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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)

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ClinicalTrials.gov Identifier: NCT02078960
Recruitment Status : Terminated (Inability to accrue additional sites and enroll an adequate number of subjects.)
First Posted : March 5, 2014
Results First Posted : January 14, 2019
Last Update Posted : January 14, 2019
Sponsor:
Information provided by (Responsible Party):
Teva Pharmaceutical Industries ( Teva Branded Pharmaceutical Products, R&D Inc. )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Other
Condition Chronic Myeloid Leukemia
Intervention Drug: Omacetaxine mepesuccinate
Enrollment 10
Recruitment Details 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.
Pre-assignment Details 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.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description 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. 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. 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.
Period Title: Treatment Period
Started 2 4 4
Completed [1] 0 1 1
Not Completed 2 3 3
Reason Not Completed
Not specified             0             0             1
Lack of Efficacy             0             1             0
Disease progression - accelerated phase             0             1             0
Disease progression - blast phase             1             0             0
Withdrawal by Subject             0             1             1
Adverse Event             1             0             0
Death             0             0             1
[1]
Treatment completed
Period Title: Follow-up Period
Started 2 2 1
Completed [1] 0 1 1
Not Completed 2 1 0
Reason Not Completed
Death             2             0             0
Lost to Follow-up             0             1             0
[1]
Monitored for progression and survival for at least 1 year following treatment.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Total
Hide Arm/Group Description 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. 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. 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 of all reporting groups
Overall Number of Baseline Participants 2 4 4 10
Hide Baseline Analysis Population Description
Enrolled patients
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 4 participants 4 participants 10 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
1
  50.0%
3
  75.0%
1
  25.0%
5
  50.0%
>=65 years
1
  50.0%
1
  25.0%
3
  75.0%
5
  50.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 2 participants 4 participants 4 participants 10 participants
66.5  (9.19) 46.3  (22.87) 68.3  (17.31) 59.1  (20.16)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 4 participants 4 participants 10 participants
Female
0
   0.0%
1
  25.0%
1
  25.0%
2
  20.0%
Male
2
 100.0%
3
  75.0%
3
  75.0%
8
  80.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 4 participants 4 participants 10 participants
Hispanic or Latino
1
  50.0%
1
  25.0%
0
   0.0%
2
  20.0%
Not Hispanic or Latino
1
  50.0%
2
  50.0%
4
 100.0%
7
  70.0%
Unknown or Not Reported
0
   0.0%
1
  25.0%
0
   0.0%
1
  10.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 4 participants 4 participants 10 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
1
  25.0%
0
   0.0%
1
  10.0%
White
1
  50.0%
2
  50.0%
4
 100.0%
7
  70.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
  50.0%
1
  25.0%
0
   0.0%
2
  20.0%
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 2 participants 4 participants 4 participants 10 participants
55.1  (7.78) 73.7  (4.58) 105.3  (18.34) 82.6  (23.63)
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 2 participants 4 participants 4 participants 10 participants
167.0  (1.41) 167.3  (8.02) 169.7  (9.55) 168.2  (7.33)
Body Surface Area (BSA)  
Mean (Standard Deviation)
Unit of measure:  M^2
Number Analyzed 2 participants 4 participants 4 participants 10 participants
1.6  (0.08) 1.8  (0.09) 2.2  (0.25) 2.0  (0.30)
Phase of Chronic Myeloid Leukemia (CML) at Study Entry   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 4 participants 4 participants 10 participants
Chronic Phase (CP)
2
 100.0%
3
  75.0%
4
 100.0%
9
  90.0%
Accelerated Phase (AP)
0
   0.0%
1
  25.0%
0
   0.0%
1
  10.0%
[1]
Measure Description:

CP: patients typically have less than 10% blasts in their blood or bone marrow samples. Patients usually have fairly mild symptoms and usually respond to standard treatments.

