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TerminatedNCT03151408Updated Mar 10, 2022Results posted

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

A Phase 3 interventional study of Pracinostat and Placebos in Acute Myeloid Leukemia, sponsored by Helsinn Healthcare SA. Terminated at 138 sites in 16 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-03-10.

Sponsored by Helsinn Healthcare SA · Phase 3, Interventional, and Treatment

Why this study was terminated
The IDMC recommended to stop the study prematurely due to a lack of efficacy.
Phase
Phase 3
Study type
Interventional
Enrollment
406
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

This is a Phase III, multicenter, double-blind, randomized study of pracinostat vs. placebo with azacitidine (AZA) as background therapy in patients ≥ 18 years of age with newly diagnosed acute myeloid leukemia (AML), excluding acute promyelocytic leukemia and cytogenetic low-risk AML, who are unfit to receive intensive remission induction chemotherapy due to age ≥ 75 years or comorbidities. Patients will be randomized in a 1:1 ratio to one of two groups: Group A (experimental group) to receive pracinostat plus AZA and Group B (control group) to receive placebo plus AZA. Randomization will be stratified by cytogenetic risk category (intermediate vs. unfavorable-risk, according to SWOG Cytogenetic Risk Category Definitions) and ECOG performance status (0-1 vs. 2). Treatments will be administered based on 28-day cycles, with pracinostat/placebo administered orally once every other day, 3 times a week for 3 weeks, followed by one week of no treatment and AZA administered for 7 days of each cycle. Study treatment should continue until there is documented disease progression, relapse from complete remission (CR), or non-manageable toxicity. A minimum of 6 cycles may be required to achieve a complete remission. Once permanently discontinued from study treatment, patients will enter the Long-term Follow-up phase of the study and will be followed for assessment of disease progression, if applicable, and survival every 3 months (±1 month) until death. The end of this study is defined when 390 events (deaths) have occurred and the study is unblinded for final overall survival analysis. Patients who are receiving study treatment at the end of the study may have the opportunity to continue to receive the study drugs to which they were randomized to (Post- Study Observation Period), until the Sponsor informs the Investigators of the appropriate course of action based on the study results. The Post-Study Observation Period is defined as the period starting from the end of the study for a maximum of 12 months.

02

Conditions studied

  • Acute Myeloid Leukemia

Keywords

  • AML
03

In context

Leukemia

5,442 studies on the registry are indexed under Leukemia; 637 are open to participants now.

This study's enrollment of 406 is above the median of 38 across 4,248 interventional studies indexed under Leukemia.

Browse Leukemia studies →

Lead sponsor

Helsinn Healthcare SA is the lead sponsor of 31 studies on the registry; 1 is open to participants now.

Of its 8 completed or terminated interventional studies of FDA-regulated products, 7 (88%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Male or female patient ≥ 18 years of age with newly diagnosed, histologically or cytologically confirmed, AML including de novo, secondary to antecedent hematologic disorders, or treatment-related disease with intermediate or unfavorable-risk cytogenetics

  2. Unable to receive intensive chemotherapy regimens at enrollment, based on one of the following:

    I. Age ≥ 75 years, or

    II. Age \< 75 years with at least 1 of the following co-morbidities:

    1. An ECOG performance status of 2
    2. Clinically significant cardiovascular disease defined as:

    i. Left ventricular ejection fraction (LVEF) ≤ 50%, measured within 3 months prior to Day 1 confirmed by ECHO/MUGA ii. Congestive heart failure requiring medical therapy iii. Chronic stable angina requiring medical therapy iv. Prior cerebrovascular accident with sequelae c. Clinically significant pulmonary disease defined as: i. Forced expiratory volume in 1 second (FEV1) ≤ 65% of expected ii. Lung diffusing capacity for carbon monoxide (DLCO) ≤ 65% of expected Confirmed by pulmonary tests. d. Diabetes mellitus with symptomatic end-organ damage (e.g., retinopathy, nephropathy, neuropathy, vasculopathy) e. Autoimmune inflammatory conditions (e.g., rheumatoid arthritis, systemic lupus erythematous, inflammatory bowel disease, or similar) requiring chronic disease modifying therapy (e.g., etanercept, adalimumab, infliximab, rituximab, methotrexate, or similar) f. Class III obesity defined as a Body Mass Index (BMI) > 40 kg/m2 g. Renal impairment defined as serum creatinine > 1.3 mg/dL (> 115 µmol/L) or creatinine clearance \<70 ml/min h. Clinically significant cognitive impairment defined as requiring medical therapy and/or assistance with activities of daily living

