Phase II Trial of Enbrel in Patients With Primary Systemic Amyloidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 5 June 2023
• Main ID: NCT00224393
• Date of registration: 21/09/2005
• Prospective Registration: No
• Primary sponsor: The Cleveland Clinic
• Public title: Phase II Trial of Enbrel in Patients With Primary Systemic Amyloidosis
• Date of first enrolment: February 2001
• Target sample size: 60
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00224393
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Contacts

• Name: Mohamad A Hussein, MD
• Affiliation: The Cleveland Clinic

• Name: John A Lust, MD, PhD
• Affiliation: Mayo Clinic

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Systemic Amyloidosis

Intervention(s)

Drug: Enbrel

Primary Outcome(s)

clinical efficacy

Secondary Outcome(s)

Evaluate overall survival

Identify prognostic factors

Duration of response and time to progression

Evaluate qualitative and quantitative toxicities of Enbrel

Secondary ID(s)

MC018A

Source(s) of Monetary Support

Please refer to primary and secondary sponsors