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

Mayo Clinic

Status and phase: Completed; Phase 2

Conditions: Amyloidosis

Treatments: Drug: CC-5013

Study type: Interventional

Funder types: Other

Identifiers

NCT00166413

1105-04 (Other Identifier)

MC0484 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 38 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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38 participants in 1 patient group

CC5013

Experimental group

Description:

Assess the proportion of confirmed hematologic responses (HCR, HPR) resulting from treatment with CC5013 after 3 months in patients with primary systemic amyloidosis.

Treatment:

Drug: CC-5013