AP: patients have >=1 of the following:

  • Blood samples have 15% or more, but fewer than 30% blasts
  • Basophils make up 20% or more of the blood
  • Blasts and promyelocytes combined make up 30% or more of the blood
  • Very low platelet counts (100*1,000/mm^3 or less) that are not caused by treatment
  • New chromosome changes in the leukemia cells with the Philadelphia chromosome.
1.Primary Outcome
Title Number of Participants Who Achieved a Major Response at Any Time During Treatment
Hide Description

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).

Time Frame Day 1 up to Month 15 (longest treatment duration)
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 2 4 4
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
1
  25.0%
2
  50.0%
2.Secondary Outcome
Title Longest Duration of Response At Study Termination
Hide Description 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.
Time Frame Day 1 to Day 541 (longest progression/survival follow-up)
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants who had a response
Arm/Group Title All Enrolled Participants
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: days
316
3.Secondary Outcome
Title Number of Participants Who Had a Molecular Response at Any Time During Treatment
Hide Description 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).
Time Frame Day 1 up to Month 15
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants
Arm/Group Title All Enrolled Participants
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Measure Type: Count of Participants
Unit of Measure: Participants
1
  10.0%
4.Secondary Outcome
Title Number of Participants Who Were Alive and Progression-Free at Study Termination
Hide Description 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.
Time Frame Day 1 to Day 541 (longest progression/survival follow-up)
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants
Arm/Group Title All Enrolled Participants
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Measure Type: Count of Participants
Unit of Measure: Participants
4
  40.0%
5.Secondary Outcome
Title Number of Participants Who Were Alive at Study Termination
Hide Description 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.
Time Frame Day 1 to Day 541 (longest progression/survival follow-up)
Hide Outcome Measure Data
Hide Analysis Population Description
Enrolled participants
Arm/Group Title All Enrolled Participants
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Measure Type: Count of Participants
Unit of Measure: Participants
7
  70.0%
6.Secondary Outcome
Title Maximum Observed Plasma Concentration (Cmax) for Omacetaxine
Hide Description Maximum observed plasma drug concentration (Cmax) by inspection (without interpolation). Nominal PK sampling times were used.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
PK analysis set
Arm/Group Title Total Omacetaxine
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: ng/mL
23.88  (13.32)
7.Secondary Outcome
Title Time to Maximum Observed Plasma Concentration (Cmax) for Omacetaxine
Hide Description Time to maximum observed plasma drug concentration (Cmax) by inspection (without interpolation). Nominal PK sampling times were used.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
PK analysis set
Arm/Group Title Total Omacetaxine
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: hour
0.25
(0.25 to 1.0)
8.Secondary Outcome
Title Area Under the Drug Concentration by Time Curve From Time 0 to the Time of the Last Measurable Drug Concentration (AUC0-t) for Omacetaxine
Hide Description Nominal PK sampling times were used.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
PK analysis set
Arm/Group Title Total Omacetaxine
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: ng*hr/mL
150.2  (55.8)
9.Secondary Outcome
Title Area Under the Drug Concentration by Time Curve From Time 0 to Infinity (AUC0-inf) for Omacetaxine
Hide Description Nominal PK sampling times were used.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
PK analysis set
Arm/Group Title Total Omacetaxine
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: ng*hr/mL
154.8  (60.8)
10.Secondary Outcome
Title Terminal Elimination Half-Life (t1/2) for Omacetaxine
Hide Description Nominal PK sampling times were used.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
PK analysis set
Arm/Group Title Total Omacetaxine
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: hour
13.3  (1.6)
11.Secondary Outcome
Title Total Oral Clearance (CL/F) for Omacetaxine
Hide Description Nominal PK sampling times were used.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
PK analysis set
Arm/Group Title Total Omacetaxine
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: L/hour
18.1  (6.1)
12.Secondary Outcome
Title Apparent Volume of Distribution (V/F) for Omacetaxine
Hide Description Nominal PK sampling times were used.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
PK analysis set
Arm/Group Title Total Omacetaxine
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: L
344.9  (117.0)
13.Secondary Outcome
Title Participants With Treatment-Emergent Adverse Events (TEAEs)
Hide Description 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.
