The main part of this trial is a phase II study of vosaroxin with azacitidine in older patients with newly diagnosed AML and intermediate or adverse genetic risk or MDS-EB-2. An initial safety run-in phase of the study will be performed administering the study drug vosaroxin with azacitidine in up to 18 patients. After completion of the run-in phase, toxicity and response data will be provided to the external Data and Safety Monitoring Board (DSMB) and the Trial Committee by the Coordinating Investigator. The Trial Committee will decide on the basis of these data and the recommendation of the DSMB on dose modification and the vosaroxin dose for the phase II part of the study, which will include 150 patients in total.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Cycle 1-8: Dose Level 0: 70mg/m², Dose Level -1: 50mg/m², Dose Level -2: 40mg/m², IV over ten minutes, d 1+4 .
Cycle 1-8 Azacitidine: 75 mg/m²/d subcutaneously, d 1-7; Maintenance with single agent azacitidine at 75 mg/m²/d on days 1-7 until relapse or progression.
Kliniken Essen-Süd, Evang. Krankenhaus Essen-Werden gGmbH
Essen, Germany
Klinikum Oldenburg, Klinik für Innere Medizin II
Oldenburg, Germany
University Hospital Ulm
Ulm, Germany
Rate of complete remission (CR) and CR with incomplete blood count recovery (CRi)
Efficacy assessments
Time frame: 2 months
CR and CRi in a pre-defined subgroup analysis in patients with complex karyotype
Efficacy assessments
Time frame: 2 months
CR and rate of combined CR/CRi and CR with negativity for minimal residual disease (CRMRD-)
Efficacy assessments
Time frame: 2 months
Duration of response (DOR)
Efficacy assessments
Time frame: 4 years
Event-free survival (EFS)
Efficacy assessments
Time frame: 4 years
Overall survival (OS)
Efficacy assessments
Time frame: 4 years
30-day and 60-day mortality
Safety assessments
Time frame: 30 and 60 days
Incidence and intensity of adverse events (AEs) according to Common Terminology Criteria for Adverse Events (CTCAE) version v4.0
Safety assessments
Time frame: 12 months
Assessment of Quality of life by the EORTC Quality of Life Core Questionnaire (QLQ-C30)
Quality of life
Time frame: 4 years
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