Chronic Myeloid LeukemiaPhase 2NCT00462943
Open Label Study of Subcutaneous Homoharringtonine (Omacetaxine Mepesuccinate) in Patients With Advanced CML
A Phase 2 study testing Omacetaxine mepesuccinate in people with Chronic Myeloid Leukemia, run by Teva Branded Pharmaceutical Products R&D, Inc. at 29 sites in 10 countries.
Trial summary
A Phase II open-label trial of subcutaneous HHT (omacetaxine mepesuccinate) in the treatment of patients who are resistant to or intolerant to Tyrosine Kinase Inhibitors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloid Leukemia
- What is being tested?
- Omacetaxine mepesuccinate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 29 sitesin 10 countries
- Study dates
- 7 Mar 2007to 27 Jun 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients, age 18 years or older
- Philadelphia chromosome (Ph) positive chronic myelogenous leukemia in either chronic, accelerated, or blast phase
- Patients will have either failed, demonstrated intolerance, or a combination of prior failure and intolerance, to prior treatments with at least two tyrosine kinase inhibitors (TKI's). Failure of TKI treatment may either be primary (never achieved a response) or secondary resistance (loss of response).
- Acceptable Renal and Liver Function
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
- Sexually active patients and their partners must use an effective double barrier method of contraception
Who cannot join
- New York Heart Association classification (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition
- Myocardial infarction in the previous 12 weeks.
- Other concurrent illness which would preclude study conduct and assessment
- uncontrolled and active infection, and positive HIV or positive HTLV I/II status, whether on treatment or not.
- Pregnant or lactating.
- Any medical or psychiatric condition, which may compromise the ability to give written informed consent or to comply with the study protocol.
- Lymphoid Ph+ blast crisis
- Patient is enrolled in another clinical investigation within 30 days of enrollment or is receiving another investigational agent
Trial locations and contacts
29 sites in 10 countries.
29 sites in 10 countries
France9 sites
Teva Investigational Site 329
Bordeaux, 33076
Teva Investigational Site 321
Le Chesnay, 78157
Teva Investigational Site 322
Lille, 59000
Teva Investigational Site 320
Lyon, 69437
Teva Investigational Site 324
Nice, 06202
Teva Investigational Site 328
Paris, 75475
Teva Investigational Site 323
Poitiers, 86021
Teva Investigational Site 327
Strasbourg, 67100
Teva Investigational Site 325
Toulouse, 31059
United States8 sites
Teva Investigational Site 311
Baltimore, Maryland, 21201
Teva Investigational Site 308
Beech Grove, Indiana, 46107
Teva Investigational Site 305
Buffalo, New York, 14263
Teva Investigational Site 301
Houston, Texas, 77030
Teva Investigational Site 303
Los Angeles, California, 90033
Teva Investigational Site 310
Philadelphia, Pennsylvania, 19111
Teva Investigational Site 314
Seattle, Washington, 98109
Teva Investigational Site 302
The Bronx, New York, 10466
Canada2 sites
Teva Investigational Site 313
Montreal, H3a 1a1
Teva Investigational Site 309
Toronto, M5G 2M9
Germany2 sites
Teva Investigational Site 331
Berlin, 10117
Teva Investigational Site 330
Mannheim, 68169
India2 sites
Teva Investigational Site 371
Hyderabad, 500082
Teva Investigational Site 370
Mumbai, 400 014
Poland2 sites
Teva Investigational Site 360
Gdansk, 80-952
Teva Investigational Site 361
Warsaw, 02776
About this study
Official title: A Phase II Open-Label Study of the Subcutaneous Administration of Homoharringtonine. (Omacetaxine Mepesuccinate; OMA) in the Treatment of Patients With Chronic Myeloid. Leukemia (CML) Who Have Failed or Are Intolerant to Tyrosine Kinase Inhibitor Therapy
A Phase II open-label trial of subcutaneous HHT (omacetaxine mepesuccinate) in the treatment of patients who are resistant to or intolerant to Tyrosine Kinase Inhibitors.
Read the detailed description
This will be an open label, multicenter study of subcutaneous HHT (omacetaxine mepesuccinate) therapy of patients with chronic myeloid leukemia (CML) in chronic, accelerated, or blast phase who have failed or are intolerant to tyrosine kinase inhibitor therapy. Patients will be treated with induction course cycles consisting of subcutaneous (SC) HHT 1.25 mg/m² twice daily for 14 consecutive days every 28 days. Patients will be evaluated every 7 days with complete blood and platelet counts while undergoing induction therapy; the number of consecutive doses of HHT or intervals between subsequent cycles may be adjusted, as clinically indicated, according to guidelines provided in the treatment plan.
