NCT00030550

Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: —
• Sponsor: Roswell Park Cancer Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Conditions

• Leukemia
• Myelodysplastic Syndromes
• Myelodysplastic/Myeloproliferative Neoplasms

Interventions

Type	Name	Description
DRUG	thalidomide

Timeline

• Start date: 2001-09-01
• Primary completion: 2003-06-01
• First posted: 2003-01-27
• Last updated: 2013-01-31

Locations

1 site across 1 country: United States