Multiple MyelomaPhase 2NCT00075621
Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis
A Phase 2 study testing filgrastim, melphalan and autologous peripheral blood stem cell transplantation in people with Multiple Myeloma, run by Boston Medical Center at 1 site in United States.
Trial summary
RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis. PURPOSE: This phase II trial is studying how well tandem (two) autologous stem cell transplantation works in treating patients with primary systemic (AL) amyloidosis. 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, melphalan and autologous peripheral blood stem cell transplantation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 62 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2000to 4 Sep 2020
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- DISEASE CHARACTERISTICS:
- Histologically confirmed AL amyloidosis, meeting 1 of the following criteria:
- Plasma cell dyscrasia, evidenced by 1 of the following:
- Monoclonal protein in the serum or urine by immunofixation electrophoresis
- Plasmacytosis of the bone marrow with monoclonal staining for kappa or lambda light chain isotype
- Macroglossia with at least 1 other site having biopsy proven amyloidosis and absence of a mutant transthyretin is ruled out
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- PATIENT CHARACTERISTICS:
- Age
- 18 to 65
- Performance status
- SWOG 0-2
- Life expectancy
- At least 1 year
- Hematopoietic
- Not specified
- Hepatic
- Not specified
- Renal
- Not specified
- Cardiovascular
- LVEF ≥ 45% by MUGA or echocardiogram
- Pulmonary
- DLCO ≥ 50%
- Other
- Not pregnant or nursing
- Fertile patients must use effective contraception
- Able to tolerate 2 courses of high-dose therapy
- HIV negative
- PRIOR CONCURRENT THERAPY:
- Biologic therapy
- Not specified
- Chemotherapy
- Prior alkylating agent chemotherapy allowed provided there is no morphologic or cytogenetic evidence of myelodysplastic syndromes
- Prior total cumulative oral melphalan dose < 300 mg
- Endocrine therapy
- Not specified
- Radiotherapy
- Not specified
- Surgery
- Not specified
- Other
- At least 4 weeks since prior cytotoxic therapy and recovered
Who cannot join
- No senile, secondary, localized, dialysis-related, or familial amyloidosis
- No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive [more than 2] lytic lesions, hypercalcemia)
- Cardiovascular
- No myocardial infarction within the past 6 months
- No congestive heart failure
- No arrhythmia refractory to therapy
- No evidence of symptomatic transient ischemic attacks or strokes
- No other malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I or II cancer currently in complete remission
Trial locations and contacts
1 site in 1 country.
United States1 site
Cancer Research Center at Boston Medical Center
Boston, Massachusetts, 02118
About this study
Official title: A Phase II Trial of Tandem Transplantation in AL Amyloidosis
RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis.
PURPOSE: This phase II trial is studying how well tandem (two) autologous stem cell transplantation works in treating patients with primary systemic (AL) amyloidosis.
Read the detailed description
OBJECTIVES:
- Determine the tolerability of tandem autologous stem cell transplantation in patients with AL amyloidosis.
- Determine whether this regimen can convert a hematologic non-complete response (CR) to CR in these patients.
- Determine the overall survival of patients treated with this regimen.
OUTLINE:
- First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily beginning 3 days before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection. Patients may undergo bone marrow harvest if an inadequate number of peripheral blood stem cells are collected.
Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo autologous stem cell transplantation (ASCT) on day 0.
- Second transplantation: Within 6-12 months after the first ASCT, patients not achieving a complete response receive high-dose melphalan IV over 20 minutes on days -3 and -2 and a second ASCT on day 0.
Treatment continues in the absence of unacceptable toxicity.
Patients are followed at 3 and 6 months, 1 year, and then annually thereafter.
PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 years.
- Conditions
- Multiple Myeloma
- Being tested
- filgrastim, melphalan, autologous peripheral blood stem cell transplantation
- 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.
filgrastimDrug
16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
melphalanDrug
Also called alkeran
200 mg/kg over 2 days
autologous peripheral blood stem cell transplantationProcedure or surgery
autologous peripheral blood stem cell transplantation
| Study group | Role | What they receive |
|---|---|---|
| tandem transplant | Other | Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
Drug: melphalan 200 mg/kg over 2 days
Procedure/Surgery: autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation Receives: Drug: filgrastim; Drug: melphalan; Procedure: autologous peripheral blood stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
safety
Measured: 100 days, 6 months, and annual
Secondary outcomes (1)
Efficacy
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2004 (estimated)
- Study startAug 2000
- Primary completionJun 2005
- Study completion4 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 62 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Vaishali Sanchorawala, MD, Principal investigator, Boston Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00075621
- Sponsor's protocol ID
- H-23645
- Status last verified
- Sep 2020
- First submitted
- 9 Jan 2004
- Last updated
- 17 Sep 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Tandem Transplantation in AL Amyloidosis
- ClinicalTrials.gov ID
- NCT00075621
- Acronym / sponsor ID
- H-23645
- Conditions
- Multiple Myeloma
- Keywords
- primary systemic amyloidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Boston Medical CenterUniversity, hospital or other
- Study start
- Aug 2000 (actual)
- Primary completion
- Jun 2005 (actual)
- Study completion
- 4 Sep 2020 (actual)
- First posted
- 12 Jan 2004 (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.
RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis.
PURPOSE: This phase II trial is studying how well tandem (two) autologous stem cell transplantation works in treating patients with primary systemic (AL) amyloidosis.
Detailed description
OBJECTIVES:
- Determine the tolerability of tandem autologous stem cell transplantation in patients with AL amyloidosis.
- Determine whether this regimen can convert a hematologic non-complete response (CR) to CR in these patients.
- Determine the overall survival of patients treated with this regimen.
OUTLINE:
- First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily beginning 3 days before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection. Patients may undergo bone marrow harvest if an inadequate number of peripheral blood stem cells are collected.
Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo autologous stem cell transplantation (ASCT) on day 0.
- Second transplantation: Within 6-12 months after the first ASCT, patients not achieving a complete response receive high-dose melphalan IV over 20 minutes on days -3 and -2 and a second ASCT on day 0.
Treatment continues in the absence of unacceptable toxicity.
Patients are followed at 3 and 6 months, 1 year, and then annually thereafter.
PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| tandem transplant | Other | Drug: filgrastim, Drug: melphalan, Procedure: autologous peripheral blood stem cell transplantationDrug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection Drug: melphalan 200 mg/kg over 2 days Procedure/Surgery: autologous peripheral blood stem cell transplantation autologous peripheral blood stem cell transplantation |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (42 listed)
- Histologically confirmed AL amyloidosis, meeting 1 of the following criteria:
- Age
- 18 to 65
- Performance status
- SWOG 0-2
Key exclusion criteria (8 listed)
- No senile, secondary, localized, dialysis-related, or familial amyloidosis
- No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive [more than 2] lytic lesions, hypercalcemia)
- Cardiovascular
- No myocardial infarction within the past 6 months
- No congestive heart failure
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 |
|---|---|---|
| filgrastim16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection | Drug | tandem transplant |
| melphalanAlso: alkeran200 mg/kg over 2 days | Drug | tandem transplant |
| autologous peripheral blood stem cell transplantationautologous peripheral blood stem cell transplantation | Procedure or surgery | tandem transplant |
Primary outcome
safety
Time frame: 100 days, 6 months, and annual
1 secondary outcome
Efficacy
Time frame: one year
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 17 Sep 2020.
- 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, melphalan and autologous peripheral blood stem cell transplantation 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 42 inclusion and 8 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 17 Sep 2020. 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?
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?
safety (time frame: 100 days, 6 months, and annual). 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 17 Sep 2020.