- Conditions
- Chronic Myeloid Leukemia
- Being tested
- Omacetaxine mepesuccinate
- Keywords
- CML, HHT, Homoharringtonine, Omacetaxine
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Omacetaxine mepesuccinateDrug
Also called Homoharringtonine, HHT, Synribo, OMA
Induction: 1.25mg/m^2 subcutaneously twice daily for 14 consecutive days, every 28 days.
Maintenance: 1.25mg/m^2 subcutaneously twice daily for 7 consecutive days, every 28 days.
Response targets during induction vary by chronic myeloid leukemia (CML) subclass (chronic, accelerated, or blast phase). Participants will complete at least one cycle (14 days treatment of a 28 day cycle) of induction therapy before changing to maintenance therapy. Participants not demonstrating evidence of clinical response after 6 induction cycles will be considered for removal from the study.
| Study group | Role | What they receive |
|---|---|---|
| OMA | Experimental | Omacetaxine mepesuccinate (OMA) Induction: 1.25mg/m^2 subcutaneously twice daily for 14 consecutive days, every 28 days for up to six cycles.
Omacetaxine mepesuccinate (OMA) Maintenance: 1.25mg/m^2 subcutaneously twice daily for 7 consecutive days, every 28 days for up to 24 months. Receives: Drug: Omacetaxine mepesuccinate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population
Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses.
Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last >= 8 weeks to be considered meaningful.
Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Measured: Day 1 up to 6 months
Percentage of Participants Achieving a Major Cytogenetic Response by Subpopulation and Total Population
Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses.
Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available.
Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows >0% - 35% Ph+ cells.
Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Measured: Day 1 up to 9 months
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
TEAE are any untoward events that were newly occurring or worsening from Baseline.
Treatment related toxicity was considered by the investigator to be unrelated, possibly, probably or unknown related to study drug. Severity was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v3.0 on the following scale: Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe, Grade 4 = life-threatening, Grade 5 = death. A serious adverse event (SAE) is any untoward medical occurrence that is fatal or life-threatening; results in persistent or significant disability or incapacity; requires or prolongs in-patient hospitalization; is a congenital anomaly/birth defect in the offspring of a patient; and conditions not included in the above that may jeopardize the patient or may require intervention to prevent one of the outcomes listed above.
A participant is only counted once in each category (at worst severity or strongest relationship).
Measured: up to 4 years
Secondary outcomes (14)
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Cytogenetic response categories:
- Complete: 0% Ph+ cells
- Partial: >0%-35% Ph+ cells
- Minor: >35%-65% Ph+ cells
- Minimal: >65%-95% Ph+ cells
- No Response: >95% Ph+ cells
- Unevaluable: <20 metaphases were examined and/or response could not be assigned
Measured: Day 1 up to Month 9
Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene GUS
MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region [BCR] gene and Abelson proto-oncogene [ABL] genes). This analysis used the standard gene GUS. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.
Measured: Day 1 up to Month 6
Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene ABL
MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region [BCR] gene and Abelson proto-oncogene [ABL] genes). This analysis used the standard gene ABL. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.
Measured: Day 1 up to Month 6
Percentage of Participants in Each Hematologic Response Category
Complete Response (CHR)
- Chronic phase must last at least 8 weeks: WBC <10*10^9/liter, platelets <450*10^9/liter, myelocytes + metamyelocytes <5% in blood, no blasts or promyelocytes in blood, <20% basophils in peripheral blood, no extramedullary involvement.
- Accelerated and Blast phase must last at least 4 weeks: absolute neutrophil count 1.5*10^9/liter, platelets 100*10^9/liter, no blood blasts, bone marrow blasts <5%, no extramedullary disease.
Partial Response - CHR plus one or more of the following:
- Persistence of splenomegaly with a reduction of ≥50% from pre-treatment
- Platelets > 450*10^9/L
- Presence of immature cells in the peripheral blood
- 5% to 25% blasts in the bone marrow
- If extra-medullary disease pre-treatment, reduction by ≥50% Hematologic Improvement - CHR, except allowing persistent thrombocytopenia (<100*10^9/L), and a few immature cells No evidence of leukemia: Morphologic leukemia-free state, defined as <5% bone marrow blasts.
Measured: Day 1 up to Month 6
Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response
Clinical response was defined by disease phase and based on evaluations by the independent Data Monitoring Committee (DMC).
Chronic Phase subgroup: achieving a complete hematologic response and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed).
Accelerated Phase and Blast Phase subgroups: achieving complete hematologic response, no evidence of leukemia, return to chronic phase, and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed).
Measured: Day 1 up to Month 9
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
Summarization is based on the best of the individual response assessments. Not assessable indicates that the participant either had no baseline assessment or the % mutation could not be determined in the post-baseline assessment(s).
