An Efficacy and Safety Study Of Pracinostat In Combination With Azacitidine In Adults With Acute Myeloid Leukemia
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ClinicalTrials.gov Identifier: NCT03151408 |
Recruitment Status :
Terminated
(The IDMC recommended to stop the study prematurely due to a lack of efficacy.)
First Posted : May 12, 2017
Results First Posted : March 10, 2022
Last Update Posted : March 10, 2022
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
Condition |
Acute Myeloid Leukemia |
Interventions |
Drug: Pracinostat Drug: Placebos Drug: Azacitidine |
Enrollment | 406 |
Recruitment Details |
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 |
Pre-assignment Details | 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. |
Arm/Group Title | Pracinostat Plus AZA | Placebo Plus AZA |
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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. Pracinostat: 60 mg capsule Azacitidine: SC or IV injection |
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. Placebos: capsule Azacitidine: SC or IV injection |
Period Title: Overall Study | ||
Started | 203 [1] | 203 [1] |
Treated | 201 | 201 |
Completed | 0 | 0 |
Not Completed | 203 | 203 |
Reason Not Completed | ||
Adverse Event | 24 | 23 |
Death | 46 | 26 |
Physician Decision | 13 | 19 |
Withdrawal by Subject | 16 | 20 |
Progressive disease | 45 | 61 |
non-compliance by patient | 1 | 0 |
other reasons | 56 | 52 |
randomized and not treated | 2 | 2 |
[1]
Screened patients
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Arm/Group Title | Pracinostat Plus AZA | Placebo Plus AZA | Total | |
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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. Pracinostat: 60 mg capsule Azacitidine: SC or IV injection |
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. Placebos: capsule Azacitidine: SC or IV injection |
Total of all reporting groups | |
Overall Number of Baseline Participants | 203 | 203 | 406 | |
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[Not Specified]
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 203 participants | 203 participants | 406 participants | |
<=18 years |
0 0.0%
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0 0.0%
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0 0.0%
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Between 18 and 65 years |
7 3.4%
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5 2.5%
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12 3.0%
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>=65 years |
196 96.6%
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198 97.5%
|
394 97.0%
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 203 participants | 203 participants | 406 participants | |
75.4 (5.48) | 75.1 (5.91) | 75.3 (5.69) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 203 participants | 203 participants | 406 participants | |
Female |
87 42.9%
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87 42.9%
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174 42.9%
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Male |
116 57.1%
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116 57.1%
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232 57.1%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 203 participants | 203 participants | 406 participants | |
American Indian or Alaska Native |
0 0.0%
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1 0.5%
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1 0.2%
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Asian |
27 13.3%
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26 12.8%
|
53 13.1%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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1 0.5%
|
1 0.2%
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Black or African American |
2 1.0%
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4 2.0%
|
6 1.5%
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White |
149 73.4%
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140 69.0%
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289 71.2%
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More than one race |
3 1.5%
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3 1.5%
|
6 1.5%
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Unknown or Not Reported |
22 10.8%
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28 13.8%
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50 12.3%
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Region of Enrollment
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 203 participants | 203 participants | 406 participants |
Australia |
34 16.7%
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29 14.3%
|
63 15.5%
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Argentina |
3 1.5%
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2 1.0%
|
5 1.2%
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Brazil |
10 4.9%
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15 7.4%
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25 6.2%
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Czechia |
10 4.9%
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8 3.9%
|
18 4.4%
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France |
8 3.9%
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10 4.9%
|
18 4.4%
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Germany |
5 2.5%
|
6 3.0%
|
11 2.7%
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Hungary |
12 5.9%
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14 6.9%
|
26 6.4%
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Italy |
19 9.4%
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11 5.4%
|
30 7.4%
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Poland |
21 10.3%
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13 6.4%
|
34 8.4%
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South Korea |
8 3.9%
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7 3.4%
|
15 3.7%
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Romania |
10 4.9%
|
7 3.4%
|
17 4.2%
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Spain |
24 11.8%
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28 13.8%
