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Thalidomide in Treating Patients With Myelodysplastic Syndrome

Primary Purpose

Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
thalidomide
laboratory biomarker analysis
Sponsored by
National Cancer Institute (NCI)
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Myelomonocytic Leukemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • North Central Cancer Treatment Group

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment (thalidomide)

Arm Description

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

Outcomes

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
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

Secondary Outcome Measures

Survival time
Estimated using the method of Kaplan-Meier.
Time to disease progression
Estimated using the method of Kaplan-Meier.
Duration of response measured using criteria reported by Cheson et al
Time to treatment failure

Full Information

First Posted
May 6, 2001
Last Updated
January 23, 2013
Sponsor
National Cancer Institute (NCI)
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1. Study Identification

Unique Protocol Identification Number
NCT00015990
Brief Title
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

2. Study Status

Record Verification Date
January 2013
Overall Recruitment Status
Completed
Study Start Date
April 2001 (undefined)
Primary Completion Date
February 2007 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Cancer Institute (NCI)

4. Oversight

5. Study Description

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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
29 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment (thalidomide)
Arm Type
Experimental
Arm Description
Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence of disease progression or unacceptable toxicity.
Intervention Type
Drug
Intervention Name(s)
thalidomide
Other Intervention Name(s)
Kevadon, Synovir, THAL, Thalomid
Intervention Description
Given orally
Intervention Type
Other
Intervention Name(s)
laboratory biomarker analysis
Intervention Description
Correlative studies
Primary Outcome Measure Information:
Title
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
Description
Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
Time Frame
Up to 3 months
Title
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
Secondary Outcome Measure Information:
Title
Survival time
Description
Estimated using the method of Kaplan-Meier.
Time Frame
Time from registration to death due to any cause, assessed up to 5 years
Title
Time to disease progression
Description
Estimated using the method of Kaplan-Meier.
Time Frame
Time from registration to documentation of disease progression, assessed up to 5 years
Title
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
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alvaro Moreno Aspitia
Organizational Affiliation
North Central Cancer Treatment Group
Official's Role
Principal Investigator
Facility Information:
Facility Name
North Central Cancer Treatment Group
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States

12. IPD Sharing Statement

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Thalidomide in Treating Patients With Myelodysplastic Syndrome

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