Chronic Myelomonocytic LeukemiaPhase 2NCT00039416
Imatinib Mesylate in Treating Patients With Myelofibrosis
A Phase 2 study testing imatinib mesylate and laboratory biomarker analysis in people with Chronic Myelomonocytic Leukemia and Essential Thrombocythemia, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Phase II trial to study the effectiveness of imatinib mesylate in treating patients who have myelofibrosis. Imatinib mesylate may stop the growth of myelofibrosis by blocking certain enzymes necessary for cell growth. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myelomonocytic Leukemia, Essential Thrombocythemia, Polycythemia Vera
- What is being tested?
- imatinib mesylate and laboratory biomarker analysis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2002to Aug 2007
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have histologic confirmation of one of the following diseases-
- Myeloid metaplasia with myelofibrosis (this includes all subtypes- chronic idiopathic myelofibrosis or agnogenic myeloid metaplasia, post thrombocythemic and post polycythemic myelofibrosis) or
- Chronic myelomonocytic leukemia (CMMOL) with t(5;12)(q31-33;p12) or TEL-PDGFRβ rearrangement; patients with CMMOL and the t(5;7)(q31-33;q11.2) or other chromosomal translocations resulting in activation of PDGFR will also be eligible
- Patients must have anemia (hemoglobin < 11 g/dL) or palpable splenomegaly (measured in cm from costal margin- to eligible); patients with palpable splenomegaly must have spleen size documented ultrasonographically as well; they must also meet standard diagnostic criteria for MMM* or CMMOL; patients with MMM must have thrombocytopenia (platelet count < 100 x 10^9/L) to be eligible; they must be Philadelphia chromosome or (BCR/ABL) rearrangement negative
- Patients with CMMOL must also have the t(5;12)(q31-33;p12) or TEL-PDGFRβ rearrangement to be eligible
- The Italian diagnostic criteria for MMM
Show all 34Show fewer
- Necessary criteria
- Diffuse bone marrow fibrosis
- Absence of the Philadelphia chromosome or BCR-ABL rearrangement in peripheral blood cells
- Optional criteria
- Splenomegaly of any grade
- Anisopoikilocytosis with tear drop erythrocytes
- Presence of circulating immature myeloid cells
- Presence of circulating erythroblasts
- Presence of clusters of megakaryoblasts and anomalous megakaryocytes in bone marrow sections
- Myeloid metaplasia
- Diagnosis of MMM is acceptable if the following combinations are present
- The two necessary criteria plus any other two optional criteria when splenomegaly is present OR
- The two necessary criteria plus any other four optional criteria when splenomegaly is absent
- Patients may have had prior chemotherapy or radiation therapy including splenic irradiation; prior therapy with erythropoietin, GCSF or androgenic steroids is also permitted; there is no limit to number of prior regimens received; at least 4 weeks must have elapsed since prior chemo, radiation or other therapy
- ECOG performance status =< 2 (Karnofsky >= 60%)
- Total bilirubin < 1.5 X institutional upper limit of normal
- AST(SGOT)/ALT(SGPT) <2.5 X institutional upper limit of normal unless due to disease
- Serum creatinine < 2 X institutional upper limit of normal
- Patients must not be pregnant or nursing because STI-571 at the recommended therapeutic dose may be harmful to the developing fetus or newborn; for this reason women of child-bearing potential and men must agree to use an effective contraceptive method; women of reproductive potential must have a negative pregnancy test within 7 days prior to registration; since interactions with oral contraceptives cannot be excluded at present, male and female patients of reproductive potential must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug
- Ability to understand and the willingness to sign a written informed consent document
- World Health Organization (WHO) diagnostic criteria for CMMOL:
- Persistent peripheral blood monocytosis > 1 x 10^9/L
- Absence of the Philadelphia chromosome or BCR/ABL fusion gene
- Fewer than 20% blasts in the blood or bone marrow
- Dysplasia in one or more myeloid lineages; if myelodysplasia is absent or minimal, the diagnosis of CMML may still be made if the other criteria (1-3) are met and:
- An acquired clonal cytogenetic abnormality is present in the marrow cells
- The monocytosis has been persistent for at least 3 months
- Other causes for monocytosis have been excluded
Who cannot join
- Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
- Patients may not be receiving any other investigational agents
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to STI-571
