Trial Outcomes & Findings for Open Label Study of Subcutaneous Homoharringtonine (Omacetaxine Mepesuccinate) in Patients With Advanced CML (NCT NCT00462943)
NCT ID: NCT00462943
Last Updated: 2021-12-28
Results Overview
Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last \>= 8 weeks to be considered meaningful. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
100 participants
Primary outcome timeframe
Day 1 up to 6 months
Results posted on
2021-12-28
Participant Flow
Participant milestones
| Measure |
CML: Chronic Phase
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|
|
Overall Study
STARTED
|
46
|
31
|
23
|
|
Overall Study
COMPLETED
|
1
|
1
|
0
|
|
Overall Study
NOT COMPLETED
|
45
|
30
|
23
|
Reasons for withdrawal
| Measure |
CML: Chronic Phase
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|
|
Overall Study
Failure to achieve a response
|
5
|
5
|
1
|
|
Overall Study
Adverse Event
|
6
|
4
|
1
|
|
Overall Study
Protocol Violation
|
1
|
0
|
0
|
|
Overall Study
Request of Patient, PI, Sponsor or RA
|
8
|
2
|
4
|
|
Overall Study
Disease Progression
|
15
|
15
|
9
|
|
Overall Study
Death
|
5
|
2
|
7
|
|
Overall Study
Stem Cell Transplant
|
1
|
1
|
0
|
|
Overall Study
Bone Marrow Transplant
|
1
|
0
|
0
|
|
Overall Study
Required Lymphocyte Infusion
|
1
|
0
|
0
|
|
Overall Study
Transplant - not specified
|
1
|
0
|
1
|
|
Overall Study
Physician Decision
|
1
|
0
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
0
|
Baseline Characteristics
Open Label Study of Subcutaneous Homoharringtonine (Omacetaxine Mepesuccinate) in Patients With Advanced CML
Baseline characteristics by cohort
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total
n=100 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
58 years
n=5 Participants
|
56 years
n=7 Participants
|
57 years
n=5 Participants
|
57 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
20 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
41 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
59 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
31 participants
n=5 Participants
|
17 participants
n=7 Participants
|
12 participants
n=5 Participants
|
60 participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Black
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
5 participants
n=5 Participants
|
11 participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
3 participants
n=5 Participants
|
2 participants
n=7 Participants
|
1 participants
n=5 Participants
|
6 participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Asian
|
7 participants
n=5 Participants
|
7 participants
n=7 Participants
|
4 participants
n=5 Participants
|
18 participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
5 participants
n=4 Participants
|
|
Height
|
167.7 centimeter
n=5 Participants
|
167.5 centimeter
n=7 Participants
|
171.0 centimeter
n=5 Participants
|
167.6 centimeter
n=4 Participants
|
|
Weight
|
79.5 kilograms
n=5 Participants
|
67.4 kilograms
n=7 Participants
|
74.0 kilograms
n=5 Participants
|
74.2 kilograms
n=4 Participants
|
|
Body Surface Area (BSA)
|
1.9 meters^2
n=5 Participants
|
1.7 meters^2
n=7 Participants
|
1.9 meters^2
n=5 Participants
|
1.9 meters^2
n=4 Participants
|
|
New York Heart Association (NYHA) Classification
Class I
|
44 participants
n=5 Participants
|
29 participants
n=7 Participants
|
23 participants
n=5 Participants
|
96 participants
n=4 Participants
|
|
New York Heart Association (NYHA) Classification
Class II
|
2 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
New York Heart Association (NYHA) Classification
Class III
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
|
New York Heart Association (NYHA) Classification
Class IV
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
|
New York Heart Association (NYHA) Classification
Not available
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
0 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Eastern Cooperative Oncology Group Performance Status
Grade 0
|
27 participants
n=5 Participants
|
7 participants
n=7 Participants
|
6 participants
n=5 Participants
|
40 participants
n=4 Participants
|
|
Eastern Cooperative Oncology Group Performance Status
Grade 1
|
17 participants
n=5 Participants
|
19 participants
n=7 Participants
|
11 participants
n=5 Participants
|
47 participants
n=4 Participants
|
|
Eastern Cooperative Oncology Group Performance Status
Grade 2
|
2 participants
n=5 Participants
|
5 participants
n=7 Participants
|
5 participants
n=5 Participants
|
12 participants
n=4 Participants
|
|
Eastern Cooperative Oncology Group Performance Status
Grade 3
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Time from Initial Chronic Myeloid Leukemia (CML) Diagnosis
|
74.4 months
n=5 Participants
|
83.5 months
n=7 Participants
|
68.7 months
n=5 Participants
|
74.0 months
n=4 Participants
|
PRIMARY outcome
Timeframe: Day 1 up to 6 monthsPopulation: Intent to treat population
Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last \>= 8 weeks to be considered meaningful. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population
|
67.4 percentage of participants
Interval 51.98 to
Lower limit of the 2-sided 95% CI is reported
|
25.8 percentage of participants
Interval 11.86 to
Lower limit of the 2-sided 95% CI is reported
|
8.7 percentage of participants
Interval 1.07 to
Lower limit of the 2-sided 95% CI is reported
|
41.0 percentage of participants
Interval 31.26 to
Lower limit of the 2-sided 95% CI is reported
