Multiple MyelomaPhase 2NCT00790647
Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
A Phase 2 study testing filgrastim, bortezomib and melphalan in people with Multiple Myeloma, run by Boston Medical Center at 1 site in United States.
Trial summary
RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- filgrastim, bortezomib and melphalan
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2008to Nov 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- DISEASE CHARACTERISTICS:
- Histologically confirmed primary systemic amyloidosis based on the following criteria:
- Amyloid light-chain disease
- Deposition of amyloid material by congo red stain showing characteristic green birefringence
- Monoclonal light chain protein (Bence Jones protein) in the serum or urine, immunohistochemical studies, or serum free light chain assay
- Evidence of tissue involvement other than carpal tunnel syndrome (i.e., positive immunohistochemical staining of bone marrow demonstrating clonal plasma cells); tissue amyloid deposits with anti-kappa or anti-lambda anti-serum; evidence for a plasma cell dyscrasia by serum/urine or bone marrow; or overwhelmingly convincing clinical features (e.g., macroglossia) associated with other systemic manifestations
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- PATIENT CHARACTERISTICS:
- Southwest Oncology Group performance status 0-1
- Fertile patients must use effective contraception
- Left ventricular ejection fraction ≥ 45% by Echocardiogram within the past 60 days
- diffusion capacity of lung for carbon monoxide ≥ 50%
- PRIOR CONCURRENT THERAPY:
- Prior chemotherapy with alkylating agent allowed provided there is no morphological or cytogenetic evidence of myelodysplastic syndromes
- Prior total cumulative dose of oral melphalan < 300 mg
- At least 4 weeks since prior cytotoxic therapy and fully recovered
Who cannot join
- No senile, secondary, localized, dialysis-related, or familial amyloidosis
- No overt multiple myeloma (> 30% of bone marrow plasmacytosis, extensive [> 2] lytic lesions, or hypercalcemia)
- Not pregnant or nursing
- No myocardial infarction within the past 6 months, congestive heart failure, or arrhythmia refractory to therapy
- No prior malignancy except for any of the following:
- Adequately treated basal cell or squamous cell skin cancer
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- In situ cervical cancer
- Adequately treated stage I or II cancer currently in complete remission
- Any cancer from which the patient has been disease-free ≥ 5 years
- No advanced (grade 3-4) pre-existing neuropathy
- No HIV positivity
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston University Cancer Research Center
Boston, Massachusetts, 02118
About this study
Official title: Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.
Read the detailed description
OBJECTIVES:
- To determine if hematologic responses to high-dose melphalan and autologous stem cell transplantation increase with addition of bortezomib in the conditioning regimen in patients with primary systemic amyloidosis.
OUTLINE:
- Autologous stem cell mobilization and collection: Patients receive filgrastim to mobilize stem cells, which are then collected.
- Conditioning regimen: Patients receive bortezomib intravenously on days -6, -3, 1, and 4 and oral high-dose melphalan on days -2 and -1.
- Stem cell transplantation: Patients undergo autologous stem cell transplantation on day 0.
After completion of study therapy, patients are followed every 6 months for 1 year and annually thereafter.
- Conditions
- Multiple Myeloma
- Being tested
- filgrastim, bortezomib, melphalan, Stem Cell Infusion
- Keywords
- primary systemic amyloidosis
- Medical subject headings
- Multiple Myeloma, Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
Also called Growth-Colony Stimulating Factor, neupogen
16 mcg/kg daily beginning 3 days before stem cell collection through day before final stem cell collection
bortezomibDrug
Also called velcade
1.0 mg/m2/dose D -6, D-3, D +1, D + 4
melphalanDrug
Also called alkeran
100 mg/m2/dose D -2, D -1
Stem Cell InfusionProcedure or surgery
infusion of previously collected autologous stem cells
| Study group | Role | What they receive |
|---|---|---|
| Stem Cell Transplant with Bortezomib and Melphalan | Experimental | Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion Receives: Biological: filgrastim; Drug: bortezomib; Drug: melphalan; Procedure: Stem Cell Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Hematologic Response
complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy <5% plasma cells with no clonal predominance of kappa or lambda isotype.
Partial response: any one of the following
- For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells.
- For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more.
- For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).
