Phase II Trial of Enbrel in Patients With Primary Systemic Amyloidosis

This study has suspended participant recruitment.
Sponsor:
Information provided by:
The Cleveland Clinic
ClinicalTrials.gov Identifier:
NCT00224393
First received: September 21, 2005
Last updated: NA
Last verified: August 2005
History: No changes posted
  Purpose

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


Condition Intervention Phase
Primary Systemic Amyloidosis
Drug: Enbrel
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by The Cleveland Clinic:

Primary Outcome Measures:
  • clinical efficacy

Secondary Outcome Measures:
  • Duration of response and time to progression
  • Evaluate overall survival
  • Identify prognostic factors
  • Evaluate qualitative and quantitative toxicities of Enbrel

Estimated Enrollment: 60
Study Start Date: February 2001
Estimated Study Completion Date: August 2005
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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00224393

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

No publications provided

ClinicalTrials.gov Identifier: NCT00224393     History of Changes
Other Study ID Numbers: MC018A
Study First Received: September 21, 2005
Last Updated: September 21, 2005
Health Authority: United States: Institutional Review Board

Keywords provided by The Cleveland Clinic:
Amyloidosis
Enbrel

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
TNFR-Fc fusion protein
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Pharmacologic Actions
Anti-Inflammatory Agents
Therapeutic Uses
Antirheumatic Agents
Gastrointestinal Agents
Immunologic Factors
Immunosuppressive Agents
Central Nervous System Agents

ClinicalTrials.gov processed this record on August 21, 2014