Phase II Trial of Enbrel in Patients With Primary Systemic Amyloidosis
Information source: The Cleveland Clinic
Information obtained from ClinicalTrials.gov on December 31, 2007
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Primary Systemic Amyloidosis
Intervention: Enbrel (Drug)
Phase: Phase 2
Sponsored by: The Cleveland Clinic
Official(s) and/or principal investigator(s):
Mohamad A Hussein, MD, Principal Investigator, Affiliation: The Cleveland Clinic
John A Lust, MD, PhD, Study Chair, Affiliation: Mayo Clinic
The purpose of this study is to evaluate the efficacy of Enbrel in patients with primary
Study design: Treatment, Non-Randomized, Open Label, Active Control, Parallel Assignment, Efficacy Study
Primary outcome: clinical efficacy
Duration of response and time to progression
Evaluate overall survival
Identify prognostic factors
Evaluate qualitative and quantitative toxicities of Enbrel
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
Minimum age: 18 Years.
Maximum age: N/A.
>=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
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
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
Any of the following:
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.)
Clinically overt multiple myeloma (monoclonal BMPC >30%), and at least one of the
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.
Locations and Contacts
Starting date: February 2001
Ending date: August 2005
Last updated: September 21, 2005