5-Azacytidine With Lenalidomide in Patients With High Risk Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML)

This study has been completed.
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
First received: December 23, 2009
Last updated: November 2, 2016
Last verified: November 2016

The goal of Phase 1 of this clinical research study is to find the highest tolerable dose of lenalidomide that can be given in combination with azacitidine to patients with MDS or AML.

The goal of Phase 2 of this study is to learn if the combination dose of azacitidine and lenalidomide found in Phase 1 can help to control MDS and/or AML.

The safety of this drug combination will be studied in both Phases.

Condition Intervention Phase
Drug: 5-Azacytidine
Drug: Lenalidomide
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I/II Study of the Combination of 5-azacitidine With Lenalidomide in Patients With High Risk Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML)

Resource links provided by NLM:

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

Primary Outcome Measures:
  • Maximum Dose Tolerated (MTD) of Lenalidomide in Combination with 5-azacytidine (5-aza) in Participants with Leukemia [ Time Frame: 3-8 week cycles ]
    If 1 participant develops grade III-IV non-hematological toxicity, 3 more patients accrued at that particular dose level. If 2 or more participants develop grade III-IV non-hematologic toxicity, doses of the combination at which this occurs considered too toxic.

Secondary Outcome Measures:
  • Response Rate of Lenalidomide in Combination with 5-azacytidine (5-aza) in Participants with Leukemia [ Time Frame: 6 months ]
    Response defined as complete remission (CR) or complete remission with incomplete platelet recovery (CRp) for AML or any response for myelodysplastic syndrome (MDS) using IWG-06 criteria.

Enrollment: 94
Study Start Date: December 2009
Study Completion Date: November 2015
Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 5-Azacytidine + Lenalidomide
5-Azacytidine 75 mg/m^2 by vein daily x 5 days on days 1 to 5. Lenalidomide starting dose 10 mg orally daily x 5 days on days 6 to 10.
Drug: 5-Azacytidine
75 mg/m^2 IV daily x 5 days on days 1 to 5.
Other Names:
  • 5-AZA
  • Azacitidine
  • Vidaza
  • 5-AZC
  • AZA-CR
  • Ladakamycin
Drug: Lenalidomide
Starting dose 10 mg orally daily x 5 days on days 6 to 10.
Other Names:
  • CC-5013
  • Revlimid

  Show Detailed Description


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patients with higher risk MDS (bone marrow blasts >/= 10% to 30% inclusive) of any age who refuse or are not eligible for frontline chemotherapy.
  2. No prior therapy for higher risk MDS as defined above.
  3. Performance status of </= 2 by the Eastern Cooperative Oncology Group (ECOG) scale.
  4. Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of University of Texas MD Anderson Cancer Center (UTMDACC).
  5. Hydroxyurea for patients with rapidly proliferative disease can be used up to 24 hours prior to therapy but not concomitantly with 5-azacitidine or lenalidomide. Hydroxyurea can be used once the patient has completed the planned 5 azacitidine and lenalidomide treatment.
  6. Adequate liver function: Total bilirubin of </= 1.5 x upper limit of normal (ULN), AST (SGOT) and ALT (SGPT) </= 3 x ULN
  7. Renal function - assessed by calculated creatinine clearance as follows (see Appendix: Cockcroft-Gault estimation of CrCl): 1. Phase I subjects must have calculated creatinine clearance >/= 60ml/min by Cockcroft-Gault formula. 2. Phase II subjects must have calculated creatinine clearance >/= 30ml/min by Cockcroft-Gault formula.
  8. All study participants must be registered into the mandatory Revlimid REMS® program, and be willing and able to comply with the requirements of Revlimid REMS®.
  9. Females of reproductive potential must adhere to the scheduled pregnancy testing as required in the Revlimid REMS® program.
  10. Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (patients intolerant to ASA may use warfarin or low molecular weight heparin).
  11. Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 10 - 14 days prior to and again within 24 hours of prescribing lenalidomide (prescriptions must be filled within 7 days) and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking lenalidomide. FCBP must also agree to ongoing pregnancy testing.
  12. Continued from #9: Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy.

Exclusion Criteria:

  1. Nursing and pregnant females.
  2. Known or suspected hypersensitivity to azacitidine or mannitol.
  3. Patients with advanced malignant hepatic tumors.
  4. Unwilling or unable to remain in compliance with the RevAssist® program
  5. Known hypersensitivity to thalidomide or lenalidomide (if applicable).
  6. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.
  7. Known seropositive for or active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV). Patients who are seropositive because of hepatitis B virus vaccine are eligible.
  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: NCT01038635

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Study Chair: Guillermo Garcia-Manero, MD M.D. Anderson Cancer Center
  More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01038635     History of Changes
Other Study ID Numbers: 2009-0467  NCI-2011-01941 
Study First Received: December 23, 2009
Last Updated: November 2, 2016

Keywords provided by M.D. Anderson Cancer Center:
Myelodysplastic Syndrome
Acute Myelogenous Leukemia

Additional relevant MeSH terms:
Leukemia, Myeloid
Myelodysplastic Syndromes
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Immunologic Factors
Physiological Effects of Drugs
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents
Immunosuppressive Agents
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Enzyme Inhibitors

ClinicalTrials.gov processed this record on January 23, 2017