Time Frame Day 1 up to Month 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis set
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 2 4 4
Measure Type: Count of Participants
Unit of Measure: Participants
Any TEAE
2
 100.0%
4
 100.0%
4
 100.0%
TEAE of Severity Grade 3
0
   0.0%
3
  75.0%
0
   0.0%
TEAE of Severity Grade 4
1
  50.0%
1
  25.0%
3
  75.0%
TEAE of Severity Grade 5
1
  50.0%
0
   0.0%
1
  25.0%
Treatment-related TEAE
2
 100.0%
4
 100.0%
4
 100.0%
Deaths
2
 100.0%
0
   0.0%
1
  25.0%
Serious AE
2
 100.0%
2
  50.0%
4
 100.0%
Withdrawn from treatment due to a TEAE
1
  50.0%
0
   0.0%
0
   0.0%
Time Frame Day 1 up to Month 15
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title COHORT 1 COHORT 2 COHORT 3
Hide Arm/Group Description 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. 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. 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.
All-Cause Mortality
COHORT 1 COHORT 2 COHORT 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/2 (100.00%)      0/4 (0.00%)      1/4 (25.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
COHORT 1 COHORT 2 COHORT 3
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/2 (100.00%)      2/4 (50.00%)      4/4 (100.00%)    
Blood and lymphatic system disorders       
Anaemia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Neutropenia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Thrombocytopenia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Cardiac disorders       
Cardiac failure congestive  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Gastrointestinal disorders       
Abdominal pain  1  0/2 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1
Abdominal pain upper  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Diarrhoea  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Gastrointestinal haemorrhage  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Oral pain  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Stomatitis  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Vomiting  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
General disorders       
Death  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Non-cardiac chest pain  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Pain  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Pyrexia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Infections and infestations       
Abscess limb  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Gastroenteritis  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Herpes zoster  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Pneumonia  1  1/2 (50.00%)  2 0/4 (0.00%)  0 2/4 (50.00%)  2
Septic shock  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Tooth abscess  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Urinary tract infection  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Injury, poisoning and procedural complications       
Toxicity to various agents  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Metabolism and nutrition disorders       
Hyperkalaemia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Musculoskeletal and connective tissue disorders       
Pain in extremity  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Nervous system disorders       
Cerebrovascular accident  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Dizziness  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Syncope  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Renal and urinary disorders       
Nephrolithiasis  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Renal failure acute  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Respiratory, thoracic and mediastinal disorders       
Acute respiratory failure  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Pleural effusion  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Respiratory failure  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
1
Term from vocabulary, MedDRA 16.1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
COHORT 1 COHORT 2 COHORT 3
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/2 (100.00%)      4/4 (100.00%)      4/4 (100.00%)    
Blood and lymphatic system disorders       
Anaemia  1  2/2 (100.00%)  5 1/4 (25.00%)  1 2/4 (50.00%)  7
Febrile neutropenia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1
Hypofibrinogenaemia  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Leukopenia  1  0/2 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  10
Lymphopenia  1  0/2 (0.00%)  0 1/4 (25.00%)  7 1/4 (25.00%)  6
Neutropenia  1  1/2 (50.00%)  1 1/4 (25.00%)  7 1/4 (25.00%)  9
Pancytopenia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Thrombocytopenia  1  2/2 (100.00%)  2 3/4 (75.00%)  7 2/4 (50.00%)  10
Thrombocytosis  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Cardiac disorders       
Bradycardia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Tachycardia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Endocrine disorders       
Hypothyroidism  1  1/2 (50.00%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1
Gastrointestinal disorders       
Abdominal pain  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Constipation  1  2/2 (100.00%)  2 1/4 (25.00%)  3 1/4 (25.00%)  2
Diarrhoea  1  0/2 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  6
Dry mouth  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Dyspepsia  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Nausea  1  1/2 (50.00%)  1 1/4 (25.00%)  5 2/4 (50.00%)  2
Oral pain  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Toothache  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Vomiting  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
General disorders       
Injection site pain  1  0/2 (0.00%)  0 1/4 (25.00%)  5 0/4 (0.00%)  0
Injection site rash  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Injection site reaction  1  0/2 (0.00%)  0 2/4 (50.00%)  2 0/4 (0.00%)  0
Mucosal dryness  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Oedema peripheral  1  1/2 (50.00%)  1 1/4 (25.00%)  1 1/4 (25.00%)  1