|
PRIMARY outcome
Timeframe: Day 1 up to 9 monthsPopulation: Intent to treat population
Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available. Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows \>0% - 35% Ph+ cells. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants Achieving a Major Cytogenetic Response by Subpopulation and Total Population
|
21.7 percentage of participants
Interval 10.95 to
Lower limit of the 2-sided 95% CI is reported
|
3.2 percentage of participants
Interval 0.08 to
Lower limit of the 2-sided 95% CI is reported
|
0 percentage of participants
Interval 0.0 to
Lower limit of the 2-sided 95% CI is reported
|
11 percentage of participants
Interval 5.62 to
Lower limit of the 2-sided 95% CI is reported
|
PRIMARY outcome
Timeframe: up to 4 yearsPopulation: Intent to treat
TEAE are any untoward events that were newly occurring or worsening from Baseline. Treatment related toxicity was considered by the investigator to be unrelated, possibly, probably or unknown related to study drug. Severity was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v3.0 on the following scale: Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe, Grade 4 = life-threatening, Grade 5 = death. A serious adverse event (SAE) is any untoward medical occurrence that is fatal or life-threatening; results in persistent or significant disability or incapacity; requires or prolongs in-patient hospitalization; is a congenital anomaly/birth defect in the offspring of a patient; and conditions not included in the above that may jeopardize the patient or may require intervention to prevent one of the outcomes listed above. A participant is only counted once in each category (at worst severity or strongest relationship).
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Deaths during study (outcome of SAE)
|
6 participants
|
6 participants
|
11 participants
|
23 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Worst severity: Grade 3
|
18 participants
|
6 participants
|
4 participants
|
28 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Worst severity: Grade 5
|
6 participants
|
6 participants
|
11 participants
|
23 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Worst severity: Grade 4
|
17 participants
|
15 participants
|
6 participants
|
38 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
>=1 TEAE
|
46 participants
|
31 participants
|
23 participants
|
100 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
>= 1 SAE
|
26 participants
|
19 participants
|
18 participants
|
63 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Worst severity: Grade 1
|
1 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Worst severity: Grade 2
|
4 participants
|
4 participants
|
2 participants
|
10 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Relation to drug: Unrelated
|
4 participants
|
4 participants
|
7 participants
|
15 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Relation to drug: Possibly
|
5 participants
|
11 participants
|
7 participants
|
23 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Relation to drug: Probably
|
36 participants
|
14 participants
|
8 participants
|
58 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Relation to drug: Unknown
|
1 participants
|
2 participants
|
1 participants
|
4 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
With hematologic toxicity
|
36 participants
|
22 participants
|
13 participants
|
71 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Discontinued treatment due to AE
|
10 participants
|
11 participants
|
6 participants
|
27 participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Deaths during study or follow-up
|
35 participants
|
25 participants
|
21 participants
|
71 participants
|
SECONDARY outcome
Timeframe: Day 1 up to Month 9Population: Intent to treat
Cytogenetic response categories: * Complete: 0% Ph+ cells * Partial: \>0%-35% Ph+ cells * Minor: \>35%-65% Ph+ cells * Minimal: \>65%-95% Ph+ cells * No Response: \>95% Ph+ cells * Unevaluable: \<20 metaphases were examined and/or response could not be assigned
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Complete
|
4.3 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
2.0 percentage of participants
|
|
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Partial
|
17.4 percentage of participants
|
3.2 percentage of participants
|
0 percentage of participants
|
9.0 percentage of participants
|
|
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Minor
|
8.7 percentage of participants
|
9.7 percentage of participants
|
0 percentage of participants
|
7.0 percentage of participants
|
|
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Minimal
|
6.5 percentage of participants
|
6.5 percentage of participants
|
4.3 percentage of participants
|
6.0 percentage of participants
|
|
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
No Response
|
39.1 percentage of participants
|
61.3 percentage of participants
|
30.4 percentage of participants
|
44.0 percentage of participants
|
|
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Unevaluable
|
23.9 percentage of participants
|
19.4 percentage of participants
|
65.2 percentage of participants
|
32.0 percentage of participants
|
SECONDARY outcome
Timeframe: Day 1 up to Month 6Population: Intent to treat population of participants who had evaluable samples. Participants with no data for these analyses either had degraded samples or the samples were missing.
MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region \[BCR\] gene and Abelson proto-oncogene \[ABL\] genes). This analysis used the standard gene GUS. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.
Outcome measures
| Measure |
CML: Chronic Phase
n=22 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=18 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=4 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=44 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene GUS
|
13.6 percentage of participants
Interval 2.9 to 34.9
|
0 percentage of participants
no participants met criteria
|
0 percentage of participants
no participants met criteria
|
6.8 percentage of participants
Interval 1.4 to 18.7
|
SECONDARY outcome
Timeframe: Day 1 up to Month 6Population: Intent to treat population of participants who had evaluable samples. Participants with no data for these analyses either had degraded samples or the samples were missing.
MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region \[BCR\] gene and Abelson proto-oncogene \[ABL\] genes). This analysis used the standard gene ABL. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.
Outcome measures
| Measure |
CML: Chronic Phase
n=38 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=11 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=72 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene ABL
|
10.5 percentage of participants
Interval 2.9 to 24.8
|
4.3 percentage of participants
Interval 0.1 to 22.0
|
0 percentage of participants
no participants met criteria
|
6.9 percentage of participants
Interval 2.3 to 15.5
|
SECONDARY outcome
Timeframe: Day 1 up to Month 6Population: Intent to treat
Complete Response (CHR) * Chronic phase must last at least 8 weeks: WBC \<10\*10\^9/liter, platelets \<450\*10\^9/liter, myelocytes + metamyelocytes \<5% in blood, no blasts or promyelocytes in blood, \<20% basophils in peripheral blood, no extramedullary involvement. * Accelerated and Blast phase must last at least 4 weeks: absolute neutrophil count 1.5\*10\^9/liter, platelets 100\*10\^9/liter, no blood blasts, bone marrow blasts \<5%, no extramedullary disease. Partial Response - CHR plus one or more of the following: * Persistence of splenomegaly with a reduction of ≥50% from pre-treatment * Platelets \> 450\*10\^9/L * Presence of immature cells in the peripheral blood * 5% to 25% blasts in the bone marrow * If extra-medullary disease pre-treatment, reduction by ≥50% Hematologic Improvement - CHR, except allowing persistent thrombocytopenia (\<100\*10\^9/L), and a few immature cells No evidence of leukemia: Morphologic leukemia-free state, defined as \<5% bone marrow blasts.
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants in Each Hematologic Response Category
Complete response
|
67.4 percentage of participants
|
19.4 percentage of participants
|
8.7 percentage of participants
|
39.0 percentage of participants
|
|
Percentage of Participants in Each Hematologic Response Category
Partial response
|
0 percentage of participants
|
3.2 percentage of participants
|
0 percentage of participants
|
1.0 percentage of participants
|
|
Percentage of Participants in Each Hematologic Response Category
Hematologic improvement
|
0 percentage of participants
|
9.7 percentage of participants
|
4.3 percentage of participants
|
4.0 percentage of participants
|
|
Percentage of Participants in Each Hematologic Response Category
Return to chronic phase
|
NA percentage of participants
Participants enrolled in chronic phase and therefore cannot respond (improve) to chronic phase.
