Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation

• Sponsor: Teva Branded Pharmaceutical Products R&D, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00375219
• Collaborator: Cephalon (Industry), ChemGenex Pharmaceuticals (Industry)
• Enrollment: 103
• Locations: 32
• Arm: 1
• Actual Duration (Months): 81.2
• Patients Per Site: 3.2
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

To evaluate the safety and efficacy of subcutaneous administration of omacetaxine mepesuccinate (HHT) in achieving a clinical response in CML patients in chronic, accelerated, or blast phase who have failed prior imatinib therapy and have the T315I kinase domain gene mutation.

Condition or Disease	Intervention/Treatment	Phase
Chronic Myeloid Leukemia	Drug: Omacetaxine mepesuccinate	Phase 2

Detailed Description

Point mutations within the ABL kinase domain of the BCR-ABL gene are emerging as the most frequent mechanism for resistance to imatinib and resultant reactivation of kinase activity. The risk of mutation development is particularly high in patients who are beyond chronic phase, as well as those with a long duration of disease prior to imatinib therapy.

The T315I kinase domain (KD) point mutation has merited particular attention, as T315I expressing CML cells are markedly resistant to imatinib. CML patients with the T315I KD mutation, therefore, do not respond to continued treatment with imatinib, and preliminary clinical data indicate that neither of two newer tyrosine kinase inhibitors will have activity in patients with T315I KD mutation either.

Omacetaxine mepesuccinate (HHT) is a potent inducer of apoptosis (programmed cell death) in myeloid cells and inhibits angiogenesis (blood vessel formation). In Phase 2 studies, HHT has demonstrated clinical activity in patients with CML, both as a single agent and in-combination with other chemotherapeutic drugs. HHT works via a different mechanism than imatinib or other tyrosine kinase inhibitors (TKI's), and HHT has been shown to inhibit in vitro CML cell lines which harbor the T315I KD mutation and are highly resistant to imatinib. Therefore, CML patients who have the T315I KD mutation may still respond to treatment with HHT. HHT may therefore be an attractive therapeutic option for patients with the T315I KD mutation.

On this basis, a multicenter clinical trial is being conducted of HHT therapy for CML patients who have failed prior imatinib therapy and have the T315I KD mutation.

Patients will be treated with an induction course consisting of subcutaneous (SC) HHT twice daily for 14 consecutive days every 28 days. Patients who demonstrate a response, may receive maintenance therapy for up to 24 months, consisting of subcutaneous (SC) HHT twice daily for 7 days every 28 days.

Study Design

• Study Type: Interventional
• Actual Enrollment: 103 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase II Open-Label Study of the Subcutaneous Administration of Homoharringtonine. (Omacetaxine) (CGX-635) in the Treatment of Patients With Chronic Myeloid Leukemia. (CML) With the T315I BCR-ABL Gene Mutation
• Actual Study Start Date: Sep 20, 2006
• Actual Primary Completion Date: Mar 23, 2010
• Actual Study Completion Date: Jun 28, 2013

Arms and Interventions

Arm

Intervention/Treatment

Experimental: omacetaxine 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 3 years.

Drug: Omacetaxine mepesuccinate; Induction: 1.25 mg/m^2 subcutaneously, twice daily for 14 consecutive days every 28 days until response. Patients not demonstrating evidence of clinical response after 6 induction cycles will be considered for removal from the study. Maintenance: 1.25 mg/m^2 subcutaneously, twice daily for 7 consecutive days in a 28-day cycle, for up to 3 years.; Other Names: Homoharringtonine OMA Synribo HHT CGX-635

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population [Day 1 up to 6 months]

   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.

2. Percentage of Participants Achieving a Major Cytogenetic Response by Subpopulation and Total Population [Day 1 up to 6 months]

   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.

3. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total [up to 3 years]

   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).

Secondary Outcome Measures

1. Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+) [Day 1 up to Month 9]

   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

2. Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene GUS [Day 1 up to Month 6]

   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.

3. Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene ABL [Day 1 up to Month 6]

   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.

4. Percentage of Participants in Each Hematologic Response Category [Day 1 up to Month 6]

   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.

5. Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response [Day 1 up to Month 9]

   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). The percentage of participants achieving response with extramedullary disease at Baseline was to be summarized, if the sample size was sufficient. This analysis was not done as the sample was ultimately insufficient

6. Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL [Day 1 up to Month 9]

   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).

