Clinical trial NCT00166413: Efficacy of CC-5013 (Revlimid or Lenalidomide) in Patients With Primary Systemic Amyloidosis
Official title: A Phase II Trial of CC-5013 in Patients With Primary Systemic Amyloidosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyloidosis
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
First posted: 2005-09-14
Enrollment: 38
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: To evaluate the hematologic response rate of CC-5013 in patients with primary systemic amyloidosis
timeFrame: 12 months
Secondary outcomes:
measure: Toxicity of single agent CC-5013 and combination CC-5013 and dexamethasone
timeFrame: 12 months
measure: Hematologic response rate of CC-5013 and dexamethasone
timeFrame: 12 months
measure: Organ response of CC-5013 and the CC-5013 dexamethasone combination
timeFrame: 12 months
measure: Time to progression
timeFrame: 5 years
measure: Survival
timeFrame: 5 years
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Angela Dispenzieri, M.D.
role: PRINCIPAL_INVESTIGATOR
References: