A Phase II Trial of the Immunomodulatory Drug CC-5013 for Patients With AL Amyloidosis
18 Years
N/A
Both
Multiple Myeloma and Plasma Cell Neoplasm
Trial Information
A Phase II Trial of the Immunomodulatory Drug CC-5013 for Patients With AL 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
- No secondary or familial amyloidosis
- No multiple myeloma, defined as ≥ 30% plasma cells in bone marrow biopsy specimen OR
lytic bone lesions
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
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
PRIOR CONCURRENT THERAPY:
Biologic therapy
- No prior CC-5013
- 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
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Tolerability and objective hematologic response with CC-5013 every 3 months
Outcome Time Frame:
3 months
Safety Issue:
Yes
Principal Investigator
David C. Seldin, MD, PhD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Boston Medical Center
Authority:
United States: Federal Government
Study ID:
CDR0000385687
NCT ID:
NCT00091260
Start Date:
January 2004
Completion Date:
Related Keywords:
- Multiple Myeloma and Plasma Cell Neoplasm
- primary systemic amyloidosis
- Amyloidosis
- Neoplasms
- Multiple Myeloma
- Neoplasms, Plasma Cell
- Plasmacytoma
Name | Location |
|---|---|
| Cancer Research Center at Boston Medical Center | Boston, Massachusetts 02118 |