Measured: one year
Secondary outcomes (3)
Number of Participants Surviving at 100 Days From Transplant
Measured: 100 Days from transplant date
Number of Participants Surviving at 1 Year
Measured: one year from transplant
Number of Participants Surviving at 2 Years
Measured: 2 years from transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2008 (estimated)
- Study startJun 2008
- Primary completionDec 2012
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- More trials run by Boston Medical CenterThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vaishali Sanchorawala, Boston Medical Center
- Investigators
- Vaishali Sanchorawala, MD, Principal investigator, Boston Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00790647
- Sponsor's protocol ID
- CDR0000618857
- Other IDs
- BUMC-H-27277
- Status last verified
- Dec 2016
- First submitted
- 12 Nov 2008
- Last updated
- 6 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
- ClinicalTrials.gov ID
- NCT00790647
- Acronym / sponsor ID
- CDR0000618857
- Conditions
- Multiple Myeloma
- Keywords
- primary systemic amyloidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Boston Medical CenterUniversity, hospital or other
- Study start
- Jun 2008
- Primary completion
- Dec 2012 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 13 Nov 2008 (estimated)
- Results
- Posted 6 Feb 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.
Detailed description
OBJECTIVES:
- To determine if hematologic responses to high-dose melphalan and autologous stem cell transplantation increase with addition of bortezomib in the conditioning regimen in patients with primary systemic amyloidosis.
OUTLINE:
- Autologous stem cell mobilization and collection: Patients receive filgrastim to mobilize stem cells, which are then collected.
- Conditioning regimen: Patients receive bortezomib intravenously on days -6, -3, 1, and 4 and oral high-dose melphalan on days -2 and -1.
- Stem cell transplantation: Patients undergo autologous stem cell transplantation on day 0.
After completion of study therapy, patients are followed every 6 months for 1 year and annually thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Stem Cell Transplant with Bortezomib and Melphalan | Experimental | Biological: filgrastim, Drug: bortezomib, Drug: melphalan, Procedure: Stem Cell InfusionMobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Histologically confirmed primary systemic amyloidosis based on the following criteria:
- Southwest Oncology Group performance status 0-1
- Fertile patients must use effective contraception
- Left ventricular ejection fraction ≥ 45% by Echocardiogram within the past 60 days
- diffusion capacity of lung for carbon monoxide ≥ 50%
Key exclusion criteria (11 listed)
- No senile, secondary, localized, dialysis-related, or familial amyloidosis
- No overt multiple myeloma (> 30% of bone marrow plasmacytosis, extensive [> 2] lytic lesions, or hypercalcemia)
- Not pregnant or nursing
- No myocardial infarction within the past 6 months, congestive heart failure, or arrhythmia refractory to therapy
- No prior malignancy except for 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 |
|---|---|---|
| filgrastimAlso: Growth-Colony Stimulating Factor, neupogen16 mcg/kg daily beginning 3 days before stem cell collection through day before final stem cell collection | Biological | Stem Cell Transplant with Bortezomib and Melphalan |
| bortezomibAlso: velcade1.0 mg/m2/dose D -6, D-3, D +1, D + 4 | Drug | Stem Cell Transplant with Bortezomib and Melphalan |
| melphalanAlso: alkeran100 mg/m2/dose D -2, D -1 | Drug | Stem Cell Transplant with Bortezomib and Melphalan |
| Stem Cell Infusioninfusion of previously collected autologous stem cells | Procedure or surgery | Stem Cell Transplant with Bortezomib and Melphalan |
Primary outcome
Number of Participants With Hematologic Response
Time frame: one year
complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy <5% plasma cells with no clonal predominance of kappa or lambda isotype. Partial response: any one of the following 1. For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. 2. For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more. 3. For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).
3 secondary outcomes
Number of Participants Surviving at 100 Days From Transplant
Time frame: 100 Days from transplant date
Number of Participants Surviving at 1 Year
Time frame: one year from transplant
Number of Participants Surviving at 2 Years
Time frame: 2 years from transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Vaishali Sanchorawala, MD principal investigator
Boston Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing filgrastim, bortezomib and melphalan in people with Multiple Myeloma, run by Boston Medical Center at 1 site in United States.
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 15 inclusion and 11 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 6 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Yes. Results are posted in the registry (first posted 6 Feb 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Hematologic Response (time frame: one year). 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 6 Feb 2017.