Clinical trial NCT00413478: 5-Azacytidine (Azacytidine; Vidaza) in Chronic Lymphocytic Leukemia
Official title: Phase II Study of 5-Azacytidine (Azacytidine; Vidaza) in Chronic Lymphocytic Leukemia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Chronic Lymphocytic Leukemia
Leukemia
Keywords: 5-Aza
Azacytidine
Vidaza
CLL
Chronic Lymphocytic Leukemia
Richter's transformation
Leukemia
Lead sponsor: M.D. Anderson Cancer Center
Sponsor class: OTHER
Collaborators: name: Celgene Corporation
First posted: 2006-12-19
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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).

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bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic, and a small amount of bone marrow is withdrawn through a large needle.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.

Eligibility criteria: 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.

Primary outcomes:
measure: Tumor Response Rate (Complete, Partial) of Azacytidine
timeFrame: 3 to 8 weeks treatment cycles, continuation up to 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: University of Texas MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Zeev Estrov, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Malik A, Shoukier M, Garcia-Manero G, Wierda W, Cortes J, Bickel S, Keating MJ, Estrov Z. Azacitidine in fludarabine-refractory chronic lymphocytic leukemia: a phase II study. Clin Lymphoma Myeloma Leuk. 2013 Jun;13(3):292-5. doi: 10.1016/j.clml.2012.11.009. Epub 2012 Dec 21.