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


Phase 2
18 Years
N/A
Not Enrolling
Both
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

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Trial Information

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


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)


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

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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

Outcome Description:

Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.

Outcome Time Frame:

Up to 3 months

Safety Issue:

No

Principal Investigator

Alvaro Moreno Aspitia

Investigator Role:

Principal Investigator

Investigator Affiliation:

North Central Cancer Treatment Group

Authority:

United States: Food and Drug Administration

Study ID:

NCI-2012-01856

NCT ID:

NCT00015990

Start Date:

April 2001

Completion Date:

Related Keywords:

  • 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
  • Anemia
  • Anemia, Refractory
  • Anemia, Refractory, with Excess of Blasts
  • Leukemia
  • Leukemia, Myelomonocytic, Chronic
  • Myelodysplastic Syndromes
  • Preleukemia
  • Leukemia, Myelomonocytic, Acute
  • Anemia, Aplastic

Name

Location

North Central Cancer Treatment GroupRochester, Minnesota  55905