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Pregnant women are excluded from this study because STI-571 is an agent with the potential for teratogenic or abortifacient effects; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with STI-571, STI-571 should not be administered patients who are breastfeeding
- HIV-positive patients receiving combination anti-retroviral therapy are excluded from the study because of possible pharmacokinetic interactions with STI-571; appropriate studies will be undertaken in patients receiving combination anti-retroviral therapy when indicated
- Because warfarin is metabolized through the CYP450 system, and since gastrointestinal bleeding may occur with STI-571, no therapeutic anticoagulation with warfarin will be permitted in patients participating in this study; as an alternative, therapeutic anticoagulation may be accomplished using low-molecular weight heparin (e.g. Lovenox) or heparin
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II Study Of Gleevec (Imatinib Mesylate Formerly Known as STI-571) In Patients With Myelofibrosis
Phase II trial to study the effectiveness of imatinib mesylate in treating patients who have myelofibrosis. Imatinib mesylate may stop the growth of myelofibrosis by blocking certain enzymes necessary for cell growth.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the response rate (complete and partial) to STI-571 in patients with myelofibrosis.
II. To determine the safety of STI-571 in patients with myelofibrosis.
SECONDARY OBJECTIVES:
- To determine the effects of STI-571 on bone marrow morphology (including effects on marrow fibrosis, osteosclerosis and cellularity) in patients with myelofibrosis.
II. To assess the effects of STI-571 on surrogate biologic endpoints including PDGFR expression (by immunohistochemistry), PDGFR signaling, and circulating progenitor (CD34 positive) cells.
III. To determine the effects of STI-571 on bone marrow cytogenetics in patients with an abnormal karyotype.
OUTLINE: This is a multicenter study. Patients are stratified according to Dupriez risk score (low vs intermediate vs high).
Patients receive oral imatinib mesylate once or twice daily on days 1-28. Courses repeat every 28 days for 12 months in the absence of disease progression or unacceptable toxicity.
- Conditions
- Chronic Myelomonocytic Leukemia, Essential Thrombocythemia, Polycythemia Vera, Primary Myelofibrosis
- Being tested
- imatinib mesylate, laboratory biomarker analysis
- Medical subject headings
- Leukemia, Myelomonocytic, Chronic, Thrombocythemia, Essential, Polycythemia Vera, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
imatinib mesylateDrug
Also called CGP 57148, Gleevec, Glivec
Given orally
laboratory biomarker analysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (imatinib mesylate) | Experimental | Patients receive oral imatinib mesylate once or twice daily on days 1-28. Courses repeat every 28 days for 12 months in the absence of disease progression or unacceptable toxicity. Receives: Drug: imatinib mesylate; Other: laboratory biomarker analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical responses in terms of improvement in anemia and splenomegaly as previously published for myelofibrosis
Measured: Up to 12 months
Frequency of adverse events graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0
Exact 95% confidence intervals generated using the binomial distribution.
Measured: Up to 12 months
Secondary outcomes (2)
Progression-free survival
Analyzed using the Kaplan-Meier method.
Measured: Up to 12 months
Bone marrow response
Measured: Up to 12 months
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 startApr 2002
- Primary completionAug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myelomonocytic 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
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Olatoyosi Odenike, Principal investigator, University of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00039416
- Sponsor's protocol ID
- NCI-2012-02895
- Other IDs
- NCI-2012-02895, CDR0000069381, 11498A, 5669, N01CM62201, P30CA014599
- Status last verified
- Dec 2012
- First submitted
- 6 Jun 2002
- Last updated
- 25 Feb 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study Of Gleevec (Imatinib Mesylate Formerly Known as STI-571) In Patients With Myelofibrosis
- ClinicalTrials.gov ID
- NCT00039416
- Acronym / sponsor ID
- NCI-2012-02895
- Conditions
- Chronic Myelomonocytic Leukemia, Essential Thrombocythemia, Polycythemia Vera, Primary Myelofibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Apr 2002
- Primary completion
- Aug 2007 (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 imatinib mesylate in treating patients who have myelofibrosis. Imatinib mesylate may stop the growth of myelofibrosis by blocking certain enzymes necessary for cell growth.
