Clinical Trial: Thalidomide in Treating Patients With Myelodysplastic Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study

Brief Summary: Phase II trial to study the effectiveness of thalidomide in treating patients who have myelodysplastic syndrome. Thalidomide may improve the immune system's ability to fight myelodysplastic syndrome

Detailed Summary:

OBJECTIVES:

I. Determine whether thalidomide improves cytopenias in patients with myelodysplastic syndromes.

II. Determine the toxicity of this regimen in these patients. III. Determine whether this regimen down regulates the peripheral blood levels of tumor necrosis factor alpha, interferon gamma, and interleukin-12 and whether these changes correlate with clinical response in these patients.

IV. Determine whether this regimen alters the peripheral blood T-cell subset distribution and whether these changes correlate with clinical response in these patients.

V. Determine the effect of this regimen on bone marrow microvessel density and whether these effects correlate with clinical response in these patients.

OUTLINE: This is a multicenter study. Patients are stratified according to prognosis (favorable vs unfavorable). (Favorable stratum closed to accrual 12/28/01)

Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.

Patients are followed every 6 months for 1 year and then annually for 4 years.

PROJECTED ACCRUAL: A total of 20-58 patients (10-29 per stratum) will be accrued for this study within 20 months. (Favorable stratum closed to accrual 12/28/01)


Sponsor: National Cancer Institute (NCI)

Current Primary Outcome:

  • Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al [ Time Frame: Up to 3 months ]
    Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
  • Incidence and severity of toxicities, graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0 [ Time Frame: Up to 5 years ]


Original Primary Outcome:

Current Secondary Outcome:

  • Survival time [ Time Frame: Time from registration to death due to any cause, assessed up to 5 years ]
    Estimated using the method of Kaplan-Meier.
  • Time to disease progression [ Time Frame: Time from registration to documentation of disease progression, assessed up to 5 years ]
    Estimated using the method of Kaplan-Meier.
  • Duration of response measured using criteria reported by Cheson et al [ Time Frame: Date at which the patient's objective status is first noted to be either a CHR or PR to the date progression is documented, assessed up to 5 years ]
  • Time to treatment failure [ Time Frame: Time from the date of registration to the date at which the patient is removed from treatment due to progression, toxicity, or refusal, assessed up to 5 years ]


Original Secondary Outcome:

Information By: National Cancer Institute (NCI)

Dates:
Date Received: May 6, 2001
Date Started: April 2001
Date Completion:
Last Updated: January 23, 2013
Last Verified: January 2013