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Phase II Study of Azacitidine in Myelofibrosis

Phase 2
18 Years
Not Enrolling
Chronic Myeloproliferative Disorders, Secondary Myelofibrosis

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Trial Information

Phase II Study of Azacitidine in Myelofibrosis



- Determine the efficacy of azacitidine in patients with myelofibrosis (MF) with myeloid

- Evaluate the safety of azacitidine in these patients. Secondary

- Evaluate pertinent biologic characteristics of MF before and during therapy with

- Assess the effects of study treatment on constitutional symptoms in these patients.

- Estimate time to event distributions for overall survival and progression. OUTLINE:
Patients receive azacitidine subcutaneously once daily on days 1-5. Treatment repeats
every 4 weeks for up to 12 courses in the absence of disease progression or
unacceptable toxicity.

After completion of study treatment, patients are followed periodically for up to 3 years.

PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study.

Inclusion Criteria


- Histologically confirmed myelofibrosis with myeloid metaplasia (MMM), including any
of the following subtypes:

- Agnogenic myeloid metaplasia

- Post-polycythemic myeloid metaplasia

- Post-thrombocythemic myeloid metaplasia

- Evaluable and symptomatic disease, defined as 1 of the following:

- Anemia (hemoglobin < 10 g/dL or erythrocyte transfusion-dependent, requiring 1
transfusion ≤ 8 weeks)

- Treatment required* for symptomatic palpable splenomegaly (palpable hepatomegaly
is acceptable if previously splenectomized) NOTE: *Subjective but painful enough
to mandate intervention

- Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard
cytogenetics (by peripheral blood or marrow)

- Previous demonstration of a lack of this translocation (at any point) is sufficient

- No advanced malignant hepatic tumors


- ECOG performance status 0-2

- Absolute neutrophil count ≥ 1,000/mm³

- Platelet count ≥ 50,000/mm³

- Creatinine ≤ 2.0 mg/dL

- Total bilirubin ≤ 2.0 mg/dL OR direct bilirubin ≤ 2.0 mg/dL if total bilirubin
elevated (unless attributed to underlying disease)

- AST and ALT ≤ 2 times upper limit of normal (unless clinically attributed to hepatic
extramedullary hematopoiesis)

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- No baseline peripheral or autonomic neuropathy ≥ grade 2

- No condition, including the presence of laboratory abnormalities, that would preclude
study compliance

- No hypersensitivity to mannitol or azacitidine

- Not incarcerated in a municipality (i.e., county, state, or federal prison)


- At least 14 days since prior chemotherapy, including interferon alfa, anagrelide, or
other myelosuppressive agents

- At least 14 days since prior systemic corticosteroids

- At least 14 days since prior investigational agents

Type of Study:


Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Patients With Confirmed Response (Complete Remission or Partial Remission on 2 Consecutive Evaluation at Least 4 Weeks Apart) During the First 4 Months of Treatment

Outcome Description:

Response Definitions: Completion Remission (CR):complete resolution of disease-related symptoms, ultrasound-documented resolution of hepastosplenomegaly, normalization of the peripheral blood count, white cell differential, and smear, normalization of bone marrow histology including disappearance of fibrosis and osteosclerosis. Residual cytogenetic abnormalities are allowed. Partial Remission (PR): a major response in any baseline applicable criteria (except constitutional symptoms) without progression in any other category.

Outcome Time Frame:

4 months

Safety Issue:


Principal Investigator

Ruben A. Mesa, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Mayo Clinic


United States: Food and Drug Administration

Study ID:




Start Date:

August 2006

Completion Date:

April 2009

Related Keywords:

  • Chronic Myeloproliferative Disorders
  • Secondary Myelofibrosis
  • primary myelofibrosis
  • polycythemia vera
  • essential thrombocythemia
  • secondary myelofibrosis
  • Primary Myelofibrosis
  • Myeloproliferative Disorders



Mayo Clinic Rochester, Minnesota  55905
Mayo Clinic in Arizona Scottsdale, Arizona  85259-5404