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

NCT00166413 | Completed Phase 2 DMC

• 2 other identifiers: 1105-04MC0484
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Amyloidosis

Outcome Measures

Primary Outcomes (1)

• To evaluate the hematologic response rate of CC-5013 in patients with primary systemic amyloidosis

  12 months

Secondary Outcomes (5)

• 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

Study Arms (1)

CC5013

EXPERIMENTAL

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

Interventions

CC-5013 DRUG

40 mg/day orally on days 1-4 and 15-18 of each 28-day cycle

Also known as: amino substituted analog of thalidomide

CC5013

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Amyloidosis

Interventions

Lenalidomide

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Angela Dispenzieri, M.D.

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: September 12, 2005
• First Posted: September 14, 2005
• Study Start: April 1, 2005
• Primary Completion: December 1, 2006
• Last Updated: May 6, 2011

Record last verified: 2011-05

Locations

US (1)