|
6.5 percentage of participants
|
0 percentage of participants
|
2.0 percentage of participants
|
|
Percentage of Participants in Each Hematologic Response Category
No evidence of leukemia
|
NA percentage of participants
An overall hematologic response for chronic phase participants only included a complete hematologic response.
|
0 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants in Each Hematologic Response Category
No response
|
21.7 percentage of participants
|
58.1 percentage of participants
|
78.3 percentage of participants
|
46.0 percentage of participants
|
|
Percentage of Participants in Each Hematologic Response Category
Unevaluable
|
10.9 percentage of participants
|
3.2 percentage of participants
|
8.7 percentage of participants
|
8.0 percentage of participants
|
SECONDARY outcome
Timeframe: Day 1 up to Month 9Population: Intent to treat population of study participants who had extramedullary disease at baseline
Clinical response was defined by disease phase and based on evaluations by the independent Data Monitoring Committee (DMC). Chronic Phase subgroup: achieving a complete hematologic response and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed). Accelerated Phase and Blast Phase subgroups: achieving complete hematologic response, no evidence of leukemia, return to chronic phase, and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed).
Outcome measures
| Measure |
CML: Chronic Phase
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=2 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response
Clinical response
|
—
|
—
|
0 percentage of participants
|
—
|
|
Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response
Unevaluable
|
—
|
—
|
50 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Day 1 up to Month 9Population: Intent to treat population of participants with post-baseline assessment of T315I mutated BCR ABL
Summarization is based on the best of the individual response assessments. Not assessable indicates that the participant either had no baseline assessment or the % mutation could not be determined in the post-baseline assessment(s).
Outcome measures
| Measure |
CML: Chronic Phase
n=38 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=11 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=72 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
100% reduction
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
|
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
75-99% reduction
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
|
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
50-74% reduction
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
|
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
25-49% reduction
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
9.1 percentage of participants
Interval 0.3 to 52.7
|
1.4 percentage of participants
Interval 0.0 to 9.1
|
|
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
1-24% reduction
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
0 percentage of participants
Interval 0.0 to 0.0
|
|
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
0% reduction
|
78.9 percentage of participants
Interval 76.4 to 100.0
|
91.3 percentage of participants
Interval 83.9 to 100.0
|
63.3 percentage of participants
Interval 47.4 to 99.7
|
80.6 percentage of participants
Interval 79.9 to 100.0
|
|
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
Not assessable
|
21.1 percentage of participants
95% CI not calculated for a non-response category.
|
8.7 percentage of participants
95% CI not calculated for a non-response category.
|
27.3 percentage of participants
95% CI not calculated for a non-response category.
|
18.1 percentage of participants
95% CI not calculated for a non-response category.
|
SECONDARY outcome
Timeframe: Day 1 up to Month 6Population: Intent to treat population of participants who had a response to treatment
Induction therapy was administered for 14 consecutive days for each 28 days cycle, for up to 6 cycles. All treatment arms were given omacetaxine mepesuccinate via subcutaneous (SC) administration at 1.25 mg/m\^2 twice a day (BID) for the 14 consecutive days.
Outcome measures
| Measure |
CML: Chronic Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=8 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=2 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=41 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Number of Treatment Cycles Needed to Achieve Best Hematologic Response
|
1.0 treatment cycles
Interval 1.0 to 5.0
|
2.0 treatment cycles
Interval 1.0 to 4.0
|
2.0 treatment cycles
Interval 1.0 to 3.0
|
1.0 treatment cycles
Interval 1.0 to 5.0
|
SECONDARY outcome
Timeframe: Day 1 up to Month 9Population: Intent to treat population of participants who had a cytogenetic response
Outcome measures
| Measure |
CML: Chronic Phase
n=17 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=6 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=1 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=24 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Number of Treatment Cycles Needed to Achieve Best Cytogenetic Response
|
2.0 treatment cycles
Interval 1.0 to 9.0
|
1.5 treatment cycles
Interval 1.0 to 4.0
|
1.0 treatment cycles
Interval 1.0 to 1.0
|
2.0 treatment cycles
Interval 1.0 to 9.0
|
SECONDARY outcome
Timeframe: Day 1 up to Month 6Population: Intent to treat population.
Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last \>= 8 weeks to be considered meaningful.
