NCT00030550CompletedPhase 2
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
Conditions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Locations
- 1
Study Overview
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.
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Primary Purpose
- Treatment
- Masking
- Double
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion 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:
- •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
- •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
- •Creatinine no greater than 1.5 times ULN
- •Cardiovascular:
- •No uncontrolled hypertension
- •No clinically significant, symptomatic, unstable cardiovascular disease unrelated to MDS
- •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
- •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
- +19 more not shown
Exclusion Criteria
- Not provided
Investigators
Study Sites (1)
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