Clinical Trials List
Protocol NumberPRAN-16-52
NCT Number(ClinicalTrials.gov Identfier)NCT03151408
Not yet recruiting
2017-08-01 - 2022-12-31
Phase III
Terminated7
ICD-10C92.A0
Acute myeloid leukemia with multilineage dysplasia, not having achieved remission
A Phase III, Double-Blind, Placebo-Controlled, Multicenter, Randomized Study Of Pracinostat In Combination With Azacitidine In Patients ≥18 Years With Newly Diagnosed Acute Myeloid Leukemia Unfit For Standard Induction Chemotherapy
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Trial Applicant
Clinipace Taiwan Co., Ltd
-
Sponsor
Helsinn Healthcare SA
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Trial scale
Multi-Regional Multi-Center
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Update
2026/04/01
Investigators and Locations
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Co-Principal Investigator
- Ming-Hung Tsai Division of Hematology & Oncology
- Che-Hung Lin Division of Hematology & Oncology
- Ching-Chan Lin Division of Hematology & Oncology
- Tzu-Ting Chen Division of Hematology & Oncology
- Ming-Yu Lien Division of Hematology & Oncology
- Chi-Ching Chen Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Co-Principal Investigator
- Tung-Liang Lin Division of Hematology & Oncology
- Po-Nan Wang Division of Hematology & Oncology
- 高小雯 Division of Hematology & Oncology
- Ming-Chung Kao Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Stop recruiting
The Actual Total Number of Participants Enrolled
0 Stop recruiting
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Co-Principal Investigator
- Jeng-Shiun Du Division of Hematology & Oncology
- 蘇裕傑 Division of Hematology & Oncology
- Hui-Ching Wang Division of Hematology & Oncology
- 林明慧 Division of Hematology & Oncology
- 蔡郁棻 Division of Hematology & Oncology
- Yi-Chang Liu Division of Hematology & Oncology
- 許瑞峰 Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Co-Principal Investigator
Audit
CRO
Principal Investigator
Co-Principal Investigator
- Hui-Hua Hsiao 未分科
Audit
None
Co-Principal Investigator
- Wen-Chien Chou Division of General Internal Medicine
- Huai-Hsuan Huang Division of General Internal Medicine
- 林建廷 Division of General Internal Medicine
- CHENG-HONG TSAI Division of General Internal Medicine
- Jih-Luh Tang Division of General Internal Medicine
- 劉家豪 Division of General Internal Medicine
- Tai-Chung Huang Division of General Internal Medicine
- - - Division of General Internal Medicine
- Chieh-Lung Cheng Division of General Internal Medicine
- - - Division of General Internal Medicine
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Audit
None
Condition/Disease
Acute Myeloid Leukemia (AML)
Objectives
Primary Objective:
To show superiority in terms of overall survival (OS) of treatment with pracinostat (Group A – experimental group) versus placebo (Group B – control group) in patients treated with AZA as background therapy
Test Drug
Pracinostat
Active Ingredient
Pracinostat
Dosage Form
capsule
Dosage
60mg/capsule, 45mg /capsule
Endpoints
Primary Outcome Measures :
Estimate efficacy rate [ Time Frame: 1 year ]
To show superiority in terms of overall survival (OS) of treatment with pracinostat (Group A - experimental group) versus placebo (Group B - control group) in patients treated with AZA as background therapy
Estimate efficacy rate [ Time Frame: 1 year ]
To show superiority in terms of overall survival (OS) of treatment with pracinostat (Group A - experimental group) versus placebo (Group B - control group) in patients treated with AZA as background therapy
Inclution Criteria
Inclusion Criteria:
Patients must meet the following criteria for study entry:
1. Male or female patient ≥ 18 years of age with newly diagnosed,
histologically confirmed, AML including de novo, secondary to antecedent
hematologic disorders, or treatment-related disease with intermediate or
unfavorable-risk cytogenetics (Appendix B)
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:
a. An ECOG performance status of 2
b. 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:
a. Total bilirubin ≤ 2 × upper limit of normal (ULN) or < 3 x ULN
for patients with Gilbert-Meulengracht Syndrome
b. 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 (Appendix J)
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 (Appendix J)
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.
Patients must meet the following criteria for study entry:
1. Male or female patient ≥ 18 years of age with newly diagnosed,
histologically confirmed, AML including de novo, secondary to antecedent
hematologic disorders, or treatment-related disease with intermediate or
unfavorable-risk cytogenetics (Appendix B)
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:
a. An ECOG performance status of 2
b. 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:
a. Total bilirubin ≤ 2 × upper limit of normal (ULN) or < 3 x ULN
for patients with Gilbert-Meulengracht Syndrome
b. 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 (Appendix J)
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 (Appendix J)
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:
Patients who meet any of the following criteria will be excluded from study
entry:
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 (Appendix
E)
6. Evidence of AML central nervous system (CNS) involvement
7. Previous chemotherapy for AML except for the following, which are
allowed:
a. Hydroxyurea for cytoreduction
b. 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
Patients who meet any of the following criteria will be excluded from study
entry:
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 (Appendix
E)
6. Evidence of AML central nervous system (CNS) involvement
7. Previous chemotherapy for AML except for the following, which are
allowed:
a. Hydroxyurea for cytoreduction
b. 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
The Estimated Number of Participants
-
Taiwan
28 participants
-
Global
500 participants