Measured: Day 1 up to Month 9
Number of Treatment Cycles Needed to Achieve Best Hematologic Response
Induction therapy was administered for 14 consecutive days for each 28 days cycle, for up to 6 cycles. All treatment arms were given omacetaxine mepesuccinate via subcutaneous (SC) administration at 1.25 mg/m^2 twice a day (BID) for the 14 consecutive days.
Measured: Day 1 up to Month 6
Number of Treatment Cycles Needed to Achieve Best Cytogenetic Response
Measured: Day 1 up to Month 9
Kaplan-Meier Estimates for Time to Onset of Best Hematologic Response
Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day.
Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last >= 8 weeks to be considered meaningful.
Measured: Day 1 up to Month 6
Kaplan-Meier Estimates for Time to Onset of Best Cytogenetic Response
Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day.
Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available.
Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows >0% - 35% Ph+ cells.
Measured: Day 1 up to Month 9
Kaplan-Meier Estimates for Duration of Best Hematologic Response
Duration of response is defined as the time from first reported date of hematologic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.
Measured: up to four years
Kaplan-Meier Estimates for Duration of Best Cytogenetic Response
Duration of response is defined as the time from first reported date of cytogenetic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.
Measured: up to four years
Kaplan-Meier Estimates for Time to Disease Progression
Time to disease progression is defined as the time from the initiation of treatment until the onset date of death, the development of CML accelerated phase or blast phase, or the loss of complete hematologic response or major cytogenetic response, whichever came first. Participants were censored only if they did not have progression or if they discontinued treatment for reasons other than AE, progression or death.
Measured: up to 4 years
Kaplan-Meier Estimates for Overall Survival
Overall survival is defined as the time from the initiation of treatment until death from any cause or the last day of participant contact or evaluation for participants that were lost to follow-up. Participants were censored t the last recorded contract or evaluation when a participant was alive at time of analysis. A quarterly phone survey was conducted to collect survival data for participants who discontinued from the study.
Measured: up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2007 (estimated)
- Study start7 Mar 2007
- Primary completion4 Aug 2009
- Study completion27 Jun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cephalon, ChemGenex Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Jorge Cortes, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00462943
- Sponsor's protocol ID
- CGX-635-CML-203
- Other IDs
- 2007-001286-15
- Status last verified
- Dec 2021
- First submitted
- 17 Apr 2007
- Last updated
- 28 Dec 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Cortes J, Digumarti R, Parikh PM, Wetzler M, Lipton JH, Hochhaus A, Craig AR, Benichou AC, Nicolini FE, Kantarjian HM; Omacetaxine 203 Study Group. Phase 2 study of subcutaneous omacetaxine mepesuccinate for chronic-phase chronic myeloid leukemia patients resistant to or intolerant of tyrosine kinase inhibitors. Am J Hematol. 2013 May;88(5):350-4. doi: 10.1002/ajh.23408. Epub 2013 Mar 7.
PubMed 23468307
Study at a glance
From the registry record, last updated by the sponsor on 28 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Open-Label Study of the Subcutaneous Administration of Homoharringtonine. (Omacetaxine Mepesuccinate; OMA) in the Treatment of Patients With Chronic Myeloid. Leukemia (CML) Who Have Failed or Are Intolerant to Tyrosine Kinase Inhibitor Therapy
- ClinicalTrials.gov ID
- NCT00462943
- Acronym / sponsor ID
- CGX-635-CML-203
- Conditions
- Chronic Myeloid Leukemia
- Keywords
- CML, HHT, Homoharringtonine, Omacetaxine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Cephalon, ChemGenex Pharmaceuticals
- Study start
- 7 Mar 2007 (actual)
- Primary completion
- 4 Aug 2009 (actual)
- Study completion
- 27 Jun 2013 (actual)
- First posted
- 19 Apr 2007 (estimated)
- Results
- Posted 30 Jun 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase II open-label trial of subcutaneous HHT (omacetaxine mepesuccinate) in the treatment of patients who are resistant to or intolerant to Tyrosine Kinase Inhibitors.
Detailed description
This will be an open label, multicenter study of subcutaneous HHT (omacetaxine mepesuccinate) therapy of patients with chronic myeloid leukemia (CML) in chronic, accelerated, or blast phase who have failed or are intolerant to tyrosine kinase inhibitor therapy. Patients will be treated with induction course cycles consisting of subcutaneous (SC) HHT 1.25 mg/m² twice daily for 14 consecutive days every 28 days. Patients will be evaluated every 7 days with complete blood and platelet counts while undergoing induction therapy; the number of consecutive doses of HHT or intervals between subsequent cycles may be adjusted, as clinically indicated, according to guidelines provided in the treatment plan.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OMA | Experimental | Drug: Omacetaxine mepesuccinateOmacetaxine mepesuccinate (OMA) Induction: 1.25mg/m^2 subcutaneously twice daily for 14 consecutive days, every 28 days for up to six cycles. Omacetaxine mepesuccinate (OMA) Maintenance: 1.25mg/m^2 subcutaneously twice daily for 7 consecutive days, every 28 days for up to 24 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female patients, age 18 years or older
- Philadelphia chromosome (Ph) positive chronic myelogenous leukemia in either chronic, accelerated, or blast phase
- Patients will have either failed, demonstrated intolerance, or a combination of prior failure and intolerance, to prior treatments with at least two tyrosine kinase inhibitors (TKI's). Failure of TKI treatment may either be primary (never achieved a response) or secondary resistance (loss of response).
