Efficacy of CC-5013 (Revlimid or Lenalidomide) in Patients With Primary Systemic Amyloidosis

Study Overview
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Brief Summary
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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.

Official Title
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A Phase II Trial of CC-5013 in Patients With Primary Systemic Amyloidosis

Conditions
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Amyloidosis

Intervention / Treatment
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* Drug: CC-5013


Participation Criteria
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Eligibility Criteria
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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 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 ECOG performance status (PS) 0, 1, 2, or 3 >=18 years of age 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 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 Previously treated or untreated. No limit to prior therapy provided there is adequate residual organ function Ability to provide informed consent Anticipated life expectancy of at least 3 months 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CC5013<br>Assess the proportion of confirmed hematologic responses (HCR, HPR) resulting from treatment with CC5013 after 3 months in patients with primary systemic amyloidosis. | Drug: CC-5013<br>* 40 mg/day orally on days 1-4 and 15-18 of each 28-day cycle<br>* Other names: amino substituted analog of thalidomide;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the hematologic response rate of CC-5013 in patients with primary systemic amyloidosis |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Toxicity of single agent CC-5013 and combination CC-5013 and dexamethasone |  | 12 months | 
| Hematologic response rate of CC-5013 and dexamethasone |  | 12 months | 
| Organ response of CC-5013 and the CC-5013 dexamethasone combination |  | 12 months | 
| Time to progression |  | 5 years | 
| Survival |  | 5 years |