Trial Outcomes & Findings for AML-02: Omacetaxine With Standard-of-Care Induction With Cytarabine & Idarubicin in Newly-Diagnosed AML Patients (NCT NCT02440568)

Last Updated: 2021-07-06

Results Overview

The primary endpoint is determination of the optimally active and safe dose (OD) of Omacetaxine when added to the standard-of-care induction chemotherapy for AML and estimation of the efficacy and response rate. OD will be defined as a dose level at which fewer than 30% of patients experience hematologic toxicity and greater than 50% of patients achieve a CR (50% is an accepted CR rate in AML when using a single induction).

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

22 participants

Primary outcome timeframe

Within 50 days (duration of hematologic recovery)

Results posted on

2021-07-06

Participant Flow

Participant milestones

Participant milestones
Measure	Patients With Newly Diagnosed AML, Cohort 1 Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Overall Study STARTED	5	4	10	3
Overall Study COMPLETED	1	1	5	1
Overall Study NOT COMPLETED	4	3	5	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Patients With Newly Diagnosed AML, Cohort 1 Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Overall Study Death	1	3	4	2
Overall Study Lack of Efficacy	2	0	1	0
Overall Study Lost to Follow-up	1	0	0	0

Baseline Characteristics

AML-02: Omacetaxine With Standard-of-Care Induction With Cytarabine & Idarubicin in Newly-Diagnosed AML Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients With Newly Diagnosed AML Cohort 1 n=5 Participants Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine (at assigned dose level) administered subcutaneously Q12 hours Days 1 to 7. Dose levels include: 0.625, 1.25, 2.0, 3.0, and 4.2 mg/m\^2 Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML Cohort 2 n=4 Participants Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine (at assigned dose level) administered subcutaneously Q12 hours Days 1 to 7. Dose levels include: 0.625, 1.25, 2.0, 3.0, and 4.2 mg/m\^2 Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days.	Patients With Newly Diagnosed AML Cohort 3 n=10 Participants Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine (at assigned dose level) administered subcutaneously Q12 hours Days 1 to 7. Dose levels include: 0.625, 1.25, 2.0, 3.0, and 4.2 mg/m\^2 Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days.	Patients With Newly Diagnosed AML Cohort 4 n=3 Participants Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine (at assigned dose level) administered subcutaneously Q12 hours Days 1 to 7. Dose levels include: 0.625, 1.25, 2.0, 3.0, and 4.2 mg/m\^2 Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days.	Total n=22 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants	2 Participants n=4 Participants	18 Participants n=21 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	1 Participants n=4 Participants	4 Participants n=21 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants	2 Participants n=4 Participants	13 Participants n=21 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	1 Participants n=4 Participants	9 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants	0 Participants n=4 Participants	6 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants	3 Participants n=4 Participants	16 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	1 Participants n=4 Participants	6 Participants n=21 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants	2 Participants n=4 Participants	14 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Region of Enrollment United States	5 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants	3 Participants n=4 Participants	22 Participants n=21 Participants

PRIMARY outcome

Timeframe: Within 50 days (duration of hematologic recovery)

Population: Number of participants in each cohort

Outcome measures

Outcome measures
Measure	Patients With Newly Diagnosed AML, Cohort 1 n=5 Participants Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 n=4 Participants Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 n=10 Participants Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 n=3 Participants Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Optimally Tolerated Dose	0 Participants treated with OD	0 Participants treated with OD	10 Participants treated with OD	0 Participants treated with OD

SECONDARY outcome

Timeframe: Up to 6 months after last dose of Omacetaxine

Population: All patients enrolled into the study

Describe the adverse events associated with Omacetaxine when administered in combination with cytarabine and Idarubicin as induction therapy for AML, using CTCAE grading

Outcome measures

Outcome measures
Measure	Patients With Newly Diagnosed AML, Cohort 1 n=5 Participants Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 n=4 Participants Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 n=10 Participants Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 n=3 Participants Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Toxicity: Describe by the Adverse Events as Assessed by the CTCAE Grading	47 Total Adverse Events	88 Total Adverse Events	219 Total Adverse Events	37 Total Adverse Events

SECONDARY outcome

Timeframe: Within 6 months of last dose of Omacetaxine

Population: Patients who achieved a CR/CRi when treated with the study medications.

Time to Absolute Neutrophil Count \> 1.0 x 10e9/L and Platelet Count \> 100 x 10e9/L, whichever was later, for those patients who achieved a remission and achieved these hematologic goals.

