Trial Outcomes & Findings for A Study to Evaluate the Pharmacokinetics, Safety,and Efficacy of Omacetaxine Given Subcutaneously as a Fixed Dose in Patients With Chronic Phase (CP) or Accelerated Phase (AP) Chronic Myeloid Leukemia (CML) (Referred to as the SYNSINCT Study) (NCT NCT02078960)
NCT ID: NCT02078960
Last Updated: 2021-11-09
Results Overview
The Independent Data Monitoring Committee assessments are summarized. Major response for participants with chronic phase CML was a major cytogenetic response (MCyR) defined as a complete cytogenetic response with no Ph+ metaphases, or a partial cytogenetic response with up to 35% Ph+ metaphases. Note that a complete hematologic response was not considered a major response. Major response for participants with accelerated phase CML was a major hematologic response (MaHR) defined as a complete hematologic response or no evidence of leukemia, and/or a major cytogenic response (MCyR).
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Day 1 up to Month 15 (longest treatment duration)
Results posted on
2021-11-09
Participant Flow
A total of 14 patients with chronic myeloid leukemia were screened for enrollment into this study. Of the 4 patients who were not enrolled, 2 were excluded on the basis of inclusion/exclusion criteria and 2 for "other" reasons.
Of the 14 patients screened, 9 patients at 3 centers in the US and 1 patient at a single centre in France met entry criteria and were considered to be eligible for enrollment into the study.
Participant milestones
| Measure |
Cohort 1
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Treatment Period
STARTED
|
2
|
4
|
4
|
|
Treatment Period
COMPLETED
|
0
|
1
|
1
|
|
Treatment Period
NOT COMPLETED
|
2
|
3
|
3
|
|
Follow-up Period
STARTED
|
2
|
2
|
1
|
|
Follow-up Period
COMPLETED
|
0
|
1
|
1
|
|
Follow-up Period
NOT COMPLETED
|
2
|
1
|
0
|
Reasons for withdrawal
| Measure |
Cohort 1
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Treatment Period
Other
|
0
|
0
|
1
|
|
Treatment Period
Lack of Efficacy
|
0
|
1
|
0
|
|
Treatment Period
Disease progression - accelerated phase
|
0
|
1
|
0
|
|
Treatment Period
Disease progression - blast phase
|
1
|
0
|
0
|
|
Treatment Period
Withdrawal by Subject
|
0
|
1
|
1
|
|
Treatment Period
Adverse Event
|
1
|
0
|
0
|
|
Treatment Period
Death
|
0
|
0
|
1
|
|
Follow-up Period
Death
|
2
|
0
|
0
|
|
Follow-up Period
Lost to Follow-up
|
0
|
1
|
0
|
Baseline Characteristics
A Study to Evaluate the Pharmacokinetics, Safety,and Efficacy of Omacetaxine Given Subcutaneously as a Fixed Dose in Patients With Chronic Phase (CP) or Accelerated Phase (AP) Chronic Myeloid Leukemia (CML) (Referred to as the SYNSINCT Study)
Baseline characteristics by cohort
| Measure |
Cohort 1
n=2 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
n=4 Participants
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
n=4 Participants
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Total
n=10 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
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Age, Continuous
|
66.5 years
STANDARD_DEVIATION 9.19 • n=5 Participants
|
46.3 years
STANDARD_DEVIATION 22.87 • n=7 Participants
|
68.3 years
STANDARD_DEVIATION 17.31 • n=5 Participants
|
59.1 years
STANDARD_DEVIATION 20.16 • n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 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
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 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
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
7 Participants
n=4 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
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Weight
|
55.1 kg
STANDARD_DEVIATION 7.78 • n=5 Participants
|
73.7 kg
STANDARD_DEVIATION 4.58 • n=7 Participants
|
105.3 kg
STANDARD_DEVIATION 18.34 • n=5 Participants
|
82.6 kg
STANDARD_DEVIATION 23.63 • n=4 Participants
|
|
Height
|
167.0 cm
STANDARD_DEVIATION 1.41 • n=5 Participants
|
167.3 cm
STANDARD_DEVIATION 8.02 • n=7 Participants
|
169.7 cm
STANDARD_DEVIATION 9.55 • n=5 Participants
|
168.2 cm
STANDARD_DEVIATION 7.33 • n=4 Participants
|
|
Body Surface Area (BSA)
|
1.6 m^2
STANDARD_DEVIATION 0.08 • n=5 Participants
|
1.8 m^2
STANDARD_DEVIATION 0.09 • n=7 Participants
|
2.2 m^2
STANDARD_DEVIATION 0.25 • n=5 Participants
|
2.0 m^2
STANDARD_DEVIATION 0.30 • n=4 Participants
|
|
Phase of Chronic Myeloid Leukemia (CML) at Study Entry
Chronic Phase (CP)
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Phase of Chronic Myeloid Leukemia (CML) at Study Entry
Accelerated Phase (AP)
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Day 1 up to Month 15 (longest treatment duration)Population: Enrolled participants
The Independent Data Monitoring Committee assessments are summarized. Major response for participants with chronic phase CML was a major cytogenetic response (MCyR) defined as a complete cytogenetic response with no Ph+ metaphases, or a partial cytogenetic response with up to 35% Ph+ metaphases. Note that a complete hematologic response was not considered a major response. Major response for participants with accelerated phase CML was a major hematologic response (MaHR) defined as a complete hematologic response or no evidence of leukemia, and/or a major cytogenic response (MCyR).
