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
21 Years
N/A
Both
Anemia, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Trial Information
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
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..
Inclusion 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
Type of Study:
Interventional
Study Design:
Masking: Open Label, Primary Purpose: Supportive Care
Outcome Measure:
Clinical response
Principal Investigator
Laszlo Leb, MD
Investigator Role:
Study Chair
Investigator Affiliation:
Fallon Clinic
Authority:
United States: Federal Government
Study ID:
FALLON-PR01-09-010
NCT ID:
NCT00053001
Start Date:
June 2001
Completion Date:
October 2007
Related Keywords:
- Anemia
- Leukemia
- Myelodysplastic Syndromes
- Myelodysplastic/Myeloproliferative Neoplasms
- 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
- Anemia
- Neoplasms
- Leukemia
- Myelodysplastic Syndromes
- Preleukemia
- Myeloproliferative Disorders
- Myelodysplastic-Myeloproliferative Diseases
Name | Location |
|---|---|
| UMASS Memorial Cancer Center - University Campus | Worcester, Massachusetts 01605-2982 |
| Fallon Clinic at Worcester Medical Center | Worcester, Massachusetts 01608 |
