Azacitidine in Treating Patients With Myelofibrosis
RATIONALE: Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing.
PURPOSE: This phase II trial is studying how well azacitidine works in treating patients with myelofibrosis.
Chronic Myeloproliferative Disorders
|Study Design:||Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study of Azacitidine in Myelofibrosis|
- 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 [ Time Frame: 4 months ]
- 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.
- Overall Survival (OS) [ Time Frame: From date of registration until death or 3 years after registration if patient is still alive ]OS was defined as the time from registration to death due to any cause or time from registration to 3 years after registration if patient is still alive.
- Time to Progression [ Time Frame: up to 3 years ]Time to progression was defined as the time from registration to progression of disease. Those who die without documentation of disease progression will be considered to have had disease progression at the time of their death unless documented evidence clearly indicates no progression has occured.
- Number of Participants With Treatment Related Adverse Events [ Time Frame: Every 4 weeks during treatment ]
Adverse events (AE) that are classified as either possibly, probably, or definitely related to study treatment according to the National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI CTCAE version 3.0). The maximum grade for each type of AE will be recorded for each patient. Grade refers to the severity of the AE.
Grade 1: Mild AE, Grade 2: Moderate AE, Grade 3: Severe AE, Grade 4: Life-threatening or disabling AE, Grade 5: Death related AE
|Study Start Date:||August 2006|
|Study Completion Date:||April 2009|
|Primary Completion Date:||March 2008 (Final data collection date for primary outcome measure)|
- Determine the efficacy of azacitidine in patients with myelofibrosis (MF) with myeloid metaplasia.
- Evaluate the safety of azacitidine in these patients. Secondary
- Evaluate pertinent biologic characteristics of MF before and during therapy with azacitidine.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00381693
|United States, Arizona|
|Mayo Clinic in Arizona|
|Scottsdale, Arizona, United States|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Study Chair:||Ruben A. Mesa, MD||Mayo Clinic|