7. Number of Treatment Cycles Needed to Achieve Best Hematologic Response [Day 1 up to Month 6]

   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.

8. Number of Treatment Cycles Needed to Achieve Best Cytogenetic Response [Day 1 up to 22 months]

9. Kaplan-Meier Estimates for Time to Onset of Best Hematologic Response [Day 1 up to Month 6]

   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.

10. Kaplan-Meier Estimates for Time to Onset of Best Cytogenetic Response [up to 3 years]

    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.

11. Kaplan-Meier Estimates for Duration of Best Hematologic Response [up to 4 years]

    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.

12. Kaplan-Meier Estimates for Duration of Best Cytogenetic Response [up to 4 years]

    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.

13. Kaplan-Meier Estimates for Time to Disease Progression [up to 4 years]

    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.

14. Kaplan-Meier Estimates for Overall Survival [up to 4 years]

    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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Male or female patients, age 18 years or older

• Philadelphia chromosome (Ph) positive chronic myelogenous leukemia in either chronic, accelerated, or blast phase

• The patient will have the T315I BCR-ABL gene mutation

• Patients will have failed prior imatinib therapy

• ECOG performance status 0-2

Exclusion Criteria:

• NYHA class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia and requiring therapy, uncontrolled hypertension or congestive heart failure

• Myocardial infarction in the previous 12 weeks

• Lymphoid Ph+ blast crisis

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Teva Investigational Site 003	Los Angeles	California	United States	90033
2	Teva Investigational Site 007	Jacksonville	Florida	United States	32224
3	Teva Investigational Site 006	Atlanta	Georgia	United States	30329
4	Teva Investigational Site 008	Beech Grove	Indiana	United States	46107
5	Teva Investigational Site 011	Baltimore	Maryland	United States	21201
6	Teva Investigational Site 004	Boston	Massachusetts	United States	02111
7	Teva Investigational Site 002	Bronx	New York	United States	10467
8	Teva Investigational Site 005	Buffalo	New York	United States	14263
9	Teva Investigational Site 010	Philadelphia	Pennsylvania	United States	19111
10	Teva Investigational Site 001	Houston	Texas	United States	77030
11	Teva Investigational Site 013	Montreal		Canada	H3a 1a1
12	Teva Investigational Site 009	Toronto		Canada	M5G 2M9
13	Teva Investigational Site 029	Bordeaux		France	33076
14	Teva Investigational Site 021	Le Chesnay Cedex		France	78157
15	Teva Investigational Site 022	Lille		France	59000
16	Teva Investigational Site 020	Lyon Cedex 03		France	69437
17	Teva Investigational Site 024	Nice		France	06202
18	Teva Investigational Site 028	Paris		France	75475
19	Teva Investigational Site 023	Poitiers Cedex		France	86021
20	Teva Investigational Site 027	Strasbourg		France	67100
21	Teva Investigational Site 025	Toulouse		France	31059
22	Teva Investigational Site 026	Vandoeuvre-Les-Nancy CEDEX		France	54511
23	Teva Investigational Site 031	Berlin		Germany	10117
24	Teva Investigational Site 030	Mannheim		Germany	68169
25	Teva Investigational Site 050	Budapest		Hungary	1096
26	Teva Investigational Site 071	Hyderabad		India	500082
27	Teva Investigational Site 070	Mumbai		India	400 014
28	Teva Investigational Site 090	Bologna		Italy	41038
29	Teva Investigational Site 060	Gdansk		Poland	80-952
30	Teva Investigational Site 061	Warszawa		Poland	02776
31	Teva Investigational Site 080	Singapore		Singapore	169608
32	Teva Investigational Site 040	London		United Kingdom	W12 0HS

Sponsors and Collaborators

• Teva Branded Pharmaceutical Products R&D, Inc.
• Cephalon
• ChemGenex Pharmaceuticals

Investigators

• Principal Investigator: Jorge Cortes, MD, Univ. of Texas M.D. Anderson Cancer Center
• Principal Investigator: Andreas Hochhaus, MD Prof Dr, Mannheim der Universitat Heidelberg

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Teva Branded Pharmaceutical Products R&D, Inc.
• ClinicalTrials.gov Identifier: NCT00375219
• Other Study ID Numbers: CGX-635-CML-202; 2006-000176-32
• First Posted: Sep 12, 2006
• Last Update Posted: Nov 15, 2021
• Last Verified: Nov 1, 2021

