Trial Outcomes & Findings for An Efficacy and Safety Study Of Pracinostat In Combination With Azacitidine In Adults With Acute Myeloid Leukemia (NCT NCT03151408)

Approx. 130 sites worldwide (planned), 116 sites where patients were randomized. Date of 1st patient screened: 12 Jul 2017 and Date of last patient completed: 08 Aug 2020 A total of 725 patients were screened. Of these, 319 were considered screening failures, so a total of 406 patients were randomized

Based on request of the IDMC, the interim analysis was actually done on 30Jun2020 when 232/390 events occurred in the study, The study was stopped for futility.

An Efficacy and Safety Study Of Pracinostat In Combination With Azacitidine In Adults With Acute Myeloid Leukemia

OS measures the time from randomization to death due to any cause.

The CR rate is the proportion of patients who achieve a morphologic CR according to the response criteria * \<5% blasts in a bone marrow aspirate sample with spicules * There should be no blasts with Auer rods * No EMD * Absolute Neutrophil Count (ANC) ≥1,000/μL * Platelet count of ≥100,000/μL * Patient must be independent of transfusions (for at least 1week before each assessment)

proportion of patients who achieve a CR without minimal residual disease by multicolor flow cytometry according to the following criteria * Morphologic CR * Minimal Residual Disease (MRD) by MFC negative

The CRc rate is the proportion of patients who achieve a reversion to a normal karyotype at CR within the study period. This endpoint applies only to patients with abnormal cytogenetic at enrollment according to the following criterion Morphologic CR plus reversion to a normal karyotype (defined as no clonal abnormalities detected in a minimum of 20 mitotic cells)

Transfusion independence rate is defined as the proportion of patients who show eight weeks or over without red blood cell (RBC-TI) and/or platelet (PLT-TI) transfusion during study period

Composite complete remission (cCR) rate is the proportion of patients who achieve either a disease response of CR, CRi or MLFS (i.e., cCR = CR + CRi + MLFS) within the study period, according to the response criteria

Duration of cCR response is the time from first cCR until documented relapse (the definition of relapse from CR will be applied) or death. Duration of cCR is only defined for patients who achieve a cCR

QLQ-C30 is made of multi-item scales and single-item measures (functional and symptom scales, a global health status/QoL scale and single items). Item range is the difference between possible max and min response to individual items of the scale; most items take values from 1 to 4 (range=3). Global health status takes values from 1 to 7 (range = 6). For statistical analysis purpose, single-item and scale values were all standardized (according to linear transformation described in Scoring Manual) to obtain scores ranging 0-100. An high scale score represents an higher response level. Thus an high score for a functional scale represents an high/healthy level of functioning, an high score for the global health status/QoL represents a high QoL, an high score for a symptom scale/item represents an high level of symptomatology/problems.

the time from the date of achievement of CR or CRi until the date of relapse or death from any cause

PFS is defined as the time from the date of randomization until the date of relapse (progression), or death from any cause, whichever occurs first.

Duration of morphologic CR is defined as the time from the date of achievement of CR until the date of relapse (progression).

Time to CR is defined as the time from the date of randomization until the date of CR in the absence of interceding therapies. The analysis set was the ITT set.

Morphologic CR within 6 cycles rate is defined as the proportion of patients who achieved CR in the absence of interceding therapies within 6 treatment cycles (i.e., during treatment phase up to Day 1 of Cycle 7 included). Analysis was performed in the ITT set.