- Acceptable Renal and Liver Function
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
Key exclusion criteria (8 listed)
- New York Heart Association classification (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition
- Myocardial infarction in the previous 12 weeks.
- Other concurrent illness which would preclude study conduct and assessment
- uncontrolled and active infection, and positive HIV or positive HTLV I/II status, whether on treatment or not.
- Pregnant or lactating.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Omacetaxine mepesuccinateAlso: Homoharringtonine, HHT, Synribo, OMAInduction: 1.25mg/m^2 subcutaneously twice daily for 14 consecutive days, every 28 days. Maintenance: 1.25mg/m^2 subcutaneously twice daily for 7 consecutive days, every 28 days. Response targets during induction vary by chronic myeloid leukemia (CML) subclass (chronic, accelerated, or blast phase). Participants will complete at least one cycle (14 days treatment of a 28 day cycle) of induction therapy before changing to maintenance therapy. Participants not demonstrating evidence of clinical response after 6 induction cycles will be considered for removal from the study. | Drug | OMA |
Primary outcome
Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population
Time frame: Day 1 up to 6 months
Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last >= 8 weeks to be considered meaningful. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Percentage of Participants Achieving a Major Cytogenetic Response by Subpopulation and Total Population
Time frame: Day 1 up to 9 months
Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available. Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows >0% - 35% Ph+ cells. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Time frame: up to 4 years
TEAE are any untoward events that were newly occurring or worsening from Baseline. Treatment related toxicity was considered by the investigator to be unrelated, possibly, probably or unknown related to study drug. Severity was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v3.0 on the following scale: Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe, Grade 4 = life-threatening, Grade 5 = death. A serious adverse event (SAE) is any untoward medical occurrence that is fatal or life-threatening; results in persistent or significant disability or incapacity; requires or prolongs in-patient hospitalization; is a congenital anomaly/birth defect in the offspring of a patient; and conditions not included in the above that may jeopardize the patient or may require intervention to prevent one of the outcomes listed above. A participant is only counted once in each category (at worst severity or strongest relationship).
14 secondary outcomes
Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Time frame: Day 1 up to Month 9
Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene GUS
Time frame: Day 1 up to Month 6
Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene ABL
Time frame: Day 1 up to Month 6
Percentage of Participants in Each Hematologic Response Category
Time frame: Day 1 up to Month 6
Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response
Time frame: Day 1 up to Month 9
Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
Time frame: Day 1 up to Month 9
Number of Treatment Cycles Needed to Achieve Best Hematologic Response
Time frame: Day 1 up to Month 6
Number of Treatment Cycles Needed to Achieve Best Cytogenetic Response
Time frame: Day 1 up to Month 9
Kaplan-Meier Estimates for Time to Onset of Best Hematologic Response
Time frame: Day 1 up to Month 6
Kaplan-Meier Estimates for Time to Onset of Best Cytogenetic Response
Time frame: Day 1 up to Month 9
Kaplan-Meier Estimates for Duration of Best Hematologic Response
Time frame: up to four years
Kaplan-Meier Estimates for Duration of Best Cytogenetic Response
Time frame: up to four years
Kaplan-Meier Estimates for Time to Disease Progression
Time frame: up to 4 years
Kaplan-Meier Estimates for Overall Survival
Time frame: up to 4 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Dec 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 23468307Cortes J, Digumarti R, Parikh PM, Wetzler M, Lipton JH, Hochhaus A, Craig AR, Benichou AC, Nicolini FE, Kantarjian HM; Omacetaxine 203 Study Group. Phase 2 study of subcutaneous omacetaxine mepesuccinate for chronic-phase chronic myeloid leukemia patients resistant to or intolerant of tyrosine kinase inhibitors. Am J Hematol. 2013 May;88(5):350-4. doi: 10.1002/ajh.23408. Epub 2013 Mar 7.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jorge Cortes, MD principal investigator
M.D. Anderson Cancer Center
29 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Omacetaxine mepesuccinate in people with Chronic Myeloid Leukemia, run by Teva Branded Pharmaceutical Products R&D, Inc. at 29 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in France, United States, Canada, Germany, India and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Jun 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population (time frame: Day 1 up to 6 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00462943, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Dec 2021.