• Keywords provided by Teva Branded Pharmaceutical Products R&D, Inc.: Chronic Myeloid Leukemia; CML; HHT; Homoharringtonine; Omacetaxine; T315i; ChemGenex; ChemGenex Pharmaceuticals
• Additional relevant MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Homoharringtonine

Study Results

Participant Flow

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.
Period Title: Overall Study
STARTED	62	20	21
COMPLETED	3	0	0
NOT COMPLETED	59	20	21

Baseline Characteristics

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	Total of all reporting groups
Overall Participants	62	20	21	103
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	59	59	50	57
Sex: Female, Male (Count of Participants)
Female	20 32.3%	5 25%	7 33.3%	32 31.1%
Male	42 67.7%	15 75%	14 66.7%	71 68.9%
Race/Ethnicity, Customized (participants) [Number]
Caucasian	48 77.4%	12 60%	13 61.9%	73 70.9%
Black	4 6.5%	6 30%	6 28.6%	16 15.5%
Hispanic	0 0%	0 0%	0 0%	0 0%
Asian	8 12.9%	2 10%	2 9.5%	12 11.7%
Other	2 3.2%	0 0%	0 0%	2 1.9%
Height (centimeter) [Median (Full Range) ]
Median (Full Range) [centimeter]	171.5	172.0	170.2	171.0
Weight (kilograms) [Median (Full Range) ]
Median (Full Range) [kilograms]	77.7	69.1	68.8	76.0
Body Surface Area (BSA) (meters^2) [Median (Full Range) ]
Median (Full Range) [meters^2]	1.9	1.8	1.8	1.9
New York Heart Association (NYHA) Classification (participants) [Number]
Class I	61 98.4%	18 90%	18 85.7%	97 94.2%
Class II	1 1.6%	2 10%	3 14.3%	6 5.8%
Class III	0 0%	0 0%	0 0%	0 0%
Class IV	0 0%	0 0%	0 0%	0 0%
Eastern Cooperative Oncology Group Performance Status (participants) [Number]
Grade 0	41 66.1%	6 30%	6 28.6%	53 51.5%
Grade 1	20 32.3%	11 55%	9 42.9%	40 38.8%
Grade 2	1 1.6%	2 10%	5 23.8%	8 7.8%
Grade 3	0 0%	1 5%	1 4.8%	2 1.9%
Time from Initial Chronic Myeloid Leukemia (CML) Diagnosis (months) [Median (Full Range) ]
Median (Full Range) [months]	50.0	98.0	46.6	59.6

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population
Description	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.
Time Frame	Day 1 up to 6 months

Outcome Measure Data

Analysis Population Description

Intent to treat population

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21	103
Number (95% Confidence Interval) [percentage of participants]	77.4 124.8%	55.0 275%	9.5 45.2%	59.2 57.5%

2. Primary Outcome

Title	Percentage of Participants Achieving a Major Cytogenetic Response by Subpopulation and Total Population
Description	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.
Time Frame	Day 1 up to 6 months

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21	103
Number (95% Confidence Interval) [percentage of participants]	22.6 36.5%	5.0 25%	0 0%	14.6 14.2%

3. Primary Outcome

Title	Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Description	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).
Time Frame	up to 3 years

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21	103
>=1 TEAE	61 98.4%	20 100%	21 100%	102 99%
>= 1 SAE	36 58.1%	12 60%	19 90.5%	67 65%
Worst severity: Grade 1	0 0%	1 5%	0 0%	1 1%
Worst severity: Grade 2	6 9.7%	2 10%	1 4.8%	9 8.7%
Worst severity: Grade 3	9 14.5%	4 20%	4 19%	17 16.5%
Worst severity: Grade 4	37 59.7%	9 45%	4 19%	50 48.5%
Worst severity: Grade 5	9 14.5%	4 20%	12 57.1%	25 24.3%
Relation to drug: Unrelated	1 1.6%	3 15%	3 14.3%	7 6.8%
Relation to drug: Possibly	6 9.7%	3 15%	5 23.8%	14 13.6%
Relation to drug: Probably	54 87.1%	13 65%	13 61.9%	80 77.7%
Relation to drug: Unknown	0 0%	1 5%	0 0%	1 1%
With hematologic toxicity	55 88.7%	13 65%	13 61.9%	81 78.6%
Discontinued treatment due to AE	18 29%	10 50%	11 52.4%	39 37.9%
Deaths during study or follow-up	31 50%	14 70%	19 90.5%	64 62.1%
Deaths during study (outcome of SAE)	9 14.5%	4 20%	12 57.1%	25 24.3%