Outcome measures

Outcome measures
Measure	Patients With Newly Diagnosed AML, Cohort 1 n=2 Participants Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 n=3 Participants Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 n=4 Participants Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 n=1 Participants Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Time to Hematologic Recovery	25.5 Days Interval 24.0 to 27.0	35 Days Interval 24.0 to 37.0	33.5 Days Interval 25.0 to 40.0	36 Days Interval 36.0 to 36.0

SECONDARY outcome

Timeframe: 3 years

Observed overall survival among participants (days)

Outcome measures

Outcome measures
Measure	Patients With Newly Diagnosed AML, Cohort 1 n=5 Participants Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 n=4 Participants Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 n=10 Participants Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 n=3 Participants Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Overall Participant Survival	726 Days Interval 360.5 to Insufficient number of participants with events	162.5 Days Interval 108.0 to Insufficient number of participants with events	792.5 Days Interval 91.0 to Insufficient number of participants with events	33 Days Cannot calculate IQR on 3 participants

SECONDARY outcome

Timeframe: 6 months

Observed length of time (days) after which patients remained free of recurrence or death.

Outcome measures

Outcome measures
Measure	Patients With Newly Diagnosed AML, Cohort 1 n=5 Participants Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 n=4 Participants Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 n=10 Participants Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 n=3 Participants Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Event Free Survival	66 Days Interval 26.5 to Insufficient number of participants with events	90.5 Days Interval 50.0 to Insufficient number of participants with events	65.5 Days Interval 32.0 to Insufficient number of participants with events	33 Days Cannot calculate IQR with 3 participants

SECONDARY outcome

Timeframe: 3 years

Among the participants who achieved CR,CRi, progression free survival in number of days, i.e. the number of days participants who achieved CR/CRi remained alive and in a remission.

Outcome measures

Outcome measures
Measure	Patients With Newly Diagnosed AML, Cohort 1 n=2 Participants Patients will receive Omacetaxine 0.625mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 n=3 Participants Patients will receive Omacetaxine 1.25mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 3 n=4 Participants Patients will receive Omacetaxine 2.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 4 n=1 Participants Patients will receive Omacetaxine 3.0mg/m\^2, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine: Omacetaxine administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.
Progression Free Survival	852.5 Days Interval 66.0 to 1639.0	100 Days Interval 81.0 to 1480.0	1241.5 Days Interval 793.0 to 1422.0	1141 Days Interval 1141.0 to 1141.0

Adverse Events

Patients With Newly Diagnosed AML, Cohort 1

Serious events: 2 serious events

Other events: 5 other events

Deaths: 2 deaths

Patients With Newly Diagnosed AML, Cohort 2

Serious events: 3 serious events

Other events: 4 other events

Deaths: 3 deaths

Patients With Newly Diagnosed AML, Cohort 3

Serious events: 4 serious events

Other events: 10 other events

Deaths: 4 deaths

Patients With Newly Diagnosed AML, Cohort 4

Serious events: 2 serious events

Other events: 3 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Patients With Newly Diagnosed AML, Cohort 1 n=5 participants at risk Patients will receive Omacetaxine, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine at Dose level at 0.625mg/m\^2 administered subcutaneously Q12 hours Days 1 to 7. Cytarabine: Cytarabine (100mg/m\^2/day) in 1000ml NS as a continuous IV infusion over 24 hours x 7 days. Idarubicin: Idarubicin (12 mg/m\^2/day) IVPB in 100 mL NS over 15 minutes daily from Days 1 to 3.	Patients With Newly Diagnosed AML, Cohort 2 n=4 participants at risk Patients will receive Omacetaxine, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine at Dose level at 1.25mg/m\^2 administered	Patients With Newly Diagnosed AML, Cohort 3 n=10 participants at risk Patients will receive Omacetaxine, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine at Dose level at 2.0mg/m\^2 administered	Patients With Newly Diagnosed AML, Cohort 4 n=3 participants at risk Patients will receive Omacetaxine, Cytarabine and Idarubicin as part of the treatment plan. Study participants will follow in outpatient clinic at least every 2 months for a total of 6 months. A final study visit will occur 6 months (+/-1 week) after the last dose of Omacetaxine. This visit will end study participation unless there is ongoing toxicity that is at least possibly related to study treatment. In this case, the patient will be followed as medically appropriate until resolution or stabilization of the adverse event. Omacetaxine at Dose level at 3.0mg/m\^2 administered
Blood and lymphatic system disorders Death	40.0% 2/5 • Number of events 2 • Adverse Events were assessed for 1 year All-Cause Mortality was assessed for 3 years	75.0% 3/4 • Number of events 3 • Adverse Events were assessed for 1 year All-Cause Mortality was assessed for 3 years	40.0% 4/10 • Number of events 4 • Adverse Events were assessed for 1 year All-Cause Mortality was assessed for 3 years	66.7% 2/3 • Number of events 2 • Adverse Events were assessed for 1 year All-Cause Mortality was assessed for 3 years

Other adverse events

Additional Information

Dr. John Quigley

University of Illinois at Chicago

Phone: 312-413-1300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place