Pyrexia  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Temperature intolerance  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Hepatobiliary disorders       
Hyperbilirubinaemia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  3
Immune system disorders       
Hypersensitivity  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Infections and infestations       
Injection site cellulitis  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Legionella infection  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Oral candidiasis  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Upper respiratory tract infection  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Injury, poisoning and procedural complications       
Contusion  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Limb injury  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Spinal fracture  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Investigations       
Activated partial thromboplastin time prolonged  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Alanine aminotransferase increased  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Blood calcium decreased  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Blood lactate dehydrogenase decreased  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
International normalised ratio increased  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Lymphocyte count decreased  1  1/2 (50.00%)  1 1/4 (25.00%)  1 2/4 (50.00%)  2
Neutrophil count decreased  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Platelet count decreased  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  3
Weight decreased  1  0/2 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1
Weight increased  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Metabolism and nutrition disorders       
Decreased appetite  1  0/2 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  2
Dehydration  1  1/2 (50.00%)  1 0/4 (0.00%)  0 3/4 (75.00%)  4
Diabetes mellitus  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Gout  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Hyperglycaemia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Hyperphosphataemia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Hyperuricaemia  1  0/2 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1
Hypomagnesaemia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Hyponatraemia  1  1/2 (50.00%)  2 0/4 (0.00%)  0 0/4 (0.00%)  0
Hypophosphataemia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1
Musculoskeletal and connective tissue disorders       
Arthralgia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 1/4 (25.00%)  2
Joint swelling  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Muscular weakness  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Myalgia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Neck pain  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Pain in extremity  1  1/2 (50.00%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0
Nervous system disorders       
Dizziness  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2
Dysaesthesia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Dysgeusia  1  0/2 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  2
Headache  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Lethargy  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Neuralgia  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Syncope  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Psychiatric disorders       
Anxiety  1  0/2 (0.00%)  0 1/4 (25.00%)  2 0/4 (0.00%)  0
Insomnia  1  2/2 (100.00%)  3 0/4 (0.00%)  0 0/4 (0.00%)  0
Sleep disorder  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Renal and urinary disorders       
Renal failure  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Renal failure acute  1  0/2 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  2
Urethritis noninfective  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Urinary retention  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Reproductive system and breast disorders       
Scrotal oedema  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Atelectasis  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Cough  1  0/2 (0.00%)  0 2/4 (50.00%)  2 1/4 (25.00%)  1
Dyspnoea  1  2/2 (100.00%)  2 0/4 (0.00%)  0 0/4 (0.00%)  0
Epistaxis  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Pleural effusion  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Rhinorrhoea  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Skin and subcutaneous tissue disorders       
Alopecia  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Dermatitis  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Petechiae  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Pruritus  1  0/2 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0
Rash  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Skin hypopigmentation  1  0/2 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1
Skin ulcer  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Vascular disorders       
Hypotension  1  1/2 (50.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0
Pallor  1  0/2 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  2
1
Term from vocabulary, MedDRA 16.1
Indicates events were collected by systematic assessment
Small sample size made it not possible to make meaningful comparisons between cohorts.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: Director, Clinical Research
Organization: Teva Branded Pharmaceutical Products, R&D Inc.
Phone: 1-888-483-8279
Responsible Party: Teva Pharmaceutical Industries ( Teva Branded Pharmaceutical Products, R&D Inc. )
ClinicalTrials.gov Identifier: NCT02078960     History of Changes
Other Study ID Numbers: C41443/2057
2013-005320-42 ( EudraCT Number )
First Submitted: February 28, 2014
First Posted: March 5, 2014
Results First Submitted: December 19, 2018
Results First Posted: January 14, 2019
Last Update Posted: January 14, 2019