5-Azacytidine (Azacytidine; Vidaza) in Chronic Lymphocytic Leukemia

This study has been terminated.
(Slow accrual.)
Celgene Corporation
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
First received: December 15, 2006
Last updated: June 26, 2015
Last verified: June 2015

The objective of this study is to determine the safety and efficacy of Azacytidine in fludarabine-resistant chronic lymphocytic leukemia (CLL), Richter's transformation, and T-cell prolymphocytic leukemia (T-PLL).

Condition Intervention Phase
Chronic Lymphocytic Leukemia
Drug: 5-Azacytidine
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Study of 5-Azacytidine (Azacytidine; Vidaza) in Chronic Lymphocytic Leukemia

Resource links provided by NLM:

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

Primary Outcome Measures:
  • Tumor Response Rate (Complete, Partial) of Azacytidine [ Time Frame: 3 to 8 weeks treatment cycles, continuation up to 1 year ] [ Designated as safety issue: No ]
    Overall response rate includes percentage of participants with complete response (CR) plus partial response (PR) responses using the National Cancer Institute (NCI) International Workshop on Chronic Lymphocytic Leukemia (IWCLL) criteria for response: Complete response defined as no palpable lymph nodes, liver or spleen and absence of symptoms. Neutrophil count > 15,00/Mic L, and platelet count more than 100,000/MicL. Hemoglobin should be > 11g/dl without transfusions. Lymphocyte count <4000/micL. On bone marrow aspirate lymphocyte % should be <30%, and biopsy showing no lymphocyte infiltrate. A partial response was defined as more than or equal to 50% decrease in lymph nodes and liver and spleen size. Neutrophils > 1500/ micL or >50 % improvement from baseline, platelet count >100,000/micL or >50 % improvement from baseline. Hemoglobin >11g/dl or >50% improvement from baseline. A reduction of >50% in Leukocyte count or <30 % lymphocytes with residual disease on biopsy for nodular PR.

Enrollment: 9
Study Start Date: September 2006
Study Completion Date: November 2014
Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 5-Azacytidine
5-Azacytidine 75mg/m^2 subcutaneously daily for seven days. Treatment cycles will be repeated every 3-8 weeks.
Drug: 5-Azacytidine
Starting dose level: 75mg/m^2 subcutaneously daily for seven days. Treatment cycles will be repeated every 3-8 weeks.
Other Names:
  • Azacytidine
  • Vidaza
  • 5-aza
  • 5-AZC
  • AZA-CR
  • Ladakamycin
  • NSC-102816

Detailed Description:

Azacytidine 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.

Before you can start treatment on this study, you will have "screening tests." These tests will help the doctor decide if you are eligible to take part in this study. You will have blood drawn (about 3 teaspoons) to check your kidney and liver function (routine blood tests). You may have a bone marrow aspiration performed (if you have not had one in recent weeks). To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic, and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative urine pregnancy test.

If you agree to take part in this study, you will receive azacytidine by subcutaneous (just under the skin) injection every day for 7 days. This course of treatment will be repeated every 3-8 weeks, depending on the results of your routine blood tests.

Your doctor may increase or decrease your dose of azacytidine, depending on if you experience any side effects. You will continue to receive treatment on this study unless the disease gets worse or you experience any intolerable side effects. If the disease gets worse or you experience any intolerable side effects, you will be taken off this study.

This is an investigational study. This is an investigational study. Azacytidine has been approved by the FDA for the treatment of myelodysplastic syndrome. Up to 37 patients will take part in this study. All will be enrolled at M. D. Anderson.


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

Inclusion Criteria:

  1. Patients with chronic lymphocytic leukemia (CLL), prolymphocytic leukemia, Richter's transformation or T-PLL who have previously been treated with fludarabine or another regime are eligible.
  2. Patients with histologically or cytologically confirmed Richter's transformation.
  3. Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) less than x 2 normal levels.
  4. Women of childbearing potential who have a negative pregnancy test prior to azacytidine treatment.
  5. Women of childbearing potential who agreed not to become pregnant and men agreed not to father a child while on azacytidine treatment.
  6. Performance 0-2 (ECOG). Adequate liver function (bilirubin of less than2mg/dl) and renal function (creatinine less than 2mg/dl). Adequate cardiac functions (NYHA cardiac III-IV excluded).
  7. Signed informed consent.

Exclusion Criteria:

  1. Breast feeding or pregnant females. Patients of (male and female) childbearing potential should practice effective methods of contraception; otherwise, they will be excluded. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
  2. Known or suspected hypersensitivity to azacytidine or Mannitol.
  3. Active and uncontrolled infections.
  4. Patients with advanced malignant hepatic tumors.
  5. Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or psychiatric illness/social situations that would limit compliance with study requirements.
  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: NCT00413478

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Celgene Corporation
Principal Investigator: Zeev Estrov, MD M.D. Anderson Cancer Center
  More Information

Additional Information:
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00413478     History of Changes
Other Study ID Numbers: 2006-0428, NCI-2010-00552
Study First Received: December 15, 2006
Results First Received: June 3, 2015
Last Updated: June 26, 2015
Health Authority: United States: Institutional Review Board

Keywords provided by M.D. Anderson Cancer Center:
Chronic Lymphocytic Leukemia
Richter's transformation

Additional relevant MeSH terms:
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Lymphoid
Immune System Diseases
Immunoproliferative Disorders
Leukemia, B-Cell
Lymphatic Diseases
Lymphoproliferative Disorders
Neoplasms by Histologic Type
Antimetabolites, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on October 07, 2015