|
52 12.8%
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Taiwan |
17 8.4%
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18 8.9%
|
35 8.6%
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United Kingdom |
6 3.0%
|
11 5.4%
|
17 4.2%
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United States |
12 5.9%
|
18 8.9%
|
30 7.4%
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Austria |
4 2.0%
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6 3.0%
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10 2.5%
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Height
Mean (Standard Deviation) Unit of measure: Cm |
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Number Analyzed | 203 participants | 203 participants | 406 participants | |
165.1 (10.23) | 165.7 (8.96) | 165.4 (9.61) | ||
Smoking Habits
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 203 participants | 203 participants | 406 participants | |
Non-smoker |
130 64.0%
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121 59.6%
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251 61.8%
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Ex-smoker |
73 36.0%
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79 38.9%
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152 37.4%
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Current smoker |
0 0.0%
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3 1.5%
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3 0.7%
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Cytogenetic Risk Category (central Lab results)
[1] Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 203 participants | 203 participants | 406 participants |
Intermediate |
109 53.7%
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107 52.7%
|
216 53.2%
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Unfavorable |
51 25.1%
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60 29.6%
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111 27.3%
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Missing |
43 21.2%
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36 17.7%
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79 19.5%
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[1]
Measure Description:
Favorable: inv(16)/t(16;16)/del(16q), t(15;17) with/without secondary aberrations; t(8;21) lacking del (9q) or lacking complex karyotypes. Intermediate: Normal, +8, +6, -Y, del(12p) Unfavorable: del(5q)/-5, -7/del(7q), abn 3q, 9q, 11q, 20q, 21q, 17p, t(6;9), t(9;22) and complex karyotypes (≥ 3 unrelated abnormalities) Unknown: All other abnormalities |
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Cytogenetic Risk Category (local or central Lab results used for randomization)
[1] Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 203 participants | 203 participants | 406 participants |
Intermediate |
136 67.0%
|
135 66.5%
|
271 66.7%
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unfavorable |
67 33.0%
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68 33.5%
|
135 33.3%
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[1]
Measure Description:
Favorable: inv(16)/t(16;16)/del(16q), t(15;17) with/without secondary aberrations; t(8;21) lacking del (9q) or lacking complex karyotypes. Intermediate: Normal, +8, +6, -Y, del(12p) Unfavorable: del(5q)/-5, -7/del(7q), abn 3q, 9q, 11q, 20q, 21q, 17p, t(6;9), t(9;22) and complex karyotypes (≥ 3 unrelated abnormalities) Unknown: All other abnormalities |
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ECOG PS (used for randomization)
[1] Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 203 participants | 203 participants | 406 participants |
Grade 0-1 |
113 55.7%
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114 56.2%
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227 55.9%
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Grade 2 |
90 44.3%
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89 43.8%
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179 44.1%
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[1]
Measure Description:
Grade and Descriptions 0 - Fully active, able to carry on all pre-disease performance without restriction.
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ECOG PS (at cycle 1 Day 1)
[1] Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 203 participants | 203 participants | 406 participants |
Grade 0-1 |
114 56.2%
|
110 54.2%
|
224 55.2%
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Grade 2 |
85 41.9%
|
88 43.3%
|
173 42.6%
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Grade 3 |
0 0.0%
|
1 0.5%
|
1 0.2%
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|
Missing |
4 2.0%
|
4 2.0%
|
8 2.0%
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[1]
Measure Description:
Grade and Descriptions 0 - Fully active, able to carry on all pre-disease performance without restriction.
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Renal impairment
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 203 participants | 203 participants | 406 participants |
Normal or high: ≥ 90 mL/mg/1.73 m2 |
24 11.8%
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28 13.8%
|
52 12.8%
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Mildly decreased: 60-89 mL/mg/1.73 m2 |
118 58.1%
|
102 50.2%
|
220 54.2%
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Mildly to moderately decreased: 45-59 mL/mg/1.73 m2 |
39 19.2%
|
41 20.2%
|
80 19.7%
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Moderately to severely decreased: 30-44 mL/mg/1.73 m2 |
18 8.9%
|
28 13.8%
|
46 11.3%
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Severely decreased: 15-29 mL/mg/1.73 m2 |
1 0.5%
|
1 0.5%
|
2 0.5%
|
|
Missing |
3 1.5%
|
3 1.5%
|
6 1.5%
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Name/Title: | Francesco Scarci |
Organization: | Helsinn Healthcare SA |
Phone: | +41 (0)91 985 1946 |
EMail: | francesco.scarci@helsinn.com |
Responsible Party: | Helsinn Healthcare SA |
ClinicalTrials.gov Identifier: | NCT03151408 |
Other Study ID Numbers: |
PRAN-16-52 |
First Submitted: | May 5, 2017 |
First Posted: | May 12, 2017 |
Results First Submitted: | August 20, 2021 |
Results First Posted: | March 10, 2022 |
Last Update Posted: | March 10, 2022 |