Childhood Chronic Myelogenous LeukemiaPhase 2NCT00006364
Homoharringtonine in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia
A Phase 2 study testing omacetaxine mepesuccinate, pharmacological study and laboratory biomarker analysis in people with Childhood Chronic Myelogenous Leukemia and Chronic Myelogenous Leukemia, BCR-ABL1 Positive, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Phase II trial to study the effectiveness of homoharringtonine in treating patients who have chronic phase chronic myelogenous leukemia. Drugs used in chemotherapy, such as homoharringtonine, work in different ways to stop cancer cells from dividing so they stop growing or die Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Childhood Chronic Myelogenous Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Phase Chronic Myelogenous Leukemia
- What is being tested?
- omacetaxine mepesuccinate, pharmacological study and laboratory biomarker analysis
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 1999to Sep 2005
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of chronic phase chronic myelogenous leukemia (CML), as defined by the following:
- Less than 15% blasts in the peripheral blood (PB) or bone marrow (BM)
- Less than 20% basophils in the PB or BM
- Platelet count > 100,000/mm^3 (unless related to therapy)
- Absence of clonal evolution*
- Philadelphia chromosome- OR BCR/ABL-positive disease by cytogenetics, fluorescence in situ hybridization, or polymerase chain reaction
Show all 21Show fewer
- Failed prior therapy with imatinib mesylate, as defined by any of the following:
- Failed to achieve or have lost a complete hematologic remission after 3 months of therapy
- Failed to achieve or have lost at least a minimal cytogenetic response after 6 months of therapy
- Failed to achieve or have lost a major or complete cytogenetic response after 12 months of therapy
- Unable to tolerate imatinib mesylate despite adequate dose adjustment
- Failed no more than 2 prior treatment regimens (in addition to imatinib mesylate)
- Treatment with hydroxyurea is not considered one regimen
- Ineligible for known regimens or protocols of higher efficacy or priority
- Performance status - Zubrod 0-2
- At least 2 months
- Bilirubin no greater than 2.0 mg/dL
- Creatinine less than 2.0 mg/dL
- No New York Heart Association class III or IV heart disease
- Not pregnant or nursing
- Fertile patients must use effective contraception
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I and Pilot Study of Subcutaneous Homoharringtonine in Chronic Myelogenous Leukemia
Phase II trial to study the effectiveness of homoharringtonine in treating patients who have chronic phase chronic myelogenous leukemia. Drugs used in chemotherapy, such as homoharringtonine, work in different ways to stop cancer cells from dividing so they stop growing or die
Read the detailed description
OBJECTIVES:
- Determine the maximum tolerated dose of homoharringtonine in patients with transformed phases of chronic myelogenous leukemia (CML). (Phase I completed as of 2/11/2004.) II. Determine the toxicity profile of this drug in these patients. III. Determine the response duration in patients with chronic phase CML treated with this drug.
IV. Compare the pharmacokinetics of this drug administered as a continuous infusion vs subcutaneously in these patients.
OUTLINE: This is a pilot, dose-escalation study. (Phase I completed as of 2/11/2004.)
Remission induction therapy: Patients receive remission induction therapy comprising homoharringtonine IV continuously over 24 hours on day 1 and then subcutaneously (SC) twice daily on days 2-14 for course 1. Subsequent courses of remission induction therapy comprise homoharringtonine SC twice daily on days 1-14. Treatment continues monthly for at least 2 courses.
Maintenance therapy: Patients with complete hematologic remission receive maintenance therapy comprising homoharringtonine SC twice daily on days 1-7 monthly for 3 years in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of homoharringtonine until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose at which 2 of 6 patients experience dose-limiting toxicity. An additional cohort of 25-30 patients with chronic phase chronic myelogenous leukemia receives remission induction and maintenance therapy as above at the MTD. (Phase I completed as of 2/11/2004.)
Patients are followed every 3 months.
PROJECTED ACCRUAL: A maximum of 50 patients will be accrued for this study.
- Conditions
- Childhood Chronic Myelogenous Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Phase Chronic Myelogenous Leukemia, Relapsing Chronic Myelogenous Leukemia
- Being tested
- omacetaxine mepesuccinate, pharmacological study, laboratory biomarker analysis
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Chronic-Phase
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
omacetaxine mepesuccinateDrug
Also called CGX-635, homoharringtonine
Given IV or SC
Also called pharmacological studies
Correlative studies
laboratory biomarker analysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (omacetaxine mepesuccinate) | Experimental | Remission induction therapy: Patients receive remission induction therapy comprising homoharringtonine IV continuously over 24 hours on day 1 and then subcutaneously (SC) twice daily on days 2-14 for course 1. Subsequent courses of remission induction therapy comprise homoharringtonine SC twice daily on days 1-14. Treatment continues monthly for at least 2 courses.
Maintenance therapy: Patients with complete hematologic remission receive maintenance therapy comprising homoharringtonine SC twice daily on days 1-7 monthly for 3 years in the absence of disease progression or unacceptable toxicity. Receives: Drug: omacetaxine mepesuccinate; Other: pharmacological study; Other: laboratory biomarker analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum-tolerated dose (MTD) of homoharringtonine as assessed by the National Cancer Institute (NCI) Common Terminology Criteria (CTC)
Measured: 14 days
Complete hematologic remission (CHR) defined as at least 4 weeks of bone marrow (less than 5% blasts) and peripheral blood with WBC < 10 x 10^9/L and no peripheral blasts, promyelocytes, or myelocytes
Using a Bayesian approach.
Measured: Up to 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startNov 1999
- Primary completionSep 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Childhood Chronic Myelogenous LeukemiaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- 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
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Jorge Cortes, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00006364
- Sponsor's protocol ID
- NCI-2012-02360
- Other IDs
- ID 99-032, N01CM17003, CDR0000068237
- Status last verified
- Jan 2013
- First submitted
- 4 Oct 2000
- Last updated
- 23 Jan 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I and Pilot Study of Subcutaneous Homoharringtonine in Chronic Myelogenous Leukemia
- ClinicalTrials.gov ID
- NCT00006364
- Acronym / sponsor ID
- NCI-2012-02360
- Conditions
- Childhood Chronic Myelogenous Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Phase Chronic Myelogenous Leukemia, Relapsing Chronic Myelogenous Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Nov 1999
- Primary completion
- Sep 2005 (actual)
- First posted
- 27 Jan 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Phase II trial to study the effectiveness of homoharringtonine in treating patients who have chronic phase chronic myelogenous leukemia. Drugs used in chemotherapy, such as homoharringtonine, work in different ways to stop cancer cells from dividing so they stop growing or die
Detailed description
OBJECTIVES:
- Determine the maximum tolerated dose of homoharringtonine in patients with transformed phases of chronic myelogenous leukemia (CML). (Phase I completed as of 2/11/2004.) II. Determine the toxicity profile of this drug in these patients. III. Determine the response duration in patients with chronic phase CML treated with this drug.
IV. Compare the pharmacokinetics of this drug administered as a continuous infusion vs subcutaneously in these patients.
OUTLINE: This is a pilot, dose-escalation study. (Phase I completed as of 2/11/2004.)
Remission induction therapy: Patients receive remission induction therapy comprising homoharringtonine IV continuously over 24 hours on day 1 and then subcutaneously (SC) twice daily on days 2-14 for course 1. Subsequent courses of remission induction therapy comprise homoharringtonine SC twice daily on days 1-14. Treatment continues monthly for at least 2 courses.
Maintenance therapy: Patients with complete hematologic remission receive maintenance therapy comprising homoharringtonine SC twice daily on days 1-7 monthly for 3 years in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of homoharringtonine until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose at which 2 of 6 patients experience dose-limiting toxicity. An additional cohort of 25-30 patients with chronic phase chronic myelogenous leukemia receives remission induction and maintenance therapy as above at the MTD. (Phase I completed as of 2/11/2004.)
Patients are followed every 3 months.
PROJECTED ACCRUAL: A maximum of 50 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (omacetaxine mepesuccinate) | Experimental | Drug: omacetaxine mepesuccinate, Other: pharmacological study, Other: laboratory biomarker analysisRemission induction therapy: Patients receive remission induction therapy comprising homoharringtonine IV continuously over 24 hours on day 1 and then subcutaneously (SC) twice daily on days 2-14 for course 1. Subsequent courses of remission induction therapy comprise homoharringtonine SC twice daily on days 1-14. Treatment continues monthly for at least 2 courses. Maintenance therapy: Patients with complete hematologic remission receive maintenance therapy comprising homoharringtonine SC twice daily on days 1-7 monthly for 3 years in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (21 listed)
- Diagnosis of chronic phase chronic myelogenous leukemia (CML), as defined by the following:
- Philadelphia chromosome- OR BCR/ABL-positive disease by cytogenetics, fluorescence in situ hybridization, or polymerase chain reaction
- Failed prior therapy with imatinib mesylate, as defined by any of the following:
- Failed no more than 2 prior treatment regimens (in addition to imatinib mesylate)
- Ineligible for known regimens or protocols of higher efficacy or priority
Key exclusion criteria (0 listed)
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: CGX-635, homoharringtonineGiven IV or SC | Drug | Treatment (omacetaxine mepesuccinate) |
| pharmacological studyAlso: pharmacological studiesCorrelative studies | Other | Treatment (omacetaxine mepesuccinate) |
| laboratory biomarker analysisCorrelative studies | Other | Treatment (omacetaxine mepesuccinate) |
Primary outcome
Maximum-tolerated dose (MTD) of homoharringtonine as assessed by the National Cancer Institute (NCI) Common Terminology Criteria (CTC)
Time frame: 14 days
Complete hematologic remission (CHR) defined as at least 4 weeks of bone marrow (less than 5% blasts) and peripheral blood with WBC < 10 x 10^9/L and no peripheral blasts, promyelocytes, or myelocytes
Time frame: Up to 6 years
Using a Bayesian approach.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2013.
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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 principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing omacetaxine mepesuccinate, pharmacological study and laboratory biomarker analysis in people with Childhood Chronic Myelogenous Leukemia and Chronic Myelogenous Leukemia, BCR-ABL1 Positive, run by National Cancer Institute (NCI) at 1 site in United States.
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 21 inclusion and 0 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 23 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Maximum-tolerated dose (MTD) of homoharringtonine as assessed by the National Cancer Institute (NCI) Common Terminology Criteria (CTC) (time frame: 14 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Jan 2013.