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Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome

This study has been completed.

Sponsors and Collaborators: Roswell Park Cancer Institute
National Cancer Institute (NCI)
Information provided by: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00030550
  Purpose

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 Intervention Phase
Leukemia
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Drug: thalidomide
Phase II

MedlinePlus related topics:   Anemia    Cancer    Leukemia, Adult Acute    Leukemia, Adult Chronic   

ChemIDplus related topics:   Thalidomide   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized, Double-Blind, Placebo Control
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

Further study details as provided by National Cancer Institute (NCI):

Study Start Date:   September 2001

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.

  Eligibility
Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00030550

Locations
United States, New York
Roswell Park Cancer Institute    
      Buffalo, New York, United States, 14263-0001
United States, North Carolina
PPD Development    
      Wilmington, North Carolina, United States, 28412

Sponsors and Collaborators
Roswell Park Cancer Institute
National Cancer Institute (NCI)

Investigators
Study Chair:     James L. Slack, MD     Roswell Park Cancer Institute    
  More Information


Clinical trial summary from the National Cancer Institute's PDQ® database  This link exits the ClinicalTrials.gov site
 

Study ID Numbers:   CDR0000069176, RPCI-DS-0116, CELGENE-T-MDS-001, NCI-G01-2044
First Received:   February 14, 2002
Last Updated:   July 23, 2008
ClinicalTrials.gov Identifier:   NCT00030550
Health Authority:   United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
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 disease, unclassifiable
atypical chronic myeloid leukemia

Study placed in the following topic categories:
Myelodysplastic syndromes
Thalidomide
Chronic myelogenous leukemia
Precancerous Conditions
Chronic myelomonocytic leukemia
Refractory anemia
Hematologic Diseases
Leukemia, Myelomonocytic, Chronic
Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative
Myelodysplasia
Myelodysplastic Syndromes
Anemia
Myeloproliferative Disorders
Leukemia, Myeloid
Myelodysplastic myeloproliferative disease
Leukemia
Preleukemia
Anemia, Refractory
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Anemia, Refractory, with Excess of Blasts
Myelodysplastic-Myeloproliferative Diseases
Bone Marrow Diseases

Additional relevant MeSH terms:
Anti-Infective Agents
Neoplasms by Histologic Type
Disease
Immunologic Factors
Antineoplastic Agents
Growth Substances
Physiological Effects of Drugs
Angiogenesis Inhibitors
Immunosuppressive Agents
Pharmacologic Actions
Anti-Bacterial Agents
Neoplasms
Pathologic Processes
Syndrome
Therapeutic Uses
Growth Inhibitors
Angiogenesis Modulating Agents
Leprostatic Agents

ClinicalTrials.gov processed this record on October 07, 2008




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