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Thalidomide and Epoetin Alfa in Treating Anemia in Patients With Myelodysplastic Syndrome

Primary Purpose

Anemia, Leukemia, Myelodysplastic Syndromes

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
epoetin alfa
thalidomide
Sponsored by
Fallon Clinic
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Anemia focused on measuring de novo myelodysplastic syndromes, secondary myelodysplastic syndromes, previously treated myelodysplastic syndromes, anemia, atypical chronic myeloid leukemia, BCR-ABL1 negative, myelodysplastic/myeloproliferative neoplasm, unclassifiable, childhood myelodysplastic syndromes

Eligibility Criteria

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

DISEASE CHARACTERISTICS: Diagnosis of myelodysplastic syndromes Newly diagnosed OR Prior treatment was unsuccessful, including treatment with chemotherapy International prognostic scoring system score no greater than 1.5 Hemoglobin no greater than 10 g/dL (untransfused) AND/OR Received at least 3 units of packed red blood cells for symptomatic anemia within the past 6 weeks PATIENT CHARACTERISTICS: Age Over 21 Performance status Karnofsky 70-100% Life expectancy At least 6 months Hematopoietic See Disease Characteristics No prior bleeding disorder Hepatic Bilirubin less than 2 mg/dL ALT/AST less than 2 times upper limit of normal Renal Creatinine less than 1.5 mg/dL Cardiovascular No prior clinically significant heart disease No uncontrolled hypertension No recent thromboembolic disease (e.g., deep vein thrombosis) Prior thromboembolic events allowed provided event occurred at least 6 weeks prior to study and patient is on anticoagulants and is clinically stable Pulmonary No unstable pulmonary disease No recent pulmonary embolism No active pulmonary infection Neurologic No pre-existing peripheral neuropathy greater than grade 2 No sustained neurologic deficit No epilepsy Other Not pregnant or nursing Negative pregnancy test Fertile patients must use 2 effective methods (including 1 highly effective method) of contraception for at least 4 weeks before, during, and for at least 4 weeks after study completion No active infection No concurrent illness that would obscure toxicity or dangerously alter drug metabolism No other serious concurrent medical illness No uncontrolled diabetes mellitus No other malignant disease (except non-melanoma skin cancer or carcinoma in situ of the cervix) unless in complete remission and off therapy for that disease for more than 1 year No known hypersensitivity to mammalian cell-derived products or human albumin PRIOR CONCURRENT THERAPY: Biologic therapy Not specified Chemotherapy See Disease Characteristics Endocrine therapy Not specified Radiotherapy Not specified Surgery Not specified Other At least 4-6 weeks since prior therapy

Sites / Locations

  • Fallon Clinic at Worcester Medical Center
  • UMASS Memorial Cancer Center - University Campus

Outcomes

Primary Outcome Measures

Clinical response

Secondary Outcome Measures

Full Information

First Posted
January 27, 2003
Last Updated
June 25, 2013
Sponsor
Fallon Clinic
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1. Study Identification

Unique Protocol Identification Number
NCT00053001
Brief Title
Thalidomide and Epoetin Alfa in Treating Anemia in Patients With Myelodysplastic Syndrome
Official Title
A Phase II Study on the Effectiveness of Thalomid (Thalidomide) Combined With Procrit (Erythropoietin) for the Treatment of Anemia in Patients With Low and Intermediate Risk-1 (IPSS Score Less Than or Equal to 1.5) Myelodysplastic Syndromes
Study Type
Interventional

2. Study Status

Record Verification Date
October 2007
Overall Recruitment Status
Completed
Study Start Date
June 2001 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
October 2007 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Fallon Clinic

