Trial Outcomes & Findings for Omacetaxine for Consolidation and Maintenance (NCT NCT01873495)

Last Updated: 2019-09-24

Results Overview

Disease status will be assessed by a bone marrow aspirate and biopsy prior to each of 3 consolidation cycles (to ensure that patients are still in remission).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

7 participants

Primary outcome timeframe

14 days

Results posted on

2019-09-24

Participant Flow

Patients were enrolled between 5/8/2013 and 7/23/2018 at Winship Cancer Institute of Emory University.

Participant milestones

Participant milestones
Measure	Omacetaxine: Consolidation/Maintenance Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Overall Study STARTED	7
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Omacetaxine: Consolidation/Maintenance Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Overall Study Withdrawal by Subject	1
Overall Study Physician Decision	3
Overall Study Adverse Event	1
Overall Study Removed for Transplantation	1
Overall Study AML (Acute Myelogenous Leukemia) Relapse	1

Baseline Characteristics

Omacetaxine for Consolidation and Maintenance

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Omacetaxine: Consolidation/Maintenance n=7 Participants Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=93 Participants
Age, Categorical >=65 years	2 Participants n=93 Participants
Sex: Female, Male Female	3 Participants n=93 Participants
Sex: Female, Male Male	4 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants
Race (NIH/OMB) White	6 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	7 Participants n=93 Participants

PRIMARY outcome

Timeframe: 14 days

Disease status will be assessed by a bone marrow aspirate and biopsy prior to each of 3 consolidation cycles (to ensure that patients are still in remission).

Outcome measures

Outcome measures
Measure	Omacetaxine: Consolidation/Maintenance n=7 Participants Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Disease Status Assessment Prior to Each Consolidation Cycle Relapsed AML	0 Participants
Disease Status Assessment Prior to Each Consolidation Cycle Remission	7 Participants

PRIMARY outcome

Timeframe: 1 month

Population: Seven patients completed at least one cycle of omacetaxine.

Bone marrow biopsy and aspirate will be obtained.

Outcome measures

Outcome measures
Measure	Omacetaxine: Consolidation/Maintenance n=7 Participants Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Assessment of Disease Status Relapsed AML	1 Participants
Assessment of Disease Status Remission	6 Participants

PRIMARY outcome

Timeframe: 3 months

Population: No patients entered the maintenance phase of this study.

Bone marrow aspirate to confirm continuous remission will be obtained before starting maintenance and at 3 and 6 months from the start of maintenance.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 24 weeks

Population: No patients entered the maintenance phase of this study.

Toxicities will be monitored by history, physical examination, and laboratory monitoring during maintenance.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 weeks

Population: Two participants experienced toxicities: thrombocytopenia and atrial flutter.

Toxicities will be monitored by history, physical examination, and laboratory monitoring (CBC, serum chemistries to include renal and liver function tests) obtained weekly during consolidation and monthly during maintenance according to standard of care (Appendices C and D). Toxicity will be assessed according to the NCI Common Toxicity Criteria Version 4.0 (available at the NCI web site http://ctep.cancer.gov/reporting/ctc.html).

Outcome measures

Outcome measures
Measure	Omacetaxine: Consolidation/Maintenance n=7 Participants Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Consolidation Toxicities Thrombocytopenia	1 Participants
Consolidation Toxicities Atrial Flutter	1 Participants
Consolidation Toxicities No Toxicities	5 Participants

Adverse Events

Omacetaxine: Consolidation/Maintenance

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Omacetaxine: Consolidation/Maintenance n=7 participants at risk Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Cardiac disorders Atrial flutter	14.3% 1/7 • Adverse events were collected weekly during consolidation and monthly during maintenance.

Other adverse events

Other adverse events
Measure	Omacetaxine: Consolidation/Maintenance n=7 participants at risk Omacetaxine: Omacetaxine 1.25 mg/m² sub-cutaneously twice daily for 5 consecutive days every 28 (± 8) days for 3 cycles. Patients in continuous remission after 3 cycles of consolidation will receive maintenance omacetaxine 1.25 mg/m² twice daily for 3 days, every 28 days for up to 6 cycles
Blood and lymphatic system disorders Thrombocytopenia	14.3% 1/7 • Adverse events were collected weekly during consolidation and monthly during maintenance.

Additional Information

Martha Arellano, MD

Emory University

Phone: 404-778-1900

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place