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Kaplan-Meier Estimates for Time to Onset of Best Hematologic Response
|
1.38 months
Interval 0.49 to 1.84
|
NA months
Interval 4.14 to
A large percentage of participants were censored.
|
NA months
A large percentage of participants were censored.
|
5.03 months
Interval 3.13 to
A large percentage of participants were censored.
|
SECONDARY outcome
Timeframe: Day 1 up to Month 9Population: Intent to treat population.
Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day. Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available. Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows \>0% - 35% Ph+ cells.
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Kaplan-Meier Estimates for Time to Onset of Best Cytogenetic Response
|
NA months
A large percentage of participants were censored.
|
NA months
A large percentage of participants were censored.
|
NA months
A large percentage of participants were censored.
|
—
|
SECONDARY outcome
Timeframe: up to four yearsPopulation: Intent to treat population of participants who had a response
Duration of response is defined as the time from first reported date of hematologic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.
Outcome measures
| Measure |
CML: Chronic Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=8 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=2 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Kaplan-Meier Estimates for Duration of Best Hematologic Response
|
7.01 months
Interval 1.41 to 59.51
|
5.47 months
Interval 2.4 to 19.7
|
2.67 months
Interval 1.74 to 3.59
|
—
|
SECONDARY outcome
Timeframe: up to four yearsPopulation: Intent to treat population of participants who had a response
Duration of response is defined as the time from first reported date of cytogenetic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.
Outcome measures
| Measure |
CML: Chronic Phase
n=10 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=1 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Kaplan-Meier Estimates for Duration of Best Cytogenetic Response
|
6.01 months
Interval 0.92 to 51.94
|
0.07 months
Interval 0.07 to 0.07
|
—
|
—
|
SECONDARY outcome
Timeframe: up to 4 yearsPopulation: Intent to treat
Time to disease progression is defined as the time from the initiation of treatment until the onset date of death, the development of CML accelerated phase or blast phase, or the loss of complete hematologic response or major cytogenetic response, whichever came first. Participants were censored only if they did not have progression or if they discontinued treatment for reasons other than AE, progression or death.
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Kaplan-Meier Estimates for Time to Disease Progression
|
7.50 months
Interval 5.86 to 9.64
|
4.84 months
Interval 3.55 to 6.81
|
2.04 months
Interval 1.35 to 2.73
|
4.38 months
Interval 3.55 to 6.45
|
SECONDARY outcome
Timeframe: up to 4 yearsPopulation: Intent to treat
Overall survival is defined as the time from the initiation of treatment until death from any cause or the last day of participant contact or evaluation for participants that were lost to follow-up. Participants were censored t the last recorded contract or evaluation when a participant was alive at time of analysis. A quarterly phone survey was conducted to collect survival data for participants who discontinued from the study.
Outcome measures
| Measure |
CML: Chronic Phase
n=46 Participants
Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Accelerated Phase
n=31 Participants
Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
CML: Blast Phase
n=23 Participants
Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
Total Participants
n=100 Participants
Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|---|---|---|
|
Kaplan-Meier Estimates for Overall Survival
|
33.91 months
Interval 23.75 to
A large percentage of participants were censored
|
17.27 months
Interval 10.56 to 28.45
|
3.52 months
Interval 2.57 to 5.16
|
17.27 months
Interval 14.24 to 28.03
|
Adverse Events
Omacetaxine
Serious events: 63 serious events
Other events: 99 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Omacetaxine
n=100 participants at risk
Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|
|
Blood and lymphatic system disorders
Pancytopenia
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
6.0%
6/100 • Number of events 10 • up to 4 years
|
|
Cardiac disorders
Acute coronary syndrome
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Cardiac disorders
Atrial fibrillation
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Cardiac disorders
Cardiac failure congestive
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Cardiac disorders
Pericardial effusion
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Cardiac disorders
Pericarditis
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Cardiac disorders