  3. 20% blasts in bone marrow
  4. Peripheral white blood cell (WBC) count 30,000/µL For cyto-reduction, hydroxyurea is allowed during screening and up to Cycle 1, Days 1-14, to reduce WBC count to \< 30,000 µL prior to Day 1. After Cycle 1, Day 14, hydroxyurea is prohibited.
  5. ECOG performance status ≤ 2

  6. Adequate organ function as evidenced by the following laboratory findings:

    1. Total bilirubin ≤ 2 × upper limit of normal (ULN) or \< 3 x ULN for patients with Gilbert-Meulengracht Syndrome
    2. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN
  7. Serum creatinine ≤ 1.5 × ULN according to institutional standards or creatinine clearance ≥ 50 mL/min
  8. QT-interval corrected according to Fridericia's formula (QTcF) ≤ 450 ms on electrocardiogram (ECG) at Screening
  9. Male patient who is surgically sterile, or male patient who is willing to agree to remain completely abstinent (refrain from heterosexual intercourse) or who use barrier contraceptive measures and agree to refrain from donating sperm during the entire study treatment period and for 3 months after the last administration of study drug
  10. Female patient who is of childbearing potential willing to use adequate contraceptive measures while participating on study, OR willing to completely abstain from heterosexual intercourse during the entire study treatment period
  11. Female patient who is of childbearing potential must have a negative serum pregnancy test result within 3 weeks prior to starting study drugs.
  12. Willing to provide voluntary written informed consent before performance of any study related procedure not part of normal medical care
  13. Willing and able to understand the nature of this study and to comply with the study and follow-up procedures.

Exclusion criteria

Exclusion Criteria:

  1. Able to receive intensive induction chemotherapy
  2. AML-associated inv(16)/t(16;16)/del(16q), t(15;17) (i.e. promyelocytic leukemia) with/without secondary aberrations; t(8;21) lacking del (9q) or complex karyotypes
  3. Presence of an active malignant disease within the last 12 months, with the exception of adequately treated cervical cancer in-situ, non-melanoma skin cancer and superficial bladder tumors (Ta [non-invasive tumor], Tis [carcinoma in situ] and T1 [tumor invades lamina propria]). Other malignancies may be considered after consultation with the Medical Monitor
  4. Life-threatening illnesses other than AML, uncontrolled medical conditions or organ system dysfunction that, in the Investigator's opinion, could compromise the patient's safety or put the study outcomes at risk
  5. Uncontrolled arrhythmias; any Class 3-4 cardiac diseases as defined by the New York Heart Association (NYHA) functional classification
  6. Evidence of AML central nervous system (CNS) involvement