4. Secondary Outcome

Title	Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Description	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
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21	103
Complete	16.1 26%	5.0 25%	0 0%	10.7 10.4%
Partial	6.5 10.5%	0 0%	0 0%	3.9 3.8%
Minor	4.8 7.7%	0 0%	0 0%	2.9 2.8%
Minimal	16.1 26%	5.0 25%	9.5 45.2%	12.6 12.2%
No Response	37.1 59.8%	30.0 150%	38.1 181.4%	35.9 34.9%
Unevaluable	19.4 31.3%	60.0 300%	52.4 249.5%	34.0 33%

5. Secondary Outcome

Title	Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene GUS
Description	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.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	37	8	4	49
Number (95% Confidence Interval) [percentage of participants]	8.1 13.1%	12.5 62.5%	0 0%	8.2 8%

6. Secondary Outcome

Title	Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene ABL
Description	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.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	52	13	8	73
Number (95% Confidence Interval) [percentage of participants]	19.2 31%	15.4 77%	0 0%	16.4 15.9%

7. Secondary Outcome

Title	Percentage of Participants in Each Hematologic Response Category
Description	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.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21	103
Complete response	77.4 124.8%	45.0 225%	4.8 22.9%	56.3 54.7%
Partial response	0 0%	0 0%	0 0%	0 0%
Hematologic improvement	0 0%	0 0%	4.8 22.9%	1.0 1%
Return to chronic phase	NA NaN	5.0 25%	4.8 22.9%	1.9 1.8%
No evidence of leukemia	NA NaN	5.0 25%	0 0%	1.0 1%
No response	19.4 31.3%	25.0 125%	81.0 385.7%	33.0 32%
Unevaluable	3.2 5.2%	20.0 100%	4.8 22.9%	6.8 6.6%

8. Secondary Outcome

Title	Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response
Description	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). The percentage of participants achieving response with extramedullary disease at Baseline was to be summarized, if the sample size was sufficient. This analysis was not done as the sample was ultimately insufficient
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat population of study participants who had extramedullary disease at baseline. Analysis not performed due to insufficient sample size.

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.
Measure Participants	0	0	0

9. Secondary Outcome

Title	Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
Description	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).
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants with post-baseline assessment of T315I mutated BCR ABL

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	50	13	8	71
100% reduction	6.0 9.7%	0 0%	0 0%	4.2 4.1%
75-99% reduction	4.0 6.5%	0 0%	25.0 119%	5.6 5.4%
50-74% reduction	14.0 22.6%	0 0%	12.5 59.5%	11.3 11%
25-49% reduction	18.0 29%	15.4 77%	0 0%	15.5 15%
1-24% reduction	8.0 12.9%	38.5 192.5%	12.5 59.5%	14.1 13.7%
0% reduction	14.0 22.6%	15.4 77%	25.0 119%	15.5 15%
Not assessable	36.0 58.1%	30.8 154%	25.0 119%	33.8 32.8%

10. Secondary Outcome

Title	Number of Treatment Cycles Needed to Achieve Best Hematologic Response
Description	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.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a response to treatment

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	48	11	2	61
Median (Full Range) [treatment cycles]	1.0	1.0	1.0	1.0

11. Secondary Outcome

Title	Number of Treatment Cycles Needed to Achieve Best Cytogenetic Response
Time Frame	Day 1 up to 22 months

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a cytogenetic response

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	27	2	2	31
Median (Full Range) [treatment cycles]	3.0	2.5	2.0	3.0

12. Secondary Outcome

Title	Kaplan-Meier Estimates for Time to Onset of Best Hematologic Response
Description	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.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21
Median (95% Confidence Interval) [months]	0.46	1.74	NA

13. Secondary Outcome

Title	Kaplan-Meier Estimates for Time to Onset of Best Cytogenetic Response
Description	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.
Time Frame	up to 3 years

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21
Median (95% Confidence Interval) [months]	NA	NA	NA

14. Secondary Outcome

Title	Kaplan-Meier Estimates for Duration of Best Hematologic Response
Description	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.
Time Frame	up to 4 years

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a response

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.
Measure Participants	48	11	2
Median (Full Range) [months]	9.08	3.59	3.31