Tachycardia
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Ear and labyrinth disorders
Vertigo
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Gastrointestinal disorders
Diarrhoea
|
4.0%
4/100 • Number of events 4 • up to 4 years
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Gastrointestinal disorders
Pancreatitis
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Gastrointestinal disorders
Stomatitis
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Blood and lymphatic system disorders
Anaemia
|
3.0%
3/100 • Number of events 3 • up to 4 years
|
|
Blood and lymphatic system disorders
Anaemia haemolytic autoimmune
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Blood and lymphatic system disorders
Bone marrow failure
|
5.0%
5/100 • Number of events 6 • up to 4 years
|
|
Blood and lymphatic system disorders
Febrile bone marrow aplasia
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
9.0%
9/100 • Number of events 14 • up to 4 years
|
|
Blood and lymphatic system disorders
Leukocytosis
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Blood and lymphatic system disorders
Neutropenia
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
General disorders
Chest pain
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
General disorders
Death
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
General disorders
Disease progression
|
8.0%
8/100 • Number of events 8 • up to 4 years
|
|
General disorders
Fatigue
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
General disorders
Localised oedema
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
General disorders
Multi-organ failure
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
General disorders
Pyrexia
|
4.0%
4/100 • Number of events 5 • up to 4 years
|
|
Hepatobiliary disorders
Cholelithiasis
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Infections and infestations
Bacteraemia
|
1.0%
1/100 • Number of events 4 • up to 4 years
|
|
Infections and infestations
Catheter sepsis
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Infections and infestations
Injection site infection
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Infections and infestations
Lung infection
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Infections and infestations
Pneumonia
|
5.0%
5/100 • Number of events 5 • up to 4 years
|
|
Infections and infestations
Sepsis
|
3.0%
3/100 • Number of events 4 • up to 4 years
|
|
Infections and infestations
Tonsillitis
|
1.0%
1/100 • Number of events 2 • up to 4 years
|
|
Infections and infestations
Viral infection
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
1.0%
1/100 • Number of events 2 • up to 4 years
|
|
Metabolism and nutrition disorders
Dehydration
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Metabolism and nutrition disorders
Failure to thrive
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
3.0%
3/100 • Number of events 3 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Blast cell crisis
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Chronic myeloid leukaemia
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukaemia
|
2.0%
2/100 • Number of events 2 • up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastatic neoplasm
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Myelodysplastic syndrome
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Nervous system disorders
Cerebral haemorrhage
|
3.0%
3/100 • Number of events 3 • up to 4 years
|
|
Nervous system disorders
Cerebral ischaemia
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Nervous system disorders
Convulsion
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Psychiatric disorders
Mood altered
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Acute pulmonary oedema
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
|
Vascular disorders
Hypotension
|
1.0%
1/100 • Number of events 1 • up to 4 years
|
Other adverse events
| Measure |
Omacetaxine
n=100 participants at risk
Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m\^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
51.0%
51/100 • Number of events 132 • up to 4 years
|
|
Blood and lymphatic system disorders
Bone marrow failure
|
6.0%
6/100 • Number of events 7 • up to 4 years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
17.0%
17/100 • Number of events 24 • up to 4 years
|
|
Blood and lymphatic system disorders
Leukocytosis
|
7.0%
7/100 • Number of events 10 • up to 4 years
|
|
Blood and lymphatic system disorders
Leukopenia
|
13.0%
13/100 • Number of events 55 • up to 4 years
|
|
Blood and lymphatic system disorders
Lymphopenia
|
8.0%
8/100 • Number of events 20 • up to 4 years
|
|
Blood and lymphatic system disorders
Neutropenia
|
31.0%
31/100 • Number of events 114 • up to 4 years
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
62.0%
62/100 • Number of events 192 • up to 4 years
|
|
Cardiac disorders
Tachycardia
|
5.0%