  7. Previous chemotherapy for AML except for the following, which are allowed:

    1. Hydroxyurea for cytoreduction
    2. One course of hypomethylating agent therapy (i.e.; up to 7 doses of azacitidine or 3-5 days of decitabine) within 30 days prior to enrollment (Day 1)
  8. Use of experimental drugs ≤ 30 days prior to screening
  9. Received prior HDAC inhibitor therapy
  10. Received prior treatment with a hypomethylating agent, except as allowed in Exclusion Criterion 7.b
  11. Known hypersensitivity to any components of pracinostat, azacitidine, or mannitol
  12. History of human immunodeficiency virus (HIV) or an active and uncontrolled infection with hepatitis C virus (HCV) or hepatitis B virus (HBV)
  13. Gastrointestinal (GI) tract disease that causes an inability to take oral medication, malabsorption syndrome, or a requirement for IV alimentation; prior surgical procedures affecting absorption; or uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis)
  14. Any disease(s), psychiatric condition, metabolic dysfunction, or findings from a physical examination or clinical laboratory test result that would cause reasonable suspicion of a disease or condition, that contraindicates the use of pracinostat and/or AZA, that may increase the risk associated with study participation, that may affect the interpretation of the results, or that would make the patient inappropriate for this study
  15. Breast-feeding woman
  16. current smokers(use of patches, chewing gums and vaping nicotine conaining fluids is permitted). Patients who stopped smoking at least 8 day prior to first pracinostat dosing can be enrolled, provided they refrain from smoking during the whole study
  17. prohibited concomitant medications
  18. uncontrolled infections
  19. receive more than 1 prior cycle of HMA or bone marrow transplant for any prior hematological disorder antecedent to AML
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
406 participants (actual)

Study arms

  • Experimental
    Pracinostat plus AZA

    60 mg capsule orally, once a day, 3 times a week for 3 weeks, followed by 1 week of rest of each 28-day cycle. As a background therapy azacitidine (AZA) will be administered at a dose of 75 mg/m2 by SC or IV injection daily for 7 days of each 28-day cycle.

    Drug: Pracinostat · Drug: Azacitidine

  • Placebo comparator
    Placebo plus AZA

    1 capsule orally, once a day, 3 times a week for 3 weeks, followed by 1 week of rest of each 28-day cycle. As a background therapy azacitidine (AZA) will be administered at a dose of 75 mg/m2 by SC or IV injection daily for 7 days of each 28-day cycle.

    Drug: Placebos · Drug: Azacitidine

Interventions

  • DrugPracinostat

    60 mg capsule

    Also known as: SB939

  • DrugPlacebos

    capsule

  • DrugAzacitidine

    SC or IV injection

    Also known as: AZA

06

What researchers measure

Primary outcomes

  1. Overall Survival

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

    Time frame: 826 days

Secondary outcomes

  1. Morphologic Complete Remission (CR) Rate

    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)

    Time frame: 744 days

  2. Complete Remission Without Minimal Residual Disease (CRmrd) Rate

    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

    Time frame: 826 days

  3. Cytogenetic Complete Remission (CRc) Rate

    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)

    Time frame: 826 days

  4. Transfusion Independence (TI)

    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

    Time frame: 826 days

Other outcomes

  1. Composite Complete Remission (cCR) Rate

    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

    Time frame: 744 days

  2. Duration of Composite Complete Remission

    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

    Time frame: 744 days

  3. Change in Quality of Life From Baseline (EORTC QLQ-C30 - European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30)

    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.

    Time frame: from baseline up to 660 days

  4. Relapse Free Survival

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

    Time frame: 744 days

  5. Progressive Free Survival Rate (PFS)

    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.

    Time frame: 800 days

  6. Duration of Morphologic CR

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

    Time frame: 744 days

  7. Time to CR

    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.

    Time frame: 616 days

  8. Morphologic CR Within 6 Cycles Rate

    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.

    Time frame: within 6 cycles

07

Results

Posted Mar 10, 2022
Limitations and caveats
Results of the interim analysis (OS curves crossing at about 300 days) demonstrated futility according to the planned threshold and concluded that it was unlikely to meet the primary endpoint compared to the control group at the final analysis. Based on this outcome, the decision taken was to discontinue the recruitment of patients and terminate the study.

