NCT03686345

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

Prospective Evaluation of a Continuation Therapy With Midostaurin in Adult Patients With Core-binding Factor Leukemia and Integrated Genetic Analysis: a Multi-center Phase II Study

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Niguarda Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

In this prospective, Interventional, Single-Arm, Open-Label, Phase-II Trial aims at demonstrating a decrease in the 2-year Relapse Incidence (RI) and in the 2-year Cumulative Relapse Incidence among the Midostaurin-treated patients compared to a cohort of historical controls. The investigators established that a 20% net reduction in the 2-year RI may be a clinically significant goal. So the investigators set our RI objective at 28%. Furthermore, the investigators will assess if the experimental treatment may obtain an increased 5-years Overall, Disease-Free and Event free Survival. The safety profile of Midostaurin given in combination with induction and consolidation chemotherapy and as a single agent in maintenance in CBFL patients will also be assessed.

Conditions

• Core Binding Factor Acute Myeloid Leukemia (CBF-AML)

Interventions

Type	Name	Description
DRUG	Midostaurin	Midostaurin associated with standard chemotherapy in patients with core-binding factor leukemia

Timeline

• Start date: 2018-07-01
• Primary completion: 2023-06-30
• Completion: 2024-12-31
• First posted: 2018-09-26
• Last updated: 2025-02-25

Locations

1 site across 1 country: Italy