospital
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	34
Start date	1 July 2018
Primary completion	30 June 2023
Estimated completion	31 December 2024
Sites	1 location across Italy

Drugs / interventions tested

• Midostaurin (MIDOSTAURIN) — full drug profile →

Conditions studied

• Core Binding Factor Acute Myeloid Leukemia (CBF-AML) — all drugs for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) →

Sponsor

Niguarda Hospital

Who can join

Adults 18 to 65, any sex, with Core Binding Factor Acute Myeloid Leukemia (CBF-AML). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this single-arm, open label, phase-II trial, is to determine whether the association of Midostaurin to standard induction, consolidation therapy and in maintenance therapy as single agent, is effective in decrease relapse incidence, in patients with CBF-AML. The single-arm, open label, phase-II study is based on data obtained from previous clinical and pre-clinical studies, obtained by use of Midostaurin in patients with Acute Myeloid Leukemia (with or without FLT3 mutations) and in patients with Mast cell disorders (characterized by mutations in the C-KIT gene). The investigators believe that Midostaurin, associated with standard therapy Anthracycline/AraC Induction, to the consolidation regimen with high doses of araC and maintenance therapy to single agent in patients with acute myeloid leukemia core-binding factor can significantly reduce the incidence of recurrence of the disease, occurring in 40-50% of cases treated with standard therapy

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. <i>FLT3</i> Mutations in Acute Myeloid Leukemia: Key Concepts and Emerging Controversies.
   Kennedy VE, Smith CC. · · 2020 · cited 132× · PMID 33425766 · DOI 10.3389/fonc.2020.612880
2. Mechanisms Underlying Resistance to FLT3 Inhibitors in Acute Myeloid Leukemia.
   Eguchi M, Minami Y, Kuzume A, Chi S. · · 2020 · cited 54× · PMID 32722298 · DOI 10.3390/biomedicines8080245
3. Midostaurin In Acute Myeloid Leukemia: An Evidence-Based Review And Patient Selection.
   Abbas HA, Alfayez M, Kadia T, Ravandi-Kashani F, et al · · 2019 · cited 23× · PMID 31632141 · DOI 10.2147/cmar.s177894
4. Midostaurin Reverses ABCB1-Mediated Multidrug Resistance, an <i>in vitro</i> Study.
   Ji N, Yang Y, Cai CY, Wang JQ, et al · · 2019 · cited 23× · PMID 31275850 · DOI 10.3389/fonc.2019.00514
5. Advances in therapeutic options for newly diagnosed, high-risk AML patients.
   Doucette K, Karp J, Lai C. · · 2021 · cited 16× · PMID 33995985 · DOI 10.1177/20406207211001138
6. Putative role of natural products as Protein Kinase C modulator in different disease conditions.
   Singh RK, Kumar S, Tomar MS, Verma PK, et al · · 2021 · cited 11× · PMID 34216003 · DOI 10.1007/s40199-021-00401-z