Participant flow

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

Participant flow — Overall Study
MilestonePracinostat Plus AZAPlacebo Plus AZA
Started203203
Treated201201
Completed00
Not completed203203
Withdrew: Adverse event2423
Withdrew: Death4626
Withdrew: Physician decision1319
Withdrew: Withdrawal by subject1620
Withdrew: Progressive disease4561
Withdrew: Non-compliance by patient10
Withdrew: Other reasons5652
Withdrew: Randomized and not treated22

Outcome measures

PrimaryOverall Survival

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

Time frame:
826 days
Reported as:
Median · days
Overall Survival
daysPracinostat Plus AZAPlacebo Plus AZA
Overall Survival303 (209 to 400)303 (248 to 346)
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Log Rank · p = 0.8275 (P-value stratified by cytogenetic risk factor and ECOG performance status)
SecondaryMorphologic Complete Remission (CR) Rate

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)

Time frame:
744 days
Reported as:
Count of participants · Participants
Morphologic Complete Remission (CR) Rate
ParticipantsPracinostat Plus AZAPlacebo Plus AZA
Morphologic Complete Remission (CR) Rate2435
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Cochran-Mantel-Haenszel · p = 0.1244
SecondaryComplete Remission Without Minimal Residual Disease (CRmrd) Rate

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

Time frame:
826 days
Reported as:
Count of participants · Participants
Complete Remission Without Minimal Residual Disease (CRmrd) Rate
ParticipantsPracinostat Plus AZAPlacebo Plus AZA
Complete Remission Without Minimal Residual Disease (CRmrd) Rate1220
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Cochran-Mantel-Haenszel · p = 0.1430
SecondaryCytogenetic Complete Remission (CRc) Rate

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)

Time frame:
826 days
Reported as:
Count of participants · Participants
Cytogenetic Complete Remission (CRc) Rate
ParticipantsPracinostat Plus AZAPlacebo Plus AZA
Cytogenetic Complete Remission (CRc) Rate78
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Cochran-Mantel-Haenszel · p = 0.9977
SecondaryTransfusion Independence (TI)

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

Time frame:
826 days
Reported as:
Count of participants · Participants
Transfusion Independence (TI)
ParticipantsPracinostat Plus AZAPlacebo Plus AZA
Transfusion Independence (TI)8181
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Cochran-Mantel-Haenszel · p = 0.9959
Other pre-specifiedComposite Complete Remission (cCR) Rate

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

Time frame:
744 days
Reported as:
Count of participants · Participants
Composite Complete Remission (cCR) Rate
ParticipantsPracinostat Plus AZAPlacebo Plus AZA
Composite Complete Remission (cCR) Rate7364
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Cochran-Mantel-Haenszel · p = 0.3502
Other pre-specifiedDuration of Composite Complete Remission

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

Time frame:
744 days
Reported as:
Median · days
Duration of Composite Complete Remission
daysPracinostat Plus AZAPlacebo Plus AZA
Duration of Composite Complete Remission576 (276 to NA)319 (170 to 744)
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Log Rank · p = 0.0502
Other pre-specifiedChange in Quality of Life From Baseline (EORTC QLQ-C30 - European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30)

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.

Time frame:
from baseline up to 660 days
Reported as:
Mean · score on a scale
Change in Quality of Life From Baseline (EORTC QLQ-C30 - European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30)
score on a scalePracinostat Plus AZAPlacebo Plus AZA
Global Health Status-7.040 ± 31.4458-2.303 ± 28.6611
Functional Scale - Physical Functioning-8.937 ± 32.1163-7.018 ± 25.8512
Functional Scale - Role Functioning-6.034 ± 46.3793-2.000 ± 40.2659
Symptom Scale - Fatigue2.107 ± 34.29284.240 ± 29.7030
Symptom Scale - Nausea and Vomiting5.460 ± 28.16495.263 ± 23.2870
Symptom Scale - Appetite Loss9.771 ± 42.81073.509 ± 40.2150
Other pre-specifiedRelapse Free Survival

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

Time frame:
744 days
Reported as:
Median · days
Relapse Free Survival
daysPracinostat Plus AZAPlacebo Plus AZA
Relapse Free Survival291 (217 to 429)190 (155 to 319)
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Log Rank · p = 0.4656
Other pre-specifiedProgressive Free Survival Rate (PFS)

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.

