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Phase II Trial of Enbrel in Patients With Primary Systemic Amyloidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00224393
Recruitment Status : Suspended
First Posted : September 23, 2005
Last Update Posted : September 23, 2005
Information provided by:
The Cleveland Clinic

Brief Summary:
The purpose of this study is to evaluate the efficacy of Enbrel in patients with primary systemic Amyloidosis.

Condition or disease Intervention/treatment Phase
Primary Systemic Amyloidosis Drug: Enbrel Phase 2

Detailed Description:
The primary goal of this study is to evaluate the efficacy of Enbrel in patients with primary systemic amyloidosis using a one-stage, phase II study design with an interim analysis. This study will also assess survival and progression times, symptom relief, and toxicity associated with Enbrel in primary systemic amyloidosis patients. Two groups of patients with very different risk profiles can be identified with respect to this disease. Patients with symptomatic cardiac disease and/or at least two involved organs ar at high risk and historically have a median survival less than six months. Patients without these conditions have a significantly better prognosis. Both subgroups will be studied in the present study, and essentially two phase-II clinical trials, one for each subgroup, will be run in parallel.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : February 2001
Study Completion Date : August 2005

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis
Drug Information available for: Etanercept

Primary Outcome Measures :
  1. clinical efficacy

Secondary Outcome Measures :
  1. Duration of response and time to progression
  2. Evaluate overall survival
  3. Identify prognostic factors
  4. Evaluate qualitative and quantitative toxicities of Enbrel

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • >=18 years of age.
  • Laboratory values obtained <=14 days prior to registration.
  • No limitation on the cardiac ejection fraction
  • Bilirubin <3 mg/dL
  • Absolute neutrophil count >=500/microliters
  • Histochemical diagnosis of amyloidosis as based on detection by polarizing microscopy of green bi-refringent material in Congo red-stained tissue specimens or characteristic electron microscopy appearance.
  • Demonstrable M-protein in the serum/urine or clonal population of plasma cells in the bone marrow or immunohistochemical stain with anti-light chain anti-sera of amyloid fibrils.
  • ECOG performance status 0, 1, 2, or 3.
  • Symptomatic organ involvement with amyloid to justify therapy. This could include liver involvement, cardiac involvement, renal involvement, peripheral neuropathy, 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.
  • Ability to self-inject medication or have a caregiver who can administer the drug.

Exclusion Criteria:

  • Amyloid-specific syndrome, such as, carpal tunnel syndrome or skin purpura as only evidence of disease. The finding of vascular amyloid only in a bone marrow biopsy specimen or in a plasmacytoma is not indicative of systemic amyloidosis.
  • Presence of non-AL amyloidosis.
  • Melphalan or other alkylating agents, high-dose dexamethasone or alpha interferon <=4 weeks prior to registration.
  • Concurrent use of corticosteroids, but patients may be on chronic steroids if they are being given for disorders other than amyloid, i.e., adrenal insufficiency, rheumatoid arthritis, etc.
  • Any of the following:

    • Pregnant women
    • Nursing women
    • Men or women of childbearing potential who are unwilling to employ adequate contraception (condoms, diaphragm, birth control pills, injections, intrauterine device [IUD], surgical sterilization, abstinence, etc.)
  • Uncontrolled infection.
  • Clinically overt multiple myeloma (monoclonal BMPC >30%), and at least one of the following:

    • Bone lesions
    • Hypercalcemia
  • Active malignancy with the exception of adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease-free for 5 years.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00224393

Sponsors and Collaborators
The Cleveland Clinic
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Principal Investigator: Mohamad A Hussein, MD The Cleveland Clinic
Study Chair: John A Lust, MD, PhD Mayo Clinic

Layout table for additonal information Identifier: NCT00224393    
Other Study ID Numbers: MC018A
First Posted: September 23, 2005    Key Record Dates
Last Update Posted: September 23, 2005
Last Verified: August 2005
Keywords provided by The Cleveland Clinic:
Additional relevant MeSH terms:
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Immunoglobulin Light-chain Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Gastrointestinal Agents
Immunosuppressive Agents
Immunologic Factors