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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

Phase 2
18 Years
Not Enrolling
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms

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Trial Information

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


- 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

Inclusion Criteria


- 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)



- 18 and over

Performance status:

- ECOG 0-2 OR

- Zubrod 0-2

Life expectancy:

- At least 6 months


- 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


- No uncontrolled hypertension

- No clinically significant, symptomatic, unstable cardiovascular disease unrelated to


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


- 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

- 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


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


- 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


- Not specified


- Not specified


- More than 30 days since prior treatment for MDS except RBC transfusion or epoetin

- 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

Type of Study:


Study Design:

Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Treatment

Principal Investigator

James L. Slack, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Roswell Park Cancer Institute


United States: Federal Government

Study ID:

DS 01-16



Start Date:

September 2001

Completion Date:

Related Keywords:

  • Leukemia
  • Myelodysplastic Syndromes
  • Myelodysplastic/Myeloproliferative Neoplasms
  • 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
  • Anemia
  • Neoplasms
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Myeloproliferative Disorders
  • Myelodysplastic-Myeloproliferative Diseases



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