Time frame:
800 days
Reported as:
Median · days
Progressive Free Survival Rate (PFS)
daysPracinostat Plus AZAPlacebo Plus AZA
Progressive Free Survival Rate (PFS)217 (126 to 257)220 (182 to 271)
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Log Rank · p = 0.7063
Other pre-specifiedDuration of Morphologic CR

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

Time frame:
744 days
Reported as:
Median · days
Duration of Morphologic CR
daysPracinostat Plus AZAPlacebo Plus AZA
Duration of Morphologic CRNA (352 to NA)319 (119 to 744)
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Log Rank · p = 0.0592
Other pre-specifiedTime to CR

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.

Time frame:
616 days
Reported as:
Median · days
Time to CR
daysPracinostat Plus AZAPlacebo Plus AZA
Time to CRNA (NA to NA)361 (311 to NA)
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Log Rank · p = 0.3835
Other pre-specifiedMorphologic CR Within 6 Cycles Rate

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.

Time frame:
within 6 cycles
Reported as:
Number · number of patients
Morphologic CR Within 6 Cycles Rate
number of patientsPracinostat Plus AZAPlacebo Plus AZA
Morphologic CR Within 6 Cycles Rate1416
Statistical analysis
  • Pracinostat Plus AZA vs Placebo Plus AZA · Cochran-Mantel-Haenszel · p = 0.7099

Adverse events

Collected over TEAEs are with onset date/time at or after start date/time of first intake of pracinostat /placebo, or AEs with onset date/time prior to start date/time of first intake of pracinostat/placebo that worsen in severity after start date/time of first intake of pracinostat /placebo, up to end of observation period. Observation period is defined as 30 days after last dose of pracinostat/placebo or start of new therapy for AML whichever occurs first up to 672 days. OS was collected up to 826 days.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Pracinostat Plus AZA121/203 (59.6%)153/201 (76.1%)197/201 (98%)
Placebo Plus AZA128/203 (63.1%)151/201 (75.1%)193/201 (96%)
Most frequent serious events
Showing 10 of 26
Most frequent serious events
EventPracinostat Plus AZAPlacebo Plus AZA
Febrile neutropeniaBlood and lymphatic system disorders60/20153/201
PneumoniaInfections and infestations25/20131/201
SepsisInfections and infestations18/20115/201
PyrexiaGeneral disorders11/20115/201
Nervous system disordersNervous system disorders10/2019/201
Metabolism and nutrition disordersMetabolism and nutrition disorders10/2015/201
Urinary tract infectionInfections and infestations9/2016/201
AnaemiaBlood and lymphatic system disorders9/2019/201
Vascular disordersVascular disorders8/2015/201
InvestigationsInvestigations8/2013/201
Most frequent other events
Showing 10 of 57
Most frequent other events
EventPracinostat Plus AZAPlacebo Plus AZA
NauseaGastrointestinal disorders85/20179/201
Respiratory, thoracic and mediastinal disordersRespiratory, thoracic and mediastinal disorders73/20185/201
InvestigationsInvestigations74/20160/201
AnaemiaBlood and lymphatic system disorders59/20169/201
Febrile neutropeniaBlood and lymphatic system disorders69/20161/201
VomitingGastrointestinal disorders64/20150/201
Nervous system disordersNervous system disorders61/20162/201
ConstipationGastrointestinal disorders56/20160/201
Musculoskeletal and connective tissue disordersMusculoskeletal and connective tissue disorders41/20159/201
Skin and subcutaneous tissue disordersSkin and subcutaneous tissue disorders56/20157/201