Detailed description
PRIMARY OBJECTIVES:
- To determine the response rate (complete and partial) to STI-571 in patients with myelofibrosis.
II. To determine the safety of STI-571 in patients with myelofibrosis.
SECONDARY OBJECTIVES:
- To determine the effects of STI-571 on bone marrow morphology (including effects on marrow fibrosis, osteosclerosis and cellularity) in patients with myelofibrosis.
II. To assess the effects of STI-571 on surrogate biologic endpoints including PDGFR expression (by immunohistochemistry), PDGFR signaling, and circulating progenitor (CD34 positive) cells.
III. To determine the effects of STI-571 on bone marrow cytogenetics in patients with an abnormal karyotype.
OUTLINE: This is a multicenter study. Patients are stratified according to Dupriez risk score (low vs intermediate vs high).
Patients receive oral imatinib mesylate once or twice daily on days 1-28. Courses repeat every 28 days for 12 months in the absence of disease progression or unacceptable toxicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (imatinib mesylate) | Experimental | Drug: imatinib mesylate, Other: laboratory biomarker analysisPatients receive oral imatinib mesylate once or twice daily on days 1-28. Courses repeat every 28 days for 12 months in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (34 listed)
- Patients must have histologic confirmation of one of the following diseases-
- The Italian diagnostic criteria for MMM
- Patients may have had prior chemotherapy or radiation therapy including splenic irradiation; prior therapy with erythropoietin, GCSF or androgenic steroids is also permitted; there is no limit to number of prior regimens received; at least 4 weeks must have elapsed since prior chemo, radiation or other therapy
- ECOG performance status =< 2 (Karnofsky >= 60%)
- Total bilirubin < 1.5 X institutional upper limit of normal
Key exclusion criteria (7 listed)
- Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
- Patients may not be receiving any other investigational agents
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to STI-571
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Pregnant women are excluded from this study because STI-571 is an agent with the potential for teratogenic or abortifacient effects; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with STI-571, STI-571 should not be administered patients who are breastfeeding
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 |
|---|---|---|
| imatinib mesylateAlso: CGP 57148, Gleevec, GlivecGiven orally | Drug | Treatment (imatinib mesylate) |
| laboratory biomarker analysisCorrelative studies | Other | Treatment (imatinib mesylate) |
Primary outcome
Clinical responses in terms of improvement in anemia and splenomegaly as previously published for myelofibrosis
Time frame: Up to 12 months
Frequency of adverse events graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0
Time frame: Up to 12 months
Exact 95% confidence intervals generated using the binomial distribution.
2 secondary outcomes
Progression-free survival
Time frame: Up to 12 months
Bone marrow response
Time frame: Up to 12 months
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 25 Feb 2014.
Related trials for Chronic Myelomonocytic Leukemia
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 2
Ropeginterferon Alfa-2b for the Treatment of Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes and Chronic Myelomonocytic Leukemia
Atypical Chronic Myeloid Leukemia, Chronic Myelomonocytic Leukemia
RecruitingPhase 1
Phase 1 Study of 6MW5311 in Adults With Relapsed or Refractory Blood Cancers
AML, Multiple Myeloma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingPhase 2
Personalized NK Cell Therapy in CBT
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia
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.
Olatoyosi Odenike principal investigator
University of Chicago
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 imatinib mesylate and laboratory biomarker analysis in people with Chronic Myelomonocytic Leukemia and Essential Thrombocythemia, 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 34 inclusion and 7 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 25 Feb 2014. 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?
Clinical responses in terms of improvement in anemia and splenomegaly as previously published for myelofibrosis (time frame: Up to 12 months). 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 25 Feb 2014.