15. Secondary Outcome

Title	Kaplan-Meier Estimates for Duration of Best Cytogenetic Response
Description	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.
Time Frame	up to 4 years

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a response

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.
Measure Participants	14	1	0
Median (Full Range) [months]	6.64	16.35

16. Secondary Outcome

Title	Kaplan-Meier Estimates for Time to Disease Progression
Description	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.
Time Frame	up to 4 years

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21	103
Median (95% Confidence Interval) [months]	7.73	4.74	2.20	5.86

17. Secondary Outcome

Title	Kaplan-Meier Estimates for Overall Survival
Description	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.
Time Frame	up to 4 years

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	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 3 years.	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 3 years.	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 3 years.	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 3 years.
Measure Participants	62	20	21	103
Median (95% Confidence Interval) [months]	49.31	18.72	3.45	21.51

Adverse Events

Time Frame	up to 4 years
Arm/Group Title	Omacetaxine
Arm/Group Description	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.
All Cause Mortality
	Omacetaxine
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Omacetaxine
	Affected / at Risk (%)	# Events
Total	67/103 (65%)
Blood and lymphatic system disorders
Anaemia	5/103 (4.9%)	5
Bone marrow failure	8/103 (7.8%)	10
Bone marrow necrosis	1/103 (1%)	1
Febrile bone marrow aplasia	2/103 (1.9%)	2
Febrile neutropenia	12/103 (11.7%)	14
Haematotoxicity	1/103 (1%)	1
Leukocytosis	1/103 (1%)	1
Leukopenia	1/103 (1%)	1
Leukostasis	1/103 (1%)	1
Neutropenia	3/103 (2.9%)	4
Pancytopenia	6/103 (5.8%)	6
Thrombocytopenia	12/103 (11.7%)	17
Cardiac disorders
Acute coronary syndrome	1/103 (1%)	1
Arrhythmia	2/103 (1.9%)	2
Coronary artery disease	1/103 (1%)	1
Extrasystoles	1/103 (1%)	1
Left ventricular failure	1/103 (1%)	1
Congenital, familial and genetic disorders
Chromosomal deletion	1/103 (1%)	1
Endocrine disorders
Diabetes insipidus	1/103 (1%)	1
Gastrointestinal disorders
Anal fissure	1/103 (1%)	1
Gastrointestinal haemorrhage	2/103 (1.9%)	2
General disorders
Aplasia	1/103 (1%)	1
Chest pain	1/103 (1%)	1
Death	1/103 (1%)	1
Disease progression	12/103 (11.7%)	12
General physical health deterioration	1/103 (1%)	1
Pyrexia	2/103 (1.9%)	2
Hepatobiliary disorders
Cholecystitis	1/103 (1%)	1
Infections and infestations
Abscess limb	1/103 (1%)	1
Bronchopulmonary aspergillosis	1/103 (1%)	1
Cellulitis	2/103 (1.9%)	2
Gastroenteritis	1/103 (1%)	1
Gastroenteritis viral	1/103 (1%)	1
Influenza	1/103 (1%)	1
Pneumonia	2/103 (1.9%)	2
Sepsis	3/103 (2.9%)	3
Staphylococcal bacteraemia	1/103 (1%)	1
Subcutaneous abscess	2/103 (1.9%)	2
Injury, poisoning and procedural complications
Subdural haematoma	1/103 (1%)	1
Transfusion reaction	2/103 (1.9%)	2
Metabolism and nutrition disorders
Hyperglycaemic hyperosmolar nonketotic syndrome	1/103 (1%)	1
Musculoskeletal and connective tissue disorders
Back pain	1/103 (1%)	1
Bone pain	3/103 (2.9%)	3
Musculoskeletal chest pain	1/103 (1%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Blast cell crisis	2/103 (1.9%)	2