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Pracinostat Plus AZAPlacebo Plus AZATotal
<=18 years000
Between 18 and 65 years7512
>=65 years196198394
Age, Continuous
Age, Continuous(years)Pracinostat Plus AZAPlacebo Plus AZATotal
Mean75.4 ± 5.4875.1 ± 5.9175.3 ± 5.69
Sex: Female, Male
Sex: Female, Male(Participants)Pracinostat Plus AZAPlacebo Plus AZATotal
Female8787174
Male116116232
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Pracinostat Plus AZAPlacebo Plus AZATotal
American Indian or Alaska Native011
Asian272653
Native Hawaiian or Other Pacific Islander011
Black or African American246
White149140289
More than one race336
Unknown or Not Reported222850
Region of Enrollment
Region of Enrollment(Participants)Pracinostat Plus AZAPlacebo Plus AZATotal
Australia342963
Argentina325
Brazil101525
Czechia10818
France81018
Germany5611
Hungary121426
Italy191130
Poland211334
South Korea8715
Romania10717
Spain242852
Taiwan171835
United Kingdom61117
United States121830
Austria4610
Height
Height(cm)Pracinostat Plus AZAPlacebo Plus AZATotal
Mean165.1 ± 10.23165.7 ± 8.96165.4 ± 9.61
Smoking Habits
Smoking Habits(Participants)Pracinostat Plus AZAPlacebo Plus AZATotal
Non-smoker130121251
Ex-smoker7379152
Current smoker033
Cytogenetic Risk Category (central Lab results)
Cytogenetic Risk Category (central Lab results)(Participants)Pracinostat Plus AZAPlacebo Plus AZATotal
Intermediate109107216
Unfavorable5160111
Missing433679

4 further baseline measures are reported on the registry.

