Primary Systemic AmyloidosisPhase 2NCT00224393
Phase II Trial of Enbrel in Patients With Primary Systemic Amyloidosis
A Phase 2 study testing Enbrel in people with Primary Systemic Amyloidosis, run by The Cleveland Clinic.
Trial summary
The purpose of this study is to evaluate the efficacy of Enbrel in patients with primary systemic Amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Systemic Amyloidosis
- What is being tested?
- Enbrel
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- Feb 2001to 31 Aug 2005
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- >=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.
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- 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.
Who cannot join
- 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
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- 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.
About this study
The purpose of this study is to evaluate the efficacy of Enbrel in patients with primary systemic Amyloidosis.
Read the 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.
- Conditions
- Primary Systemic Amyloidosis
- Being tested
- Enbrel
- Keywords
- Amyloidosis, Enbrel
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EnbrelDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
clinical efficacy
Secondary outcomes (4)
Duration of response and time to progression
Evaluate overall survival
Identify prognostic factors
Evaluate qualitative and quantitative toxicities of Enbrel
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2005 (estimated)
- Study startFeb 2001
- Study completion31 Aug 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Systemic AmyloidosisStudies of the same condition, open and under way.
- More trials run by The Cleveland ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants
Sponsor and investigators
- Lead sponsor
- The Cleveland Clinic
- Sponsor type
- University, hospital or other
- Investigators
- Mohamad A Hussein, MD, Principal investigator, The Cleveland ClinicJohn A Lust, MD, PhD, Study chair, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00224393
- Sponsor's protocol ID
- MC018A
- Status last verified
- May 2023
- First submitted
- 21 Sep 2005
- Last updated
- 30 May 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Enbrel in Patients With Primary Systemic Amyloidosis
- ClinicalTrials.gov ID
- NCT00224393
- Acronym / sponsor ID
- MC018A
- Conditions
- Primary Systemic Amyloidosis
- Keywords
- Amyloidosis, Enbrel
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants
- Lead sponsor
- The Cleveland ClinicUniversity, hospital or other
- Study start
- Feb 2001
- Study completion
- 31 Aug 2005 (actual)
- First posted
- 23 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy of Enbrel in patients with primary systemic Amyloidosis.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- >=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
Key exclusion criteria (13 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Enbrel | Drug | – |
Primary outcome
clinical efficacy
4 secondary outcomes
Duration of response and time to progression
Evaluate overall survival
Identify prognostic factors
Evaluate qualitative and quantitative toxicities of Enbrel
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mohamad A Hussein, MD principal investigator
The Cleveland Clinic
John A Lust, MD, PhD study chair
Mayo Clinic
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Enbrel in people with Primary Systemic Amyloidosis, run by The Cleveland Clinic.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 May 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
clinical efficacy. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 May 2023.