4. Oversight

5. Study Description

Brief Summary
RATIONALE: Thalidomide may stop or slow the growth of cancer cells. Epoetin alfa may stimulate red blood cell production. Combining thalidomide with epoetin alfa may improve anemia, decrease the need for blood transfusions, and improve the quality of life in patients with myelodysplastic syndrome. PURPOSE: Phase II trial to study the effectiveness of combining thalidomide with epoetin alfa in treating anemia in patients who have myelodysplastic syndrome.
Detailed Description
OBJECTIVES: Determine whether the combination of epoetin alfa and thalidomide improves the anemia and/or decreases the need for red cell transfusion in patients with low- or intermediate-risk myelodysplastic syndromes. Determine whether this regimen improves the bone marrow morphology and cytogenetics, alters the natural history of the disease, and reduces the frequency of leukemic transformation in these patients. Evaluate whether this regimen improves pathophysiologic parameters (e.g., apoptosis, tumor necrosis factor-alpha concentration, microvessel density, vascular endothelial growth factor, and cytotoxic T lymphocytes) in the bone marrow of these patients. Determine the safety of this regimen in these patients. OUTLINE: Patients receive epoetin alfa subcutaneously (SC) once weekly for 8 weeks. After 8 weeks, patients unresponsive to epoetin alfa alone receive oral thalidomide once daily in addition to epoetin alfa SC once weekly for a maximum of 24 weeks in the absence of disease progression or unacceptable toxicity. PROJECTED ACCRUAL: A total of 30-40 patients will be accrued for this study within 2 years..

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Keywords
de novo myelodysplastic syndromes, secondary myelodysplastic syndromes, previously treated myelodysplastic syndromes, anemia, atypical chronic myeloid leukemia, BCR-ABL1 negative, myelodysplastic/myeloproliferative neoplasm, unclassifiable, childhood myelodysplastic syndromes

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 2
Masking
None (Open Label)

8. Arms, Groups, and Interventions

Intervention Type
Biological
Intervention Name(s)
epoetin alfa
Intervention Type
Drug
Intervention Name(s)
thalidomide
Primary Outcome Measure Information:
Title
Clinical response

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
DISEASE CHARACTERISTICS: Diagnosis of myelodysplastic syndromes Newly diagnosed OR Prior treatment was unsuccessful, including treatment with chemotherapy International prognostic scoring system score no greater than 1.5 Hemoglobin no greater than 10 g/dL (untransfused) AND/OR Received at least 3 units of packed red blood cells for symptomatic anemia within the past 6 weeks PATIENT CHARACTERISTICS: Age Over 21 Performance status Karnofsky 70-100% Life expectancy At least 6 months Hematopoietic See Disease Characteristics No prior bleeding disorder Hepatic Bilirubin less than 2 mg/dL ALT/AST less than 2 times upper limit of normal Renal Creatinine less than 1.5 mg/dL Cardiovascular No prior clinically significant heart disease No uncontrolled hypertension No recent thromboembolic disease (e.g., deep vein thrombosis) Prior thromboembolic events allowed provided event occurred at least 6 weeks prior to study and patient is on anticoagulants and is clinically stable Pulmonary No unstable pulmonary disease No recent pulmonary embolism No active pulmonary infection Neurologic No pre-existing peripheral neuropathy greater than grade 2 No sustained neurologic deficit No epilepsy Other Not pregnant or nursing Negative pregnancy test Fertile patients must use 2 effective methods (including 1 highly effective method) of contraception for at least 4 weeks before, during, and for at least 4 weeks after study completion No active infection No concurrent illness that would obscure toxicity or dangerously alter drug metabolism No other serious concurrent medical illness No uncontrolled diabetes mellitus No other malignant disease (except non-melanoma skin cancer or carcinoma in situ of the cervix) unless in complete remission and off therapy for that disease for more than 1 year No known hypersensitivity to mammalian cell-derived products or human albumin PRIOR CONCURRENT THERAPY: Biologic therapy Not specified Chemotherapy See Disease Characteristics Endocrine therapy Not specified Radiotherapy Not specified Surgery Not specified Other At least 4-6 weeks since prior therapy
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Laszlo Leb, MD
Organizational Affiliation
Fallon Clinic
Official's Role
Study Chair
Facility Information:
Facility Name
Fallon Clinic at Worcester Medical Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01608
Country
United States
Facility Name
UMASS Memorial Cancer Center - University Campus
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States

12. IPD Sharing Statement

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Thalidomide and Epoetin Alfa in Treating Anemia in Patients With Myelodysplastic Syndrome

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