08

Study locations

138 sites
  • Mayo clinic hospital
    Phoenix, Arizona 85054, United States
  • Arizona Oncology Associates, East Valley Cancer Center
    Tempe, Arizona 85284, United States
  • University of Arizona cancer center-north campus
    Tucson, Arizona 85724, United States
  • 10666 N.Torrey Pines-Scripps Cancer Center
    La Jolla, California 92037, United States
  • UC San Diego Moores Cancer Center
    La Jolla, California 92093-0698, United States
  • Georgetown University Medical Center
    Washington, District of Columbia 20007, United States
  • Saint alphonsus Regional medical center-cancer care center
    Boise, Idaho 83706, United States
  • Loyola University Chicago
    Maywood, Illinois 60153, United States
  • Universitz of Kansas Cancer Center
    Westwood, Kansas 66205, United States
  • Norton Cancer Institute, St. Matthews Campus
    Louisville, Kentucky 40207, United States
  • Pontchartrain Cancer Center (Research Location)
    Covington, Louisiana 70433, United States
  • Rcca Md Llc
    Bethesda, Maryland 20187, United States
  • UMass Memorial medical center-university campus
    Worcester, Massachusetts 01655, United States
  • Michigan Center of Medical Research
    Farmington Hills, Michigan 48336, United States
  • Michigan State University
    Lansing, Michigan 48910, United States
  • Mayo clinic
    Rochester, Minnesota 55905, United States
  • Mercy Research
    Springfield, Missouri 65804, United States
  • 100 Mercy Way
    Joplin, Montana 64804, United States
  • Stony Brook University
    Stony Brook, New York 11794, United States
  • Duke University Medical Center-2400 Pratt Street
    Durham, North Carolina 27710, United States
  • University Hospital Cleveland Medical Center
    Cleveland, Ohio 44106, United States
  • Mercy Clinic Oncology & Hematology
    Oklahoma City, Oklahoma 73120, United States
  • Oklahoma cancer specialist and research institute
    Tulsa, Oklahoma 74146, United States
  • GHS Cancer Institute
    Greenville, South Carolina 29615, United States
  • University of Tennessee Medical Center
    Knoxville, Tennessee 37920, United States
  • VA North texas Health Care sytem,Dallas VA Medical Center div. Hematology Oncology
    Dallas, Texas 75216, United States
  • MD Anderson Cancer Center
    Houston, Texas 77030, United States
  • Emily Couric Clinical cancer center
    Charlottesville, Virginia 22908, United States
  • Swedish Cancer Institute
    Seattle, Washington 98104, United States
  • Thomas Reeve Chauncey
    Seattle, Washington 98108, United States
  • Hospital italiano de Buenos Aires
    Ciudad Autonoma de Buenos Aire, Buenos Aires C1181ACH, Argentina
  • Instituto Medico Especializado Alexander Fleming
    Ciudad Autonoma de Buenos Aire, Buenos Aires C1426ANZ, Argentina
  • hospital Italiano la Plata
    La Plata, Buenos Aires B1900AXI, Argentina
  • Sanatorio Britanico SA Paraguay 40, 3P
    Rosario, Santa Fe 2000, Argentina
  • sanatorio Allende
    Córdoba, X5000JHQ, Argentina
  • H ospi tal Privado de Cordoba
    Córdoba, X5016KEH, Argentina
  • Liverpool Hospital
    Liverpool, New South Wales 2170, Australia
  • Sunshine coast university hospital
    Birtinya, Queensland 4575, Australia
  • Royal Hobart Hospital
    Hobart, Tasmania 7000, Australia
  • The Northern hospital Pharmacy Department, Ground Floor
    Epping, Victoria 3076, Australia
  • Barwon Health, University Hospital Geelong
    Geelong, Victoria 3220, Australia
  • Austin Hospital, Clinical Trial Pharmacy
    Heidelberg, Victoria 3084, Australia
  • Liverpool hospital
    Liverpool, 2170, Australia
  • Royal Perth Hospital
    Perth, 6000, Australia
  • Prince of Wales Hospital
    Randwick, 2031, Australia
  • Krankenhaus der Elisabethinen Linz GmbH
    Linz, 4020, Austria
  • Müllner Hauptstrabe 48
    Salzburg, 5020, Austria
  • General Hospital Hietzing
    Vienna, 1130, Austria
  • Liga Paranaense de Com bate ao Cancer - Hospital Erasto Gaertner
    Curitiba, Paranà 81520-060, Brazil
  • Ce ntro de Pesquisas Hospital Amara l Ca rvalh o
    Jau, SP 17210-080, Brazil
  • Centro de Pesquisa Clinica do Hospital Santa Marcelina
    São Paulo, SP 0827-120, Brazil
  • Hospital de Cancer de Barretos
    Barretos, 1478-400, Brazil
  • Hospital Santa Casa de Belo Horizonte -Serviyo de Oncologia Clinica
    Belo Horizonte, 30.150-221, Brazil
