Thalidomide in Treating Patients With Myelodysplastic Syndrome

NCT ID: NCT00015990

Last Updated: 2013-01-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 29 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2001-04-30

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 Description

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)

Conditions

Chronic Myelomonocytic Leukemia; de Novo Myelodysplastic Syndromes; Previously Treated Myelodysplastic Syndromes; Refractory Anemia; Refractory Anemia With Excess Blasts; Refractory Anemia With Excess Blasts in Transformation; Refractory Anemia With Ringed Sideroblasts; Secondary Myelodysplastic Syndromes

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment (thalidomide)

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

Group Type: EXPERIMENTAL

thalidomide

Intervention Type: DRUG

Given orally

laboratory biomarker analysis

Intervention Type: OTHER

Correlative studies

Interventions

thalidomide

Given orally

Intervention Type: DRUG

laboratory biomarker analysis

Correlative studies

Intervention Type: OTHER

Other Intervention Names

Kevadon; Synovir; THAL; Thalomid

Eligibility Criteria

Inclusion Criteria

• Pre-transfusion hemoglobin =< 10 g/dL
• Pre-transfusion platelet count =< 50,000/μL
• Absolute neutrophil count < 1000/μL
• Total bilirubin ≤ 1.5 x UNL
• Alkaline phosphatase ≤ 3 x UNL
• AST ≤ 3 x UNL
• Creatinine ≤ 1.5 x UNL
• A diagnosis of MDS as demonstrated in the bone marrow; any subtypes are eligible including:

• Refractory anemia (cytopenia)
• Refractory anemia with ringed sideroblasts
• Chronic myelomonocytic leukemia
• Refractory anemia with excess blasts
• Refractory anemia with excess blasts in transformation
• Unclassified MDS
• Patients with refractory anemia with excess blasts in transformation who are not candidates for (or who decline) induction chemotherapy are eligible; those patients who were candidates for (and accepted) induction chemotherapy should have failed at least 1 chemotherapy regimen prior to entry
• Patients who are candidates for marrow transplantation should have this option discussed prior to study entry

Exclusion Criteria

• Any of the following as this regimen may be harmful to a developing fetus or nursing child:

• Pregnant women
• Nursing women
• Women of childbearing potential or their sexual partners who are unwilling to employ 2 adequate methods of contraception (condoms, diaphragm, birth control pills, injections, intrauterine device [IUD], surgical sterilization, subcutaneous implants, or abstinence, etc.)
• Peripheral neuropathy (by history or clinical exam)
• Concomitant therapy ≤ 30 days for myelodysplastic syndrome with any specific agent including chemotherapy, corticosteroids and/or growth factors (i.e. erythropoietin, G-CSF, GM-CSF, thrombopoietic agent); patients on chronic low-dose corticosteriods (< 20 mg/d) for reasons other than MDS are allowed
• Uncontrolled infections

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alvaro Moreno Aspitia

Role: PRINCIPAL_INVESTIGATOR

North Central Cancer Treatment Group

Locations

North Central Cancer Treatment Group

Rochester, Minnesota, United States

Countries

United States

Other Identifiers

N998B

Identifier Type: -

Identifier Source: secondary_id

U10CA025224

Identifier Type: NIH

Identifier Source: secondary_id

View Link

CDR0000068580

Identifier Type: REGISTRY

Identifier Source: secondary_id

NCI-2012-01856

Identifier Type: -

Identifier Source: org_study_id