Patients with primary systemic amyloidosis will be treated with CC-5013 (lenalidomide; Revlimid) as a single agent for 3 months. If their disease worsens or does not improve during that time frame dexamethasone will be added to the treatment program.
1. Histochemical diagnosis of amyloidosis as based on detection by polarizing microscopy of green birefringent material in Congo red-stained tissue specimens and immunohistochemical proof of AL
2. Measurable disease of AL amyloidosis as defined by one of the following:
- Serum monoclonal protein >=1.0 g by protein electrophoresis
- >200 mg of monoclonal protein in the urine on 24 hour electrophoresis
- Serum immunoglobulin free light chain & >=10 mg/dL AND abnormal serum immunoglobulin kappa to lambda free light chain ratio
3. ECOG performance status (PS) 0, 1, 2, or 3
4. >=18 years of age
5. The following laboratory values obtained <=14 days prior to registration:
- Creatinine < = 3 mg/dL
- Absolute neutrophil count >=1000/microliter
- Platelet >=75000/microliter
- Hemoglobin > = 8.0 g/dL
6. Symptomatic organ involvement with amyloid to justify therapy. This could include liver involvement, cardiac involvement, renal involvement, peripheral neuropathy grade 1, or soft tissue involvement. Must have more than purpura or carpal tunnel syndrome
7. Previously treated or untreated. No limit to prior therapy provided there is adequate residual organ function
8. Ability to provide informed consent
9. Anticipated life expectancy of at least 3 months
10. None of the following:
- Pregnant women or women of reproductive ability who are unwilling to use effective contraception
- Nursing women
- Men who are unwilling to use a condom (even if they have undergone a prior vasectomy) while having intercourse with any woman, while taking the drug and for 4 weeks after stopping treatment
- Myelosuppressive chemotherapy < 4 weeks prior to registration
- Concomitant high dose corticosteroids
- Grade 2 (or higher) peripheral neuropathy
- Uncontrolled infection
- Clinically overt multiple myeloma
- Active malignancy
- Prior hypersensitivity reaction to Thalidomide
- Syncope within the past 30 days
Before you begin your study treatment, you will have routine blood tests (6 - 8 teaspoons), urine tests, echocardiogram, electrocardiogram (EKG), bone marrow biopsy, fat aspirate, and x-rays of your bones and chest will be done to make sure that you are eligible for this study. If you are a woman of child bearing potential, you will be required to have a negative pregnancy test 7 days before to the initiation of CC-5013 and before each cycle. You will be required to use birth control if you take part in this study. If you have had a hysterectomy or no menstrual periods for at least 24 consecutives months, will you not be required to have these pregnancy tests and use birth control.
You will take CC-5013 (25 mg) once a day for 21 days of a 28 day cycle. If after 3 cycles (about 3 months), you doctor does not see evidence of improvement in your disease as judged by blood and urine tests, s/he will add another medication called dexamethasone. You will take this medicine for a total of 8 days in a given 28 day cycle, that is days 1-4 and 15-18 of the 28 day cycle. The doses of the drugs may have to be reduced if you have significant side-effects.
You will have blood tests (1-2 teaspoons in volume) done twice a month for the first 3 months then every month during treatment, and every 6 months when your treatment is completed (6-8 teaspoons in volume). Blood test may be done at your own doctor's office at weeks 2, 6 and 10. Urine tests and an echocardiogram will also be done every 3 to 6 months. CT scans and ultrasounds will be done according to clinical need. A bone marrow biopsy will be done as clinically required. You will be required to return to the Mayo Clinic every month for the first three months and then every 3 months after that. If your disease progresses at any time during the study, CC-5013 (and dexamethasone, if you are taking that too) will be stopped.
You will receive CC-5013 with or without dexamethasone as long as it appears to be helping your disease. If there are bad side effects, evidence of disease progression, or you or your doctor decide that better treatment choices are available, the CC-5013 with or without dexamethasone will be stopped sooner. As long as your disease is improving or remaining stable, we would expect that you would return to Mayo at least every three months. If you stop the drug(s) because of side effects or disease progression, we would still like to check your progress (if even by telephone contact only) every 6 months. Your progress will be followed every 6 months for up to five years after registration, but you can tell us you no longer want to be contacted.
Last updated: 05/05/2011
NCT ID: NCT00166413
IRB Number:1105-04
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