Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis
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Purpose
RATIONALE: Drugs used in chemotherapy, such as decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
PURPOSE: This phase II trial is studying the side effects and how well low-dose decitabine works in treating patients with symptomatic myelofibrosis.
| Condition | Intervention | Phase |
|---|---|---|
|
Chronic Myeloproliferative Disorders Secondary Myelofibrosis |
Drug: Dacogen |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Phase II Trial of Low Dose Decitabine (Dacogen) in Patients With Primary Myelofibrosis and Post ET/PV Myelofibrosis |
- Number of Participants Who Achieve a Confirmed Response (Complete Remission (CR), Partial Remission (PR), or Clinical Improvement (CI)), According to International Working Group (IWG) Consensus Criteria. [ Time Frame: Every 4 weeks during treatment (up to 16 weeks) ] [ Designated as safety issue: No ]
Confirmed response: objective status of CR, PR, or CI on 2 consecutive evaluations >=4 weeks apart.
CR:Complete resolution of disease-related symptoms and signs; peripheral blood count remission; normal leukocyte differential; bone marrow histologic remission.
PR: All criteria for CR except the bone marrow histologic remission. CI: one of the following in the absence of both disease progression and CR/PR: minimum (MI) 20-g/L increase (INC) in hemoglobin level; MI 50% reduction in palpable splenomegaly (>=10cm); MI 100% INC in platelet count(>=50000x10^9/L) or ANC (>=0.5x10^9/L)
- Overall Survival(OS) [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]OS was defined as the time from registration to death of any cause.
- Time to Disease Progression [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]
Time to disease progression is defined as the time from registration to progression of disease or death due to any cause.
Progression was defined as any one or more of the following:
1)progressive splenomegaly; 2) leukemic transformation confirmed by a bone marrow blast count of >= 20%; 3) an increase in peripheral blood blast percentage of >=20% that lasts for >= 8 weeks.
- Number of Participants With Constitutional Symptoms [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: Yes ]Constitutional symptoms including the presence of one or more of the following felt to be attributed to the disease: severe night sweats, fevers, weight loss and bone pain. Symptoms were assessed every cycle during treatment.
- Number of Participants With Severe Adverse Events [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: Yes ]Severe adverse events were defined as grade 3 or higher, regardless of attribution to study drugs. Adverse events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 3. Adverse events were assessed every cycle during treatment.
| Enrollment: | 4 |
| Study Start Date: | March 2008 |
| Study Completion Date: | April 2012 |
| Primary Completion Date: | August 2009 (Final data collection date for primary outcome measure) |
OBJECTIVES:
- Determine the efficacy and safety of low-dose decitabine in patients with symptomatic primary myelofibrosis (PMF) or post essential thrombocythemic (ET) or polycythemic vera (PV) myelofibrosis.
- Analyze the ability of this drug to decrease pathologic angiogenesis and other stromal reactive features intrinsic to PMF or post ET/PV myelofibrosis.
OUTLINE: Patients receive low-dose decitabine IV over 1 hour on days 1-5. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients achieving partial remission, complete remission, or clinical improvement may receive up to 12 courses of decitabine in the absence of disease progression or unacceptable toxicity.
After completion of study therapy, patients are followed periodically for up to 3 years.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Histological confirmation of primary myelofibrosis or post essential thrombocythemic or polycythemic vera myelofibrosis
- Reticulin fibrosis ≥ grade 1
Evaluable and symptomatic disease worthy of treatment, characterized by ≥ 1 of the following:
- Anemia, defined as hemoglobin < 11 g/dL or erythrocyte transfusion dependence
- Palpable and symptomatic splenomegaly (palpable and symptomatic hepatomegaly is acceptable if previously splenectomized)
Severe, disease-related constitutional symptoms, including ≥ 1 of the following:
- Severe night sweats
- Fevers
- Weight loss
- Bone pain
- Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard cytogenetics OR prior demonstration of a lack of this translocation
PATIENT CHARACTERISTICS:
- Eastern Co-operative Oncology Group (ECOG) performance status 0-3
- Absolute neutrophil count (ANC) ≥ 1,000/mm³
- Platelet count ≥ 50,000/mm³
- Creatinine ≤ 2.0 mg/dL
- Direct or total bilirubin ≤ 2.0 mg/dL
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 times upper limit of normal (ULN) (≤ 5 times ULN if elevation is attributed to hepatic extramedullary hematopoiesis)
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- Not incarcerated in a municipality, county, state, or federal prison
- No serious medical condition or psychiatric illness that would preclude signing the informed consent
- No condition that, in the opinion of the treating physician, places the patient at unacceptable risk for study participation or confounds the ability to interpret study data
- Able to adhere to the study visit schedule and other study requirements
PRIOR CONCURRENT THERAPY:
- No other concurrent chemotherapy (e.g., hydroxyurea, thalidomide, interferon alpha, anagrelide, or other myelosuppressive agent) or experimental therapy
Contacts and Locations More Information
Additional Information:
No publications provided
| Responsible Party: | Ruben A. Mesa, M.D., Mayo Clinic Cancer Center |
| ClinicalTrials.gov Identifier: | NCT00630994 History of Changes |
| Other Study ID Numbers: | CDR0000588839, P30CA015083, MC0788, NCI-2009-01330, 07-005296 |
| Study First Received: | March 6, 2008 |
| Results First Received: | January 9, 2012 |
| Last Updated: | November 9, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Mayo Clinic:
|
primary myelofibrosis secondary myelofibrosis essential thrombocythemia polycythemia vera |
Additional relevant MeSH terms:
|
Primary Myelofibrosis Myeloproliferative Disorders Bone Marrow Diseases Hematologic Diseases Decitabine Antimetabolites, Antineoplastic |
Antimetabolites Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Antineoplastic Agents Therapeutic Uses Enzyme Inhibitors |
ClinicalTrials.gov processed this record on June 18, 2013