Ph II Study of Azacitidine in Myelofibrosis

This study has been completed.
Sponsor:
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT00569660
First received: December 6, 2007
Last updated: August 1, 2012
Last verified: August 2012
  Purpose

The goal of this clinical research study is to learn if azacitidine can help to control MF. The safety of azacitidine in patients with Myelofibrosis (MF) will also be studied.


Condition Intervention Phase
Myelofibrosis
Drug: Azacitidine
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Study of Azacitidine in Myelofibrosis

Resource links provided by NLM:


Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Number of Participants With Objective Clinical Response [ Time Frame: Every 2 courses of 4 week therapy = each 8 weeks ] [ Designated as safety issue: No ]
    Objective Clinical Response includes Participants with Complete Response, Partial Response or Hematologic Improvement and No Response. Bone marrow aspiration and biopsy with cytogenetics every 2 to 4 courses.


Enrollment: 34
Study Start Date: June 2005
Study Completion Date: April 2008
Primary Completion Date: April 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Azacitidine
75 mg/m^2 Subcutaneous Daily for 7 days every 4 weeks
Drug: Azacitidine
75 mg/m^2 subcutaneous daily for 7 days (every 4 week cycle)
Other Name: Vidaza

Detailed Description:

Azacitidine is a drug that is designed to block certain genes in cancer cells whose job is to stop the function of the tumor-fighting genes. By blocking the "bad" genes, the tumor-fighting genes may be able to work better.

If you are found to be eligible to take part in this study, you will be able to begin treatment with azacitidine. You will receive azacitidine as an injection under the skin once a day for 7 days in a row. This will be repeated every 4 weeks (4 weeks equals 1 cycle). The first cycle of azacitidine will be given at M. D. Anderson, in an outpatient setting. Later cycles of treatment courses may be given at M. D. Anderson or by a cancer doctor in your community.

You may receive up to 12 cycles of treatment if you are responding well to treatment. You will be taken off study if your disease gets worse or intolerable side effects occur. Once you go off study, you will receive follow-up as is standard of care for your disease.

This is an investigational study. Azacitidine is FDA approved for the treatment of myelodysplastic syndrome. Its use in this study is experimental. A total of up to 34 patients will take part in this study. All will be enrolled at M. D. Anderson.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of MF requiring therapy, including those previously treated and relapsed or refractory, or if newly diagnosed, with intermediate or high risk according to Lille scoring system (adverse prognostic factors are: Hemoglobin (Hb) < 10 g/dl, White Blood Cell (WBC) < 4 or > 30 x 10*9/L; risk group: 0 = low, 1 = intermediate, 2 = high).
  • Performance 0-2 Eastern Cooperative Oncology Group (ECOG).
  • Signed informed consent.
  • Patients must have been off chemotherapy for 2 weeks prior to entering this study and have recovered from the toxic effects (grade 0-1) of that therapy. Patients are allowed to be on anagrelide and hydroxyurea to control high platelet and WBC counts for their safety.
  • Serum bilirubin levels </= 1.5 times the upper limit of the normal range for the laboratory Upper Limit of of Normal(ULN). Higher levels are acceptable if these can be attributed to active hemolysis or ineffective erythropoiesis.
  • Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) levels </= 2 x ULN.
  • Serum creatinine levels </= 1.5 x ULN; unless related to the MF, as judged by treating physicians.
  • Women of childbearing potential must have a negative serum pregnancy test prior to azacitidine treatment and should be advised to avoid becoming pregnant. Men must be advised to not father a child while receiving treatment with azacitidine. Both women of childbearing potential and men must practice effective methods of contraception (those generally accepted as standard of care measures).
  • Age >/= 18.

Exclusion Criteria:

  • Nursing and pregnant females. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
  • Uncontrolled intercurrent illness including, but not limited to, uncontrolled active infection, symptomatic congestive heart failure, unstable angina pectoris, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Known or suspected hypersensitivity to azacitidine or mannitol.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00569660

Locations
United States, Texas
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Celgene Corporation
Investigators
Principal Investigator: Srdan Verstovsek, M.D. M.D. Anderson Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00569660     History of Changes
Other Study ID Numbers: 2005-0033
Study First Received: December 6, 2007
Results First Received: September 21, 2009
Last Updated: August 1, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by M.D. Anderson Cancer Center:
Myelofibrosis
MF
Leukemia
Azacitidine
Vidaza

Additional relevant MeSH terms:
Primary Myelofibrosis
Bone Marrow Diseases
Hematologic Diseases
Myeloproliferative Disorders
Azacitidine
Antimetabolites
Antimetabolites, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on October 29, 2014