Clinical trial NCT00015990: Thalidomide in Treating Patients With Myelodysplastic Syndrome
Official title: Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
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
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2004-02-27
Enrollment: 29
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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)

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

Primary outcomes:
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
timeFrame: Up to 3 months
measure: Incidence and severity of toxicities, graded according to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0
timeFrame: Up to 5 years
Secondary outcomes:
measure: Survival time
timeFrame: Time from registration to death due to any cause, assessed up to 5 years
measure: Time to disease progression
timeFrame: Time from registration to documentation of disease progression, assessed up to 5 years
measure: Duration of response measured using criteria reported by Cheson et al
timeFrame: 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
measure: Time to treatment failure
timeFrame: 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
Other outcomes:
Locations:
city: Rochester
country: United States
facility: North Central Cancer Treatment Group
state: Minnesota
Study officials:
affiliation: North Central Cancer Treatment Group
name: Alvaro Moreno Aspitia
role: PRINCIPAL_INVESTIGATOR
References: