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Efficacy and Safety Study of CC-5013 Monotherapy in Subjects With Myelodysplastic Syndromes

This study has been completed.
Information provided by (Responsible Party):
Celgene ( Celgene Corporation ) Identifier:
First received: July 16, 2003
Last updated: April 3, 2013
Last verified: April 2013
This study is a multi-center, single-arm, open-label study of oral CC-5013 monotherapy administered at a dose of 10 mg daily on Days 1-21 every 28 days (28-day cycles) to red blood cell (RBC) transfusion-dependent subjects with low- or intermediate-1-risk MDS who do not have a del (5q31-33) cytogenetic abnormality. Screening procedures will take place within 28 days of first day of study drug treatment. Subjects will receive study drug (CC-5013) in 28-day cycles for up to 6 cycles, or until bone marrow disease progression or progression/relapse following erythroid hematologic improvement (Appendix I) is documented. Study visits will occur every cycle (every 28 days) and laboratory monitoring to assess hematological parameters will occur every 14 days. Safety and efficacy assessments to be performed during the study are outlined in the Schedule of Study Assessments.

Condition Intervention Phase
Myelodysplastic Syndromes
Drug: CC-5013
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Single-Arm, Open-Label Study of the Efficacy and Safety of CC-5013 Monotherapy in Subjects With Myelodysplastic Syndromes

Resource links provided by NLM:

Further study details as provided by Celgene ( Celgene Corporation ):

Primary Outcome Measures:
  • RBC Transfusion Independence

Secondary Outcome Measures:
  • ≥ 50% decrease in RBC transfusion requirement
  • Platelet Response
    Platelet Response

  • Neutrophil Response
    Neutrophil Response

  • Bone marrow Response
    Bone marrow Response

  • Duration of Response
    Duration of Response

  • Hemoglobin concentration
    Change of hemoglobin concentration from baseline

  • Number of Participants with Adverse Event
    Number of Participants with Adverse Event

Enrollment: 215
Study Start Date: June 2003
Study Completion Date: February 2007
Primary Completion Date: January 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CC-5013
CC-5013 10 mg (two 5 mg capsules) daily on days 1-28 every 28 days (28 day cycles)
Drug: CC-5013
CC-5013 10 mg (two 5 mg capsules) daily on days 1-28 every 28 days (28 day cycles)
Other Name: lenalidomide


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Must understand and voluntarily sign an informed consent form.
  • Age ≥ 18 years at the time of signing the informed consent form.
  • Must be able to adhere to the study visit schedule and other protocol requirements.
  • Diagnosis of low - or intermediate-1-risk IPSS (Appendix III) MDS without an abnormality of chromosome 5 involving a deletion between bands q31 and q33.
  • Red blood cell (RBC) transfusion-dependent anemia defined as having received ≥ to 2 units of RBCs within 8 weeks of the first day of study drug treatment.
  • Eastern Cooperative Oncology Group (ECOG) (Appendix IV) performance status score of 0, 1, or 2.
  • Women of childbearing potential (WCBP) must have a negative serum or urine pregnancy test within 7 days of starting study drug.
  • Sexually active WCBP must agree to use adequate contraceptive methods (oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) while on study drug.
  • WCBP must agree to have pregnancy tests every 4 weeks while on study drug.

Exclusion Criteria:

  • Pregnant or lactating females.
  • Prior therapy with lenalidomide.
  • An abnormality of chromosome 5 involving a deletion between bands q31 and q33.
  • Lab Abnormality: Absolute neutrophil count (ANC) <500 cells/mm3 (0.5 x 109/L)
  • Lab Abnormality: Platelet count <50,000/mm3 (50 x 109/L)
  • Lab Abnormality: Serum creatinine >2.5 mg/dL (221 mmol/L)
  • Lab Abnormality: Serum glutamic oxaloacetic transaminase/Aspartate transaminase (SGOT/AST) or Serum glutamic pyruvic transaminase/Alanine transaminase (SGPT/ALT) >3.0 x upper limit of normal (ULN)
  • Lab Abnormality: Serum total bilirubin >2.0 mg/dL (34 mmol/L)
  • Prior ≥ grade 3 National Cancer Institute (NCI) Common Toxicity Criteria (CTC) (Appendix VI) allergic reaction/hypersensitivity to thalidomide.
  • Prior ≥ grade 3 NCI CTC (Appendix VI) rash or any desquamation (blistering) while taking thalidomide.
  • Clinically significant anemia due to factors such as iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis or gastrointestinal bleeding
  • If a marrow aspirate is not evaluable for storage iron, transferrin saturation must be > 20 % and serum ferritin not less than 50 ng/mL.
  • Use of hematopoietic growth factors within 7 days of the first day of study drug treatment.
  • Chronic use (>2 weeks) of greater than physiologic doses of a corticosteroid agent (dose equivalent to >10 mg/day of prednisone) within 28 days of the first day of study drug treatment.
  • Use of experimental or standard drugs (i.e. chemotherapeutic, immunosuppressive, and cytoprotective agents) for the treatment of MDS within 28 days of the first day of study drug treatment.
  • Prior history of malignancy other than MDS (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been free of disease for greater than or equal to 3 years.
  • Use of any other experimental therapy within 28 days of the first day of study drug treatment.
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Please refer to this study by its identifier: NCT00064974

  Show 119 Study Locations
Sponsors and Collaborators
Celgene Corporation
Study Director: Robert Knight, MD Celgene Corporation
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Celgene Corporation Identifier: NCT00064974     History of Changes
Obsolete Identifiers: NCT00077506
Other Study ID Numbers: CC-5013-MDS-002
Study First Received: July 16, 2003
Last Updated: April 3, 2013

Keywords provided by Celgene ( Celgene Corporation ):

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Pathologic Processes
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 processed this record on May 25, 2017