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Imatinib Mesylate in Treating Patients With Myelofibrosis

A Phase II Pilot Study to Determine the Safety and Preliminary Efficacy of Imatinib Mesylate (Gleevec) in Patients With Myelofibrosis With Myeloid Metaplasia

Status
Terminated
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT00245128
Enrollment
10
Registered
2005-10-27
Start date
2005-08-31
Completion date
2011-10-31
Last updated
2012-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic Myeloproliferative Disorders

Keywords

chronic idiopathic myelofibrosis, polycythemia vera, essential thrombocythemia

Brief summary

RATIONALE: Imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. PURPOSE: This phase II trial is studying the side effects of imatinib mesylate and how well it works in treating patients with myelofibrosis.

Detailed description

OBJECTIVES: Primary * Determine the safety, efficacy, and tolerability of imatinib mesylate in patients with myelofibrosis with myeloid metaplasia. * Determine the 3-, 6-, and 12-month major and minor erythroid response rates in patients treated with this drug. Secondary * Determine reduction in marrow fibrosis in patients treated with this drug. * Determine decrease in spleen size in patients treated with this drug. OUTLINE: This is a multicenter, open-label, nonrandomized, pilot study. Patients receive oral imatinib mesylate once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients who do not experience a minor erythroid response or a 50% reduction in spleen size after 6 months of treatment are removed from the study. Patients experiencing clinical benefit (e.g., ongoing erythroid response) after 1 year of treatment may continue treatment with imatinib mesylate as above at the discretion of the principal investigator. PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.

Interventions

DRUGimatinib mesylate

Once daily oral administration of Imatinib Mesylate at a dose of 600mg for 12 months.

Sponsors

National Cancer Institute (NCI)
CollaboratorNIH
OHSU Knight Cancer Institute
Lead SponsorOTHER

Study design

Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No

Inclusion criteria

DISEASE CHARACTERISTICS: * Diagnosis of myelofibrosis with myeloid metaplasia (MMM), defined by all of the following: * Leukoerythroblastic blood picture * Fibrosis involving \> 1/3 sectional area of bone marrow biopsy * Splenomegaly (unless patient has undergone prior splenectomy) * Philadelphia chromosome negative * No myelodysplastic syndrome * No systemic disorders associated with marrow fibrosis * Red blood cell transfusion dependent, defined by 1 of the following: * Patient has required ≥ 2 units of red blood cells every 4 weeks within the past 8 weeks * Hemoglobin ≤ 8 g/dL on ≥ 3 occasions (≥ 2 weeks apart ) over the past 8 weeks * No evidence of disease transformation to acute myelogenous leukemia, defined as \> 20% blasts in bone marrow and/or peripheral blood PATIENT CHARACTERISTICS: Performance status * ECOG 0-3 Life expectancy * Not specified Hematopoietic * Absolute neutrophil count \> 1,000/mm\^3 * Platelet count \> 50,000/mm\^3 Hepatic * Bilirubin ≤ 1.5 times upper limit of normal (ULN) * AST or ALT ≤ 2 times ULN (unless due to extramedullary hematopoiesis in the liver) Renal * Creatinine ≤ 1.5 times ULN Cardiovascular * No New York Heart Association grade III-IV heart disease Other * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective barrier method contraception during and for 3 months after completion of study treatment * No serious, uncontrolled medical condition * No patients who are considered potentially unreliable or with a history of noncompliance to medical regimens PRIOR CONCURRENT THERAPY: Biologic therapy * More than 2 weeks since prior interferon alfa Chemotherapy * No concurrent chemotherapy except hydroxyurea to control elevated blood counts Endocrine therapy * More than 4 weeks since prior corticosteroids, danazol, or other androgens for MMM Other * More than 4 weeks since other prior treatment for MMM * No other concurrent experimental drug therapy for MMM

Design outcomes

Primary

MeasureTime frameDescription
Percentage of Participants With Major and/or Minor Erythroid Responses at 3, 6, and 12 Months of TherapyAt 3,6, and 12 months of therapyA major response = transfusion independent or a\>2.0g/dl rise in hemoglobin without transfusion maintained for at least 8 weeks. Minor response= \> 1 to 2.0g/dl incremental rise in hemoglobin maintained for at lease 8 weeks with a decrease in transfusion requirements of at least 50% compared to the mean transfusion requirement during the 8 week pre-study period.

Secondary

MeasureTime frame
Reduction in Marrow Fibrosis and Decrease in Spleen SizeAfter 6 and 12 months of therapy

Countries

United States

Participant flow

Participants by arm

ArmCount
Imatinib Mesylate (Gleevec)
Once daily oral administration of Imatinib Mesylate at a dose of 600mg for 12 months.
10
Total10

Baseline characteristics

CharacteristicImatinib Mesylate (Gleevec)
Age, Categorical
<=18 years
0 Participants
Age, Categorical
>=65 years
7 Participants
Age, Categorical
Between 18 and 65 years
3 Participants
Age Continuous68.2 years
STANDARD_DEVIATION 11.00303
Region of Enrollment
United States
10 participants
Sex: Female, Male
Female
1 Participants
Sex: Female, Male
Male
9 Participants

Adverse events

Event typeEG000
affected / at risk
deaths
Total, all-cause mortality
— / —
other
Total, other adverse events
2 / 10
serious
Total, serious adverse events
10 / 10

Outcome results

Primary

Percentage of Participants With Major and/or Minor Erythroid Responses at 3, 6, and 12 Months of Therapy

A major response = transfusion independent or a\>2.0g/dl rise in hemoglobin without transfusion maintained for at least 8 weeks. Minor response= \> 1 to 2.0g/dl incremental rise in hemoglobin maintained for at lease 8 weeks with a decrease in transfusion requirements of at least 50% compared to the mean transfusion requirement during the 8 week pre-study period.

Time frame: At 3,6, and 12 months of therapy

Secondary

Reduction in Marrow Fibrosis and Decrease in Spleen Size

Time frame: After 6 and 12 months of therapy

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026