  • Centro de Pesquisas Oncologicas - CEPON
    Florianópolis, 88034-000, Brazil
  • Hospital de ClÃ-nicas de Porto Alegre
    Porto Alegre, 90035-903, Brazil
  • Institute Nacional de Cancer Jose de Alencar Gomes da Silva-INCA
    Rio De Janeiro, 20231-050, Brazil
  • Faculdade de Medicina do ABC - Centro de Estudos e Pesquisas de Hematologia e Oncologia
    Santo André, 09060-870, Brazil
  • Hospital de Base de Sao Jose do Rio Preto
    São José Do Rio Preto, 15090-000, Brazil
  • "Fakultni nemocnice Hradec Kralove,
    Hradec Králové, 5oo 05, Czechia
  • Fakultni nemocnice Olomuc
    Olomouc, 77900, Czechia
  • Vseobecna fakultni nemocnice
    Praha 2, I28 08, Czechia
  • "Fakultni nemocnice Kralovske Vinohrady,
    Praha, 100 34, Czechia
  • Fakultni nemocnice Kralovske Vinohrady,
    Praha, 10034, Czechia
  • CHU Amiens Picardie-Site Sud-Service d'Hematologie
    Amiens, 800054, France
  • L'Hopital privé du Confluent SAS
    Nantes, 44277, France
  • CHU de Nice, Archet 1 Hospital-Hematology department
    Nice, France
  • Hospital saints Louis
    Paris, 75475, France
  • Haut-Leveque-Service d'hématologie clinique et de thérapie cellular
    Pessac, 33604, France
  • Centre Hospitalier Lyon Sud
    Pierre-Bénite, 69495, France
  • Centre Henri Becquerel
    Rouen Cedex 1, 76028, France
  • Universitatsklinikum Erlangen
    Erlangen, Bavaria 91054, Germany
  • Klinikum St. Marien Amberg
    Amberg, Bayern 92224, Germany
  • Marien Hospital Herne-Universitätsklinikum der Ruhr-Universität Bochum
    Herne, North Rhine-westphalia 44625, Germany
  • Charité-Universitätsmedizin - 1. Campus Mitte
    Berlin, 10117, Germany
  • Klinikum Chemnitz gGmbH
    Chemnitz, 09116, Germany
  • SRH Wald-Klinikum Gera GmbH
    Gera, 07548, Germany
  • Staedtisches krankenhaus kiel
    Kiel, 24116, Germany
  • Universitaet Mainz
    Mainz, 55131, Germany
  • university of Pécs
    Pécs, Baranya 7624, Hungary
  • Pecsi Egyetem I. Belgy6gyaszati Klinika
    Pécs, Baranya H-7624, Hungary
  • St. Istvan & St. Laszlo Hospital, Deapartment of Hematology and Stem Cell Transplantation
    Budapest, H-1097, Hungary
  • University of Debrecen Clinical Center
    Debrecen, H-4032, Hungary
  • Somogy Megyei Kaposi Mor Oktato Korhaz, Dep. Of Haematology
    Kaposvár, 7400, Hungary
  • Szabolcs-Szatmar-Bereg megyei Korhazak es Egyetemi Oktatokorhaz â€" Josa Andras Oktatokorhaz, Hematologiai Osztaly
    Nyiregyhaza, II-1065, Hungary
  • Ospedale San Raffaele-U.O. Ematologia e TMO
    Milano, Lombardia 20132, Italy
  • Ospedale la Maddalena, UO Oncoematologia e TMO
    Palermo, PA 90146, Italy
  • AOU Policlinico Consorziale di Bari
    Bari, 70124, Italy
  • AOU Policlinico Sant'Orsola-Malpighi
    Bologna, 40138, Italy
  • Azienda Ospedaliera-Università Careggi
    Firenze, 50134, Italy
  • Ospedale Policlinico San Martino
    Genova, 16132, Italy
  • ospedale Vito Fazzi
    Lecce, 73100, Italy
  • Azienda Ospedaliere Antonio Cardarelli,
    Naples, 80131, Italy
  • Azienda Ospedaliera Universitaria Federico II
    Napoli, 80131, Italy
  • Fondazione IRCCS policlinico San Matteo Pavia
    Pavia, 27100, Italy
  • Fondazione PTV-Policlinico Tor Vergata
    Roma, 00133, Italy
  • Policlinico Universitario Gemelli
    Roma, 00168, Italy
  • Azienda Ospedaliera Ordine Mauriziano
    Torino, 10128, Italy
  • Inje university Busan Paik Hospital
    Busan, 47392, Korea, Republic of
  • Chonnam National University Hwasun Hospital
    Hwasun, 58128, Korea, Republic of
  • Gachon University Gil medical center, div hematology
    Incheon, 21565, Korea, Republic of

Showing the first 100 of 138 sites across 16 countries.

09

References and documents

Study documents

  • Study protocol · Dec 16, 2019
  • Statistical analysis plan · Nov 11, 2020

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 10, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03151408
Lead sponsor
Helsinn Healthcare SA
Collaborators
Clinipace Worldwide
Responsible party
Sponsor
First posted
May 12, 2017
Start date
Jun 23, 2017
Primary completion
Aug 20, 2020
Completion
Aug 20, 2020
Results posted
Mar 10, 2022
Last update
Mar 10, 2022

Study contacts

Guillermo Garcia-Manero, MD
study chair · MD Anderson

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Feb 2022. You cannot join it, but the record below documents what was studied.

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