Thalidomide in Treating Anemia in Patients With Myelodysplastic Syndrome

Phase 2

Completed

• Conditions: Myelodysplastic Syndromes; Leukemia; Myelodysplastic/Myeloproliferative Neoplasms

• Registration Number: NCT00030550
• Lead Sponsor: 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.

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.

Study Timeline

• Study Start: 2001-09
• Study First Submitted: 2002-02-14
• Study First Submitted QC: 2003-01-26
• Study First Posted: 2003-01-27
• Primary Completion: 2003-06
• Status Verified: 2013-01
• Last Update Submitted: 2013-01-30
• Last Update Posted: 2013-01-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Trial Locations

Locations (1)

PPD Development; 🇺🇸 Wilmington, North Carolina, United States