Phase II Trial of Low Dose Decitabine (Dacogen) in Patients With Primary Myelofibrosis and Post ET/PV Myelofibrosis
- Determine the efficacy and safety of low-dose decitabine in patients with symptomatic
primary myelofibrosis (PMF) or post essential thrombocythemic (ET) or polycythemic vera
- 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
After completion of study therapy, patients are followed periodically for up to 3 years.
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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.
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)
Every 4 weeks during treatment (up to 16 weeks)
Ruben A. Mesa, MD
United States: Food and Drug Administration
|Mayo Clinic||Rochester, Minnesota 55905|