18 and older, any sex, with Myelodysplastic Syndromes. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Overall Response Rate (ORR)Primary· 36 months
Percentage of subjects with confirmed complete remission (CR), partial remission (PR) and marrow CR, as per modified International Working Group (IWG) criteria:
CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hemoglobin (Hb) ≥11 g/dL; Platelets ≥100 × 10\^9/L; Neutrophils ≥1.0 × 10\^9/L; Blasts 0%.
PR: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by ≥ 50% over pre-treatment but still \>5%; Cellularity and morphology not relevant Marrow CR: Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pre-treatment
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
35.9
24.3 – 48.9
Complete Response (CR) RateSecondary· 36 months
Percentage of subjects with confirmed CR (i.e., 2 CRs at least 28 days apart) as per modified IWG criteria:
CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hb ≥11 g/dL; Platelets ≥100 × 109/L; Neutrophils ≥1.0 × 109/L; Blasts 0%.
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
Percentage of subjects demonstrating major hematologic improvement according to modified IWG:
Erythroid response (pre-treatment, \<11 g/dL): Hb increase by ≥1.5 g/dL; Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of ≤9.0 g/dL pre-treatment will count in the RBC transfusion response evaluation.
Platelet response (pre-treatment, \<100 × 10\^9/L): Absolute increase of ≥30 × 10\^9/L for patients starting with \>20 × 10\^9/
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
71.4
58.7 – 82.1
Clinical Benefit RateSecondary· 36 months
Percentage of subjects with confirmed CR, PR, Marrow CR, and HI with clinical benefit rate, defines as rate of CR + PR + HI + Marrow CR. All subjects who achieve hematologic CR, PR, marrow CR, or hematologic improvement on the erythrocytic lineage per modified IWG response criteria will be considered responders
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
78.1
66.0 – 87.5
Rate of Cytogenetic CRSecondary· 36 months
Percentage of subjects with confirmed CR by cytogenetic assessment. Complete cytogenetic response is defined per modified IWG response criteria:
Complete: Disappearance of the chromosomal abnormality without appearance of new ones Partial: At least 50% reduction of the chromosomal abnormality
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
46.9
34.3 – 59.8
Duration of Response (DoR)Secondary· 36 months
For subjects who have achieved CR, PR, Marrow CR, or HI, DoR is defined as the time from the initial determination of response to the time of disease progression or death on study, whichever occurs first.
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
15.67
9.00 – 22.11
Rate of Leukemic TransformationSecondary· 6 months
Percentage of subjects with leukemic transformation at landmark time point of 6 months
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
5.4
1.8 – 15.8
Event-free Survival (EFS)Secondary· 36 months
time from the first day of study drug administration (Day 1) to failure or death from any cause
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
time from the first day of study drug administration (Day 1) to disease recurrence or progression as defined by the IWG criteria, or death on study:
Disease progression for subjects with:
Less than 5% blasts: ≥50% increase in blasts to \>5% blasts 5%-10% blasts: ≥50% increase to \>10% blasts 10%-20% blasts: ≥50% increase to \>20% blasts 20%-30% blasts: ≥50% increase to \>30% blasts
Any of the following:
At least 50% decrement from maximum remission/response in granulocytes or platelets Reduction in Hb by ≥2 g/dL Transfusion dependence
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
16.43
11.33 – 22.57
Overall Survival (OS)Secondary· form day 1 to death on study, assessed up to 36 months
time from the first day of study drug administration (Day 1) to death on study
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
23.56
16.49 – 30.26
Rate of Leukemic TransformationSecondary· 12 months
Percentage of subjects with leukemic transformation at landmark time point of 12 months
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
9.5
3.3 – 25.4
Rate of Leukemic TransformationSecondary· 18 months
Percentage of subjects with leukemic transformation at landmark time point of 18 months
Group
Value
95% CI
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
13.8
5.5 – 32.4
Adverse events — posted to ClinicalTrials.gov
Time frame: All AEs regardless of seriousness or relationship to pracinostat or azacitidine, from the start of study drug treatment until 30 calendar days after discontinuation or completion of treatment as defined by the clinical study for that subject, have been recorded..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Stage 1a and 1b Open-label Pracinostat Plus Azacitidine
This is a Phase 2, two-stage study of the safety and efficacy of pracinostat in combination with azacitidine in patients with IPSS-R high and very high risk myelodysplastic syndrome (MDS) who are previously untreated with hypomethylating agents (HMAs). Enrollment in this study will be limited to high/very high risk MDS because this group represents the highest unmet need, with median survival of less than 18 months.
Stage 1a will be conducted as an open-label single arm in up to 40 subjects to assess if this regimen with a lower pracinostat dose results in a discontinuation rate that meets a predefined threshold and in efficacy that justifies expansion of enrollment into Stage 1b.
A discontinuation rate of approximately ≤10% in Stage 1a, a rate comparable to that observed with azacitidine alone in study MEI-003 (NCT01873703), supports expansion into Stage 1b.
Stage 1b will be conducted as expansion of stage 1a. Approximately 20 additional subjects will be enrolled. Study drugs should be continued until disease progression or intolerable toxicity. It is important to note that the median time to achieving a response with azacitidine alone is 4 to 5 months. Furthermore, the median time to achieving a Complete Response (CR) in study MEI-003 (NCT01873703) was 4 cycles. Therefore, early (\<6 months) discontinuation of trial therapy for 'no response' should be avoided. The Medical Monitor should be contacted prior to discontinuing a subject from the study to discuss the rationale for discontinuation.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03151408 — An Efficacy and Safety Study Of Pracinostat In Combination With Azacitidine In Adults With Acute Myeloid Leukemia
· Phase 3
· terminated
NCT02267278 — Ruxolitinib and Pracinostat Combination Therapy for Patients With Myelofibrosis (MF)
· Phase 2
· completed
NCT01912274 — Safety and Efficacy Study of Pracinostat With Azacitadine in Elderly Patients With Newly Diagnosed Acute Myeloid Leukemi
· Phase 2
· completed
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Trials by the same sponsor.
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· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Helsinn Healthcare SA
Last refreshed: 2 March 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03151304.