Outcome measures
| Measure |
Cohort 1
n=2 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
n=4 Participants
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
n=4 Participants
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Number of Participants Who Achieved a Major Response at Any Time During Treatment
|
0 Participants
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Day 1 to Day 541 (longest progression/survival follow-up)Population: Enrolled participants who had a response
Duration of response was defined for responders as the time interval from the first reported date of a major cytogenetic response (MCyR) or a major hematologic response (MaHR) to the earliest date of objective evidence of disease progression (ie, development of accelerated-phase CML), relapse (ie, loss of complete hematologic or major cytogenetic response), or death. Kaplan-Meier estimates for duration of response were not performed due the small enrollment population and early termination of the study. What is reported is the longest duration of response observed prior to disease progression, death, lost to follow-up or study termination.
Outcome measures
| Measure |
Cohort 1
n=5 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Longest Duration of Response At Study Termination
|
316 days
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1 up to Month 15Population: Enrolled participants
Molecular response was defined by the decrease in the amount of BCR-ABL (an abnormality of chromosome 22) messenger ribonucleic acid (mRNA) measured by reverse transcriptase polymerase chain reaction (RT-PCR) or by the actual percentage of BCR-ABL mRNA transcripts (ratio of BCR-ABL transcript numbers to the number of control gene transcripts).
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Number of Participants Who Had a Molecular Response at Any Time During Treatment
|
1 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1 to Day 541 (longest progression/survival follow-up)Population: Enrolled participants
Progression-free survival was defined as the time interval from the date of first dose to the date of the earliest objective evidence of disease progression (ie, development of accelerated-phase CML), relapse (ie, loss of complete hematologic or major cytogenetic response), or death. Kaplan-Meier estimates for time for progression-free survival were not performed due the small enrollment population and early termination of the study. What is reported is the number of participants who were alive and progression-free at the time of study termination.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Number of Participants Who Were Alive and Progression-Free at Study Termination
|
4 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1 to Day 541 (longest progression/survival follow-up)Population: Enrolled participants
Overall survival was defined as the time interval from the date of the first dose to the date of death from any cause. Kaplan-Meier time estimates for overall survival were not performed due to the small enrollment population and early termination of the study. What is reported is the number of participants who were alive at the time of study termination.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Number of Participants Who Were Alive at Study Termination
|
7 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Cycle 1 - Day 1 (predose, 15, 30, 45 minutes, 1, 2, 4, 8, 24, 48, 72 hours post-dose) Day 10 (predose, 2 samples postdose), Day 13 (predose) Cycles 2+3 - Day 1 (2 samples postdose), Days 10 and 17 (1 sample predose)Population: PK analysis set
Maximum observed plasma drug concentration (Cmax) by inspection (without interpolation). Nominal PK sampling times were used.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) for Omacetaxine
|
23.88 ng/mL
Standard Deviation 13.32
|
—
|
—
|
SECONDARY outcome
Timeframe: Cycle 1 - Day 1 (predose, 15, 30, 45 minutes, 1, 2, 4, 8, 24, 48, 72 hours post-dose) Day 10 (predose, 2 samples postdose), Day 13 (predose) Cycles 2+3 - Day 1 (2 samples postdose), Days 10 and 17 (1 sample predose)Population: PK analysis set
Time to maximum observed plasma drug concentration (Cmax) by inspection (without interpolation). Nominal PK sampling times were used.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Time to Maximum Observed Plasma Concentration (Cmax) for Omacetaxine
|
0.25 hour
Interval 0.25 to 1.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Cycle 1 - Day 1 (predose, 15, 30, 45 minutes, 1, 2, 4, 8, 24, 48, 72 hours post-dose) Day 10 (predose, 2 samples postdose), Day 13 (predose) Cycles 2+3 - Day 1 (2 samples postdose), Days 10 and 17 (1 sample predose)Population: PK analysis set
Nominal PK sampling times were used.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Area Under the Drug Concentration by Time Curve From Time 0 to the Time of the Last Measurable Drug Concentration (AUC0-t) for Omacetaxine
|
150.2 ng*hr/mL
Standard Deviation 55.8
|
—
|
—
|
SECONDARY outcome
Timeframe: Cycle 1 - Day 1 (predose, 15, 30, 45 minutes, 1, 2, 4, 8, 24, 48, 72 hours post-dose) Day 10 (predose, 2 samples postdose), Day 13 (predose) Cycles 2+3 - Day 1 (2 samples postdose), Days 10 and 17 (1 sample predose)Population: PK analysis set
Nominal PK sampling times were used.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Area Under the Drug Concentration by Time Curve From Time 0 to Infinity (AUC0-inf) for Omacetaxine
|
154.8 ng*hr/mL
Standard Deviation 60.8
|
—
|
—
|
SECONDARY outcome
Timeframe: Cycle 1 - Day 1 (predose, 15, 30, 45 minutes, 1, 2, 4, 8, 24, 48, 72 hours post-dose) Day 10 (predose, 2 samples postdose), Day 13 (predose) Cycles 2+3 - Day 1 (2 samples postdose), Days 10 and 17 (1 sample predose)Population: PK analysis set
Nominal PK sampling times were used.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Terminal Elimination Half-Life (t1/2) for Omacetaxine
|
13.3 hour
Standard Deviation 1.6
|
—
|
—
|
SECONDARY outcome
Timeframe: Cycle 1 - Day 1 (predose, 15, 30, 45 minutes, 1, 2, 4, 8, 24, 48, 72 hours post-dose) Day 10 (predose, 2 samples postdose), Day 13 (predose) Cycles 2+3 - Day 1 (2 samples postdose), Days 10 and 17 (1 sample predose)Population: PK analysis set
Nominal PK sampling times were used.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Total Oral Clearance (CL/F) for Omacetaxine
|
18.1 L/hour
Standard Deviation 6.1
|
—
|
—
|
SECONDARY outcome
Timeframe: Cycle 1 - Day 1 (predose, 15, 30, 45 minutes, 1, 2, 4, 8, 24, 48, 72 hours post-dose) Day 10 (predose, 2 samples postdose), Day 13 (predose) Cycles 2+3 - Day 1 (2 samples postdose), Days 10 and 17 (1 sample predose)Population: PK analysis set
Nominal PK sampling times were used.
Outcome measures
| Measure |
Cohort 1
n=10 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Apparent Volume of Distribution (V/F) for Omacetaxine
|
344.9 L
Standard Deviation 117.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1 up to Month 15Population: Safety Analysis set
An adverse event is any untoward medical occurrence in a patient administered a pharmaceutical product, regardless of whether it has a causal relationship with this treatment. A treatment-emergent adverse event (TEAE) is an AE that that began or worsened after treatment with study drug. Severity rating of 3=Severe or medically significant but not immediately life-threatening 4=Life-threatening consequences and 5=Death. Relation to study drug is determined by the investigator. A serious adverse event (SAE) includes death, a life-threatening AE, hospitalization, persistent or significant disability or incapacity, a congenital anomaly/birth defect, or any important medical event requiring immediate intervention to prevent one of the outcomes above.
Outcome measures
| Measure |
Cohort 1
n=2 Participants
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 2
n=4 Participants
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
Cohort 3
n=4 Participants
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily. Induction cycles of 14 days twice daily treatment were followed by 14 days without treatment (1 induction cycle) for as long as they adequately recover their blood counts and/or until they achieve hematologic response. \[Cycle 1 was an exception since participants were not dosed a second time on Day 1, nor at all on Days 2+3 to accommodate the PK study objective.\] Participants then received maintenance cycles of 7 days twice daily treatment followed by 21 days without treatment (1 maintenance cycle) for as long as they continued to benefit up to a period of 12 months following the first dose.
|
|---|---|---|---|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
Withdrawn from treatment due to a TEAE
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
Any TEAE
|
2 Participants
|
4 Participants
|
4 Participants
|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
TEAE of Severity Grade 3
|
0 Participants
|
3 Participants
|
0 Participants
|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
TEAE of Severity Grade 4
|
1 Participants
|
1 Participants
|
3 Participants
|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
TEAE of Severity Grade 5
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
Treatment-related TEAE
|
2 Participants
|
4 Participants
|
4 Participants
|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
Deaths
|
2 Participants
|
0 Participants
|
1 Participants
|
|
Participants With Treatment-Emergent Adverse Events (TEAEs)
Serious AE
|
2 Participants
|
2 Participants
|
4 Participants
|
Adverse Events
COHORT 1
Serious events: 2 serious events
Other events: 2 other events
Deaths: 2 deaths
COHORT 2
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
COHORT 3
Serious events: 4 serious events
Other events: 4 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
COHORT 1
n=2 participants at risk
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily.
|
COHORT 2
n=4 participants at risk
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily.
|
COHORT 3
n=4 participants at risk
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily.
