Thalidomide in Treating Patients With Myelodysplastic Syndrome

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00015990
First received: May 6, 2001
Last updated: January 23, 2013
Last verified: January 2013
  Purpose

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


Condition Intervention Phase
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
Drug: thalidomide
Other: laboratory biomarker analysis
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study

Resource links provided by NLM:


Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Survival time [ Time Frame: Time from registration to death due to any cause, assessed up to 5 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 29
Study Start Date: April 2001
Primary Completion Date: February 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment (thalidomide)
Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.
Drug: thalidomide
Given orally
Other Names:
  • Kevadon
  • Synovir
  • THAL
  • Thalomid
Other: laboratory biomarker analysis
Correlative studies

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)

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00015990

Locations
United States, Minnesota
North Central Cancer Treatment Group
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Investigators
Principal Investigator: Alvaro Moreno Aspitia North Central Cancer Treatment Group
  More Information

No publications provided

Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00015990     History of Changes
Other Study ID Numbers: NCI-2012-01856, N998B, U10CA025224, CDR0000068580
Study First Received: May 6, 2001
Last Updated: January 23, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Leukemia, Myelomonocytic, Acute
Leukemia, Myelomonocytic, Chronic
Anemia, Refractory
Anemia
Myelodysplastic Syndromes
Preleukemia
Anemia, Refractory, with Excess of Blasts
Syndrome
Leukemia, Myeloid
Leukemia
Neoplasms by Histologic Type
Neoplasms
Myelodysplastic-Myeloproliferative Diseases
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Disease
Pathologic Processes
Thalidomide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances

ClinicalTrials.gov processed this record on September 16, 2014