Breast cancer	1/103 (1%)	1
Chronic myeloid leukaemia	2/103 (1.9%)	2
Leukaemic infiltration extramedullary	1/103 (1%)	1
Myelodysplastic syndrome	1/103 (1%)	1
Myelofibrosis	1/103 (1%)	1
Tumour lysis syndrome	1/103 (1%)	1
Nervous system disorders
Carotid artery stenosis	1/103 (1%)	1
Cerebral haemorrhage	2/103 (1.9%)	2
Convulsion	1/103 (1%)	1
Transient ischaemic attack	2/103 (1.9%)	2
Psychiatric disorders
Confusional state	1/103 (1%)	1
Delirium	1/103 (1%)	1
Mental status changes	1/103 (1%)	1
Renal and urinary disorders
Renal failure chronic	1/103 (1%)	1
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome	1/103 (1%)	1
Chronic obstructive pulmonary disease	1/103 (1%)	1
Epistaxis	1/103 (1%)	1
Pulmonary haemorrhage	1/103 (1%)	1
Skin and subcutaneous tissue disorders
Pyoderma gangrenosum	1/103 (1%)	1
Vascular disorders
Deep vein thrombosis	1/103 (1%)	1
Orthostatic hypotension	1/103 (1%)	1
Other (Not Including Serious) Adverse Events
	Omacetaxine
	Affected / at Risk (%)	# Events
Total	100/103 (97.1%)
Blood and lymphatic system disorders
Anaemia	56/103 (54.4%)	196
Bone marrow failure	8/103 (7.8%)	10
Febrile neutropenia	15/103 (14.6%)	21
Leukocytosis	10/103 (9.7%)	20
Leukopenia	18/103 (17.5%)	64
Lymphopenia	13/103 (12.6%)	39
Neutropenia	48/103 (46.6%)	139
Pancytopenia	15/103 (14.6%)	22
Thrombocytopenia	67/103 (65%)	253
Cardiac disorders
Tachycardia	8/103 (7.8%)	8
Gastrointestinal disorders
Abdominal pain	14/103 (13.6%)	26
Abdominal pain upper	14/103 (13.6%)	16
Constipation	18/103 (17.5%)	18
Diarrhoea	40/103 (38.8%)	107
Dry mouth	6/103 (5.8%)	6
Gingival bleeding	8/103 (7.8%)	9
Mouth ulceration	8/103 (7.8%)	8
Nausea	36/103 (35%)	96
Vomiting	15/103 (14.6%)	17
General disorders
Asthenia	22/103 (21.4%)	59
Chest pain	7/103 (6.8%)	10
Chills	7/103 (6.8%)	7
Disease progression	18/103 (17.5%)	19
Fatigue	35/103 (34%)	65
Injection site bruising	8/103 (7.8%)	8
Injection site erythema	17/103 (16.5%)	66
Injection site pain	8/103 (7.8%)	10
Injection site reaction	7/103 (6.8%)	7
Mucosal inflammation	6/103 (5.8%)	8
Oedema peripheral	20/103 (19.4%)	30
Pyrexia	30/103 (29.1%)	36
Hepatobiliary disorders
Hyperbilirubinaemia	7/103 (6.8%)	17
Infections and infestations
Oral herpes	7/103 (6.8%)	8
Pneumonia	6/103 (5.8%)	6
Upper respiratory tract infection	11/103 (10.7%)	15
Injury, poisoning and procedural complications
Contusion	6/103 (5.8%)	6
Metabolism and nutrition disorders
Anorexia	12/103 (11.7%)	12
Decreased appetite	6/103 (5.8%)	6
Hyperglycaemia	6/103 (5.8%)	8
Hyperuricaemia	10/103 (9.7%)	20
Hypokalaemia	6/103 (5.8%)	7
Musculoskeletal and connective tissue disorders
Arthralgia	22/103 (21.4%)	39
Back pain	10/103 (9.7%)	13
Bone pain	12/103 (11.7%)	17
Myalgia	10/103 (9.7%)	12
Pain in extremity	12/103 (11.7%)	19
Nervous system disorders
Dizziness	8/103 (7.8%)	9
Headache	15/103 (14.6%)	26
Psychiatric disorders
Anxiety	6/103 (5.8%)	6
Insomnia	14/103 (13.6%)	16
Respiratory, thoracic and mediastinal disorders
Cough	20/103 (19.4%)	24
Dyspnoea	11/103 (10.7%)	15
Epistaxis	19/103 (18.4%)	25
Skin and subcutaneous tissue disorders
Alopecia	14/103 (13.6%)	14
Dry skin	8/103 (7.8%)	10
Erythema	6/103 (5.8%)	8
Night sweats	6/103 (5.8%)	9
Petechiae	7/103 (6.8%)	8
Pruritus	8/103 (7.8%)	10
Rash	15/103 (14.6%)	20
Vascular disorders
Haematoma	7/103 (6.8%)	7
Hypertension	8/103 (7.8%)	9

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.