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Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00030550
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : January 31, 2013
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by:
Roswell Park Cancer Institute

Brief Summary:

RATIONALE: Thalidomide may be an effective treatment for anemia caused by myelodysplastic syndrome.

PURPOSE: Randomized phase II trial to study the effectiveness of thalidomide in treating anemia in patients who have myelodysplastic syndrome.


Condition or disease Intervention/treatment Phase
Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms Drug: thalidomide Phase 2

Detailed Description:

OBJECTIVES:

  • Determine the efficacy of thalidomide for the treatment of anemia in patients with myelodysplastic syndromes.
  • Determine whether this drug reduces the frequency of leukemia transformation and decreases bone marrow blast percentage in these patients.
  • Determine the effect of this drug on neutrophil and platelet production and the number of episodes of febrile neutropenia in these patients.
  • Determine the safety of this drug in these patients.

OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified according to International Prognostic Scoring System score (low and intermediate-1 vs intermediate-2 and high) and transfusion dependence (yes vs no). Patients are randomized to one of two treatment arms.

  • Arm I: Patients receive oral thalidomide once daily on weeks 1-24.
  • Arm II: Patients receive oral placebo once daily on weeks 1-24. In both arms, patients who have not progressed to leukemia after 24 weeks of therapy may receive open-label thalidomide for an additional 24 weeks in the absence of disease progression or unacceptable toxicity.

Patients are followed at 4 weeks.

PROJECTED ACCRUAL: A total of 220 patients (110 per treatment arm) will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Allocation: Randomized
Masking: Double
Primary Purpose: Treatment
Official Title: A Randomized, Multi-Center, Double-Blind, Placebo-Controlled Trial Assessing The Safety And Efficacy Of Thalidomide (THALOMID) For The Treatment Of Anemia In Red Blood Cell Transfusion-Dependent Patients With Myelodysplastic Syndromes
Study Start Date : September 2001
Actual Primary Completion Date : June 2003






Information from the National Library of Medicine

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

DISEASE CHARACTERISTICS:

  • Diagnosis of myelodysplastic syndromes (MDS) of at least 12 weeks duration

    • Refractory anemia (RA)
    • RA with ringed sideroblasts
    • RA with excess blasts
    • Chronic myelomonocytic
  • No therapy-related MDS
  • No myelosclerosis or myelofibrosis occupying more than 30% of marrow space (or assessed as grade 3+ or greater)
  • No transformation to acute myeloid leukemia
  • No more than 20% blasts in bone marrow
  • No more than 5% blasts in peripheral blood
  • Patients with an erythropoietin level 100 mU/mL or less must have failed epoetin alfa treatment (i.e., at least 30,000 units of epoetin alfa weekly for at least 6 weeks)
  • Transfusion-dependent (received at least 2 units of packed RBCs or whole blood within the past 8 weeks) OR
  • Transfusion-independent (no packed RBC or whole blood transfusions within the past 8 weeks with 2 hemoglobin levels (at least 7 days apart) less than 11 g/dL)
  • No iron deficiency (e.g., absent bone marrow iron store)

    • If marrow aspirate is not evaluable, transferrin saturation must be at least 20% and ferritin at least 50 ng/mL
  • No uncorrected B12 or folate deficiency
  • No other contributing causes of anemia (e.g., autoimmune or hereditary hemolytic disorders or gastrointestinal blood loss)

PATIENT CHARACTERISTICS:

Age:

  • 18 and over

Performance status:

  • ECOG 0-2 OR
  • Zubrod 0-2

Life expectancy:

  • At least 6 months

Hematopoietic:

  • See Disease Characteristics
  • Absolute neutrophil count at least 500/mm^3

Hepatic:

  • Bilirubin no greater than 2.0 mg/dL
  • AST and ALT less than 2 times upper limit of normal (ULN)
  • Hepatitis B surface antigen negative
  • Hepatitis C negative

Renal:

  • Creatinine no greater than 1.5 times ULN

Cardiovascular:

  • No uncontrolled hypertension
  • No clinically significant, symptomatic, unstable cardiovascular disease unrelated to MDS

Pulmonary:

  • No clinically significant, symptomatic, unstable pulmonary disease unrelated to MDS

Neurologic:

  • No clinically significant, symptomatic, unstable neurologic disease unrelated to MDS
  • No history of epilepsy
  • No sustained neurologic deficit (e.g., stroke)
  • No grade 2 or greater peripheral neuropathy

Other:

  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use at least 1 highly effective and 1 additional effective method of contraception for 4 weeks prior to, during, and for 4 weeks after study participation
  • HIV negative
  • No clinically significant, symptomatic, unstable endocrine, gastrointestinal, or genitourinary disease unrelated to MDS
  • No other malignancy within the past 5 years except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix
  • No life-threatening or active infection requiring parenteral antibiotics
  • No other serious concurrent illness

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • See Disease Characteristics
  • More than 7 days since prior hematopoietic growth factors (e.g., epoetin alfa, filgrastim (G-CSF), sargramostim (GM-CSF), or interleukin-3)
  • No prior thalidomide
  • No prior agents intended to inhibit vascular endothelial growth factor or tumor necrosis factor alfa (e.g., etanercept or infliximab)
  • No concurrent epoetin alfa

Chemotherapy:

  • No concurrent chemotherapy that may be active against MDS

Endocrine therapy:

  • More than 30 days since prior androgens
  • No requirement for ongoing therapy with systemic corticosteroids

Radiotherapy:

  • Not specified

Surgery:

  • Not specified

Other:

  • More than 30 days since prior treatment for MDS except RBC transfusion or epoetin alfa
  • More than 30 days since prior participation in another experimental clinical trial
  • More than 30 days since prior experimental drugs
  • No other concurrent investigational agents or treatments

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00030550


Locations
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United States, North Carolina
PPD Development
Wilmington, North Carolina, United States, 28412
Sponsors and Collaborators
Roswell Park Cancer Institute
National Cancer Institute (NCI)
Investigators
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Study Chair: James L. Slack, MD Roswell Park Cancer Institute
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ClinicalTrials.gov Identifier: NCT00030550    
Other Study ID Numbers: DS 01-16
RPCI-DS-0116
CELGENE-T-MDS-001
NCI-G01-2044
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: January 31, 2013
Last Verified: January 2013
Keywords provided by Roswell Park Cancer Institute:
refractory anemia
refractory anemia with ringed sideroblasts
refractory anemia with excess blasts
de novo myelodysplastic syndromes
chronic myelomonocytic leukemia
previously treated myelodysplastic syndromes
myelodysplastic/myeloproliferative neoplasm, unclassifiable
atypical chronic myeloid leukemia, BCR-ABL1 negative
Additional relevant MeSH terms:
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Leukemia
Preleukemia
Anemia
Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Syndrome
Disease
Pathologic Processes
Neoplasms by Histologic Type
Neoplasms
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Thalidomide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents