RATIONALE: CC-5013 may slow the progression of myelodysplasia and allow the body to produce
normal red blood cells.
PURPOSE: Phase II trial to study the effectiveness of CC-5013 in treating patients who
require red blood cell transfusions for anemia caused by myelodysplastic syndrome associated
with a cytogenetic abnormality.
- Determine the efficacy of CC-5013, in terms of hematological improvement, in patients
with red blood cell transfusion-dependent low- or intermediate-risk myelodysplastic
syndromes and a del(5)(q31q33) cytogenetic abnormality.
- Determine the safety of this drug in these patients.
OUTLINE: This is an open-label, multicenter study.
Patients receive oral CC-5013 on days 1-21. Treatment repeats every 28 days for up to 6
courses in the absence of disease progression or unacceptable toxicity.
PROJECTED ACCRUAL: A total of 36 patients will be accrued for this study.
- Diagnosis of low- or intermediate-risk myelodysplastic syndromes (MDS) associated
with a del(5)(q31q33) cytogenetic abnormality
- Cytogenetic abnormality may be an isolated cytogenetic finding (the 5q-
syndrome) OR may be associated with other cytogenetic abnormalities
- Red blood cell (RBC) transfusion-dependent anemia defined as having received at least
2 units of RBCs within the past 8 weeks
- 18 and over
- ECOG 0-2
- Not specified
- Absolute neutrophil count at least 500/mm^3
- Platelet count at least 50,000/mm^3
- No clinically significant anemia due to iron, B_12, or folate deficiency, autoimmune
or hereditary hemolysis, or gastrointestinal bleeding
- If marrow aspirate not evaluable for storage iron, the following criteria must be
- Transferrin saturation at least 20%
- Serum ferritin at least 50 ng/mL
- Bilirubin no greater than 2.0 mg/dL
- AST and ALT no greater than 3.0 times upper limit of normal
- Creatinine no greater than 2.5 mg/dL
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- No prior grade 3 or greater allergic reaction or hypersensitivity to thalidomide
- No prior grade 3 or greater rash or any desquamation (blistering) from thalidomide
- No other serious medical condition, laboratory abnormality, or psychiatric illness
that would preclude study participation or giving informed consent or confound study
- No other malignancy within the past 3 years except basal cell or squamous cell skin
cancer or carcinoma in situ of the cervix or breast
PRIOR CONCURRENT THERAPY:
- No prior CC-5013
- More than 7 days since prior hematopoietic growth factors
- No concurrent epoetin alfa for MDS
- More than 28 days since prior experimental or standard chemotherapy for MDS
- No concurrent chemotherapy for MDS
- More than 28 days since prior chronic use (greater than 2 weeks in duration) of more
than physiologic doses of corticosteroids
- No concurrent androgens for MDS
- Not specified
- Not specified
- More than 28 days since prior experimental or standard immunosuppressive or
cytoprotective agents for MDS
- More than 28 days since other prior experimental or standard drugs or therapy for MDS
- No other concurrent investigational agents for MDS