5/100 • Number of events 6 • up to 4 years
|
|
Gastrointestinal disorders
Abdominal distension
|
5.0%
5/100 • Number of events 5 • up to 4 years
|
|
Gastrointestinal disorders
Abdominal pain
|
15.0%
15/100 • Number of events 21 • up to 4 years
|
|
Gastrointestinal disorders
Abdominal pain upper
|
7.0%
7/100 • Number of events 7 • up to 4 years
|
|
Gastrointestinal disorders
Constipation
|
11.0%
11/100 • Number of events 12 • up to 4 years
|
|
Gastrointestinal disorders
Diarrhoea
|
42.0%
42/100 • Number of events 71 • up to 4 years
|
|
Gastrointestinal disorders
Dyspepsia
|
7.0%
7/100 • Number of events 7 • up to 4 years
|
|
Gastrointestinal disorders
Gingival bleeding
|
5.0%
5/100 • Number of events 5 • up to 4 years
|
|
Gastrointestinal disorders
Nausea
|
29.0%
29/100 • Number of events 44 • up to 4 years
|
|
Gastrointestinal disorders
Stomatitis
|
10.0%
10/100 • Number of events 16 • up to 4 years
|
|
Gastrointestinal disorders
Vomiting
|
17.0%
17/100 • Number of events 22 • up to 4 years
|
|
General disorders
Asthenia
|
19.0%
19/100 • Number of events 46 • up to 4 years
|
|
General disorders
Chills
|
10.0%
10/100 • Number of events 13 • up to 4 years
|
|
General disorders
Disease progression
|
10.0%
10/100 • Number of events 11 • up to 4 years
|
|
General disorders
Fatigue
|
24.0%
24/100 • Number of events 37 • up to 4 years
|
|
General disorders
Injection site erythema
|
11.0%
11/100 • Number of events 34 • up to 4 years
|
|
General disorders
Injection site rash
|
5.0%
5/100 • Number of events 5 • up to 4 years
|
|
General disorders
Injection site reaction
|
5.0%
5/100 • Number of events 6 • up to 4 years
|
|
General disorders
Mucosal inflammation
|
5.0%
5/100 • Number of events 5 • up to 4 years
|
|
General disorders
Oedema peripheral
|
14.0%
14/100 • Number of events 22 • up to 4 years
|
|
General disorders
Pyrexia
|
28.0%
28/100 • Number of events 50 • up to 4 years
|
|
Infections and infestations
Bronchitis
|
8.0%
8/100 • Number of events 8 • up to 4 years
|
|
Infections and infestations
Cellulitis
|
6.0%
6/100 • Number of events 8 • up to 4 years
|
|
Infections and infestations
Pneumonia
|
13.0%
13/100 • Number of events 15 • up to 4 years
|
|
Infections and infestations
Upper respiratory tract infection
|
9.0%
9/100 • Number of events 13 • up to 4 years
|
|
Infections and infestations
Urinary tract infection
|
6.0%
6/100 • Number of events 7 • up to 4 years
|
|
Injury, poisoning and procedural complications
Contusion
|
8.0%
8/100 • Number of events 9 • up to 4 years
|
|
Investigations
Alanine aminotransferase increased
|
6.0%
6/100 • Number of events 6 • up to 4 years
|
|
Metabolism and nutrition disorders
Anorexia
|
14.0%
14/100 • Number of events 15 • up to 4 years
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
8.0%
8/100 • Number of events 9 • up to 4 years
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
8.0%
8/100 • Number of events 10 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.0%
10/100 • Number of events 11 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.0%
7/100 • Number of events 7 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
9.0%
9/100 • Number of events 10 • up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
15.0%
15/100 • Number of events 18 • up to 4 years
|
|
Nervous system disorders
Dizziness
|
6.0%
6/100 • Number of events 8 • up to 4 years
|
|
Nervous system disorders
Headache
|
20.0%
20/100 • Number of events 27 • up to 4 years
|
|
Psychiatric disorders
Insomnia
|
7.0%
7/100 • Number of events 9 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.0%
13/100 • Number of events 22 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
8.0%
8/100 • Number of events 10 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
17.0%
17/100 • Number of events 19 • up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
9.0%
9/100 • Number of events 9 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
7.0%
7/100 • Number of events 7 • up to 4 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.0%
7/100 • Number of events 8 • up to 4 years
|
|
Vascular disorders
Haematoma
|
5.0%
5/100 • Number of events 12 • up to 4 years
|
|
Vascular disorders
Hypertension
|
6.0%
6/100 • Number of events 8 • up to 4 years
|
Additional Information
Director, Clinical Research
Teva Branded Pharmaceutical Products, R&D Inc.
Phone: 215-591-3000
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor has the right 60 days before submission for publication to review/provide comments. If the Sponsor's review shows that potentially patentable subject matter would be disclosed, publication or public disclosure shall be delayed for up to 90 additional days in order for the Sponsor, or Sponsor's designees, to file the necessary patent applications. In multicenter trials, each PI will postpone single center publications until after disclosure or publication of multicenter data.
- Publication restrictions are in place
Restriction type: OTHER