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Neutropenia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Cardiac disorders
Cardiac failure congestive
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Oral pain
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Stomatitis
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
General disorders
Death
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
General disorders
Pain
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
General disorders
Pyrexia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Infections and infestations
Abscess limb
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Infections and infestations
Herpes zoster
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Infections and infestations
Pneumonia
|
50.0%
1/2 • Number of events 2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
|
Infections and infestations
Septic shock
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Infections and infestations
Tooth abscess
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Injury, poisoning and procedural complications
Toxicity to various agents
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Nervous system disorders
Cerebrovascular accident
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Nervous system disorders
Dizziness
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Nervous system disorders
Syncope
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Renal and urinary disorders
Renal failure acute
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
Other adverse events
| Measure |
COHORT 1
n=2 participants at risk
Participants whose body surface area (BSA) is less than 1.7 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily.
|
COHORT 2
n=4 participants at risk
Participants whose body surface area (BSA) is between 1.7 m\^2 to 2.0 m\^2 inclusive All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily.
|
COHORT 3
n=4 participants at risk
Participants whose body surface area (BSA) is greater than 2.0 m\^2. All participants were given a fixed dose of 2.5 mg omacetaxine by subcutaneous injection twice daily.
|
|---|---|---|---|
|
Vascular disorders
Pallor
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Nervous system disorders
Dizziness
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Nervous system disorders
Dysaesthesia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
|
Nervous system disorders
Headache
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Nervous system disorders
Lethargy
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Nervous system disorders
Neuralgia
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Nervous system disorders
Syncope
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Psychiatric disorders
Insomnia
|
100.0%
2/2 • Number of events 3 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Psychiatric disorders
Sleep disorder
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Renal and urinary disorders
Renal failure
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Renal and urinary disorders
Renal failure acute
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
|
Renal and urinary disorders
Urethritis noninfective
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Renal and urinary disorders
Urinary retention
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Reproductive system and breast disorders
Scrotal oedema
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/2 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
100.0%
2/2 • Number of events 2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Vascular disorders
Hypotension
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Anaemia
|
100.0%
2/2 • Number of events 5 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 7 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Hypofibrinogenaemia
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Leukopenia
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 10 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Lymphopenia
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 7 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 6 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Neutropenia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 7 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 9 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
100.0%
2/2 • Number of events 2 • Day 1 up to Month 15
|
75.0%
3/4 • Number of events 7 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 10 • Day 1 up to Month 15
|
|
Blood and lymphatic system disorders
Thrombocytosis
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Cardiac disorders
Bradycardia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Cardiac disorders
Tachycardia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Endocrine disorders
Hypothyroidism
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Constipation
|
100.0%
2/2 • Number of events 2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 3 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 6 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Dry mouth
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Nausea
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 5 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Oral pain
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
General disorders
Injection site pain
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 5 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
General disorders
Injection site rash
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
General disorders
Injection site reaction
|
0.00%
0/2 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
General disorders
Mucosal dryness
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
General disorders
Oedema peripheral
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
General disorders
Pyrexia
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
General disorders
Temperature intolerance
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 3 • Day 1 up to Month 15
|
|
Immune system disorders
Hypersensitivity
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Infections and infestations
Injection site cellulitis
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Infections and infestations
Legionella infection
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Infections and infestations
Oral candidiasis
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Infections and infestations
Upper respiratory tract infection
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Investigations
Activated partial thromboplastin time prolonged
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Investigations
Blood calcium decreased
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Investigations
Blood lactate dehydrogenase decreased
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Investigations
International normalised ratio increased
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Investigations
Lymphocyte count decreased
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Investigations
Platelet count decreased
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 3 • Day 1 up to Month 15
|
|
Investigations
Weight decreased
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Investigations
Weight increased
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
50.0%
2/4 • Number of events 2 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Dehydration
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
75.0%
3/4 • Number of events 4 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Diabetes mellitus
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Gout
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
50.0%
1/2 • Number of events 2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
50.0%
1/2 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 2 • Day 1 up to Month 15
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/2 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/2 • Day 1 up to Month 15
|
25.0%
1/4 • Number of events 1 • Day 1 up to Month 15
|
0.00%
0/4 • Day 1 up to Month 15
|
Additional Information
Director, Clinical Research
Teva Branded Pharmaceutical Products, R&D Inc.
Phone: 1-888-483-8279
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor has the right 60 days before submission for publication to review/provide comments. If the Sponsor's review shows that potentially patentable subject matter would be disclosed, publication or public disclosure shall be delayed for up to 90 additional days in order for the Sponsor, or Sponsor's designees, to file the necessary patent applications. In multicenter trials, each PI will postpone single center publications until after disclosure or publication of multicenter data.
- Publication restrictions are in place
Restriction type: OTHER