CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis

OBJECTIVES:

Primary

- Determine the tolerability of CC-5013 in patients with primary systemic (AL)
amyloidosis.

- Determine the objective hematologic response rate in patients treated with this drug.

- Determine amyloid organ disease response in patients treated with this drug.

Secondary

- Determine hematologic and amyloid organ disease response in patients who do not achieve
a response to CC-5013 alone and are subsequently treated with CC-5013 and
dexamethasone.

- Determine the toxicity of CC-5013 in combination with dexamethasone in these patients.

OUTLINE: Patients receive oral CC-5013 once daily on days 1-21. Courses repeat every 28 days
in the absence of disease progression or unacceptable toxicity. Patients not achieving a
hematologic response continue to receive CC-5013 as before and also receive oral
dexamethasone twice daily on days 1-4, 9-12, and 17-20 of every other 28-day course for up
to 6 courses of combination therapy. Patients who maintain a hematologic response after 6
courses of combination therapy may receive CC-5013 alone in the absence of disease
progression or unacceptable toxicity. Patients not achieving a hematologic response after
the initiation of dexamethasone are removed from the study.

Patients are followed annually.

PROJECTED ACCRUAL: A total of 15-25 patients will be accrued for this study within 5-12.5
months.

Inclusion Criteria:

DISEASE CHARACTERISTICS:

- Histologically confirmed primary systemic (AL) amyloidosis

- Tissue amyloid deposits or positive fat aspirate

- Meets 1 of the following criteria for AL type disease:

- Serum or urine monoclonal protein by immunofixation electrophoresis

- Plasmacytosis of bone marrow by monoclonal staining for kappa- or lambda-light
chain isotype

PATIENT CHARACTERISTICS:

Age

- 18 and over

Performance status

- SWOG 0-2

Life expectancy

- Not specified

Hematopoietic

- WBC > 3,000/mm^3

- Hemoglobin > 8 g/dL

- Platelet count > 100,000/mm^3

- Absolute neutrophil count > 1,000/mm^3

Hepatic

- Bilirubin ≤ 2 times upper limit of normal (ULN)

- AST and ALT ≤ 2 times ULN

PRIOR CONCURRENT THERAPY:

Biologic therapy

- Prior thalidomide for AL amyloidosis allowed

Chemotherapy

- More than 4 weeks since prior cytotoxic chemotherapy

Endocrine therapy

- Prior steroids for AL amyloidosis allowed

Radiotherapy

- More than 4 weeks since prior radiotherapy

Surgery

- Prior surgery allowed

Other

- Recovered from all prior therapy

Exclusion Criteria:

- No secondary or familial amyloidosis

- No multiple myeloma, defined as ≥ 30% plasma cells in bone marrow biopsy specimen OR
lytic bone lesions

- No prior CC-5013

Renal

- No dialysis

Cardiovascular

- No symptomatic cardiac arrhythmia

- No oxygen-dependent restrictive cardiomyopathy

Other

- No untreated or uncontrolled infection

- No other malignancy except basal cell skin cancer or carcinoma in situ of the cervix
or breast

- No other serious medical illness that would preclude study participation

- No history of hypersensitivity reaction to thalidomide

- HIV negative

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception