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

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

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.

Study Overview

• Status: Completed
• Conditions: Amyloidosis
• Intervention / Treatment: Drug: CC-5013

• Study Type: Interventional
• Enrollment (Anticipated): 38
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CC5013; 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; 40 mg/day orally on days 1-4 and 15-18 of each 28-day cycle; Other Names: amino substituted analog of thalidomide

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Time Frame
Time to progression	5 years
Survival	5 years
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

Collaborators and Investigators

• Sponsor: Mayo Clinic
• Investigators: Principal Investigator: Angela Dispenzieri, M.D., Mayo Clinic

Study record dates

Study Major Dates

• Study Start: April 1, 2005
• Primary Completion (Actual): December 1, 2006

Study Registration Dates

• First Submitted: September 12, 2005
• First Submitted That Met QC Criteria: September 12, 2005
• First Posted (Estimate): September 14, 2005

Study Record Updates

• Last Update Posted (Estimate): May 6, 2011
• Last Update Submitted That Met QC Criteria: May 5, 2011
• Last Verified: May 1, 2011

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Paraproteinemias; Proteostasis Deficiencies; Neoplasms, Plasma Cell; Immunoglobulin Light-chain Amyloidosis; Amyloidosis; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Anti-Bacterial Agents; Leprostatic Agents; Thalidomide; Lenalidomide
• Other Study ID Numbers: 1105-04 (Other Identifier: Mayo Clinic IRB); MC0484 (Other Identifier: Mayo Clinic Cancer Center)