Multiple Myeloma and Plasma Cell NeoplasmPhase 2NCT00002810
High-Dose Melphalan Followed by Peripheral Stem Cell Transplant in Treating Patients With Amyloidosis
A Phase 2 study testing filgrastim, melphalan and bone marrow ablation with stem cell support in people with Multiple Myeloma and Plasma Cell Neoplasm, run by Temple University at 1 site in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that… Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Multiple Myeloma and Plasma Cell Neoplasm
- What is being tested?
- filgrastim, melphalan and bone marrow ablation with stem cell support
- Type of treatment
- Surgery or procedure
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- May 1996to May 2006
- 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
DISEASE CHARACTERISTICS:
- Primary amyloidosis diagnosed by appropriate amyloid stains or electromicroscopy of abdominal fat, bone marrow, or other target tissues
- Pathology reviewed by Temple University
- Amyloidosis secondary to any stage of multiple myeloma allowed provided plasma cell concentration in bone marrow is less than 15%
- No amyloidosis secondary to rheumatoid arthritis or chronic infection
- No familial amyloidosis
PATIENT CHARACTERISTICS:
Age:
- 16 to 65
Performance status:
- Karnofsky 80-100%
Hematopoietic:
- Not specified
Hepatic:
- Liver function tests less than twice normal
- No active liver disease
Renal:
- Creatinine clearance greater than 50 mL/min
- Nephrotic syndrome allowed
Cardiovascular:
- Cardiac evaluation required in patients with left ventricular ejection fraction less than 45% by echocardiogram or MUGA
- No poorly controlled hypertension
Pulmonary:
- FEV_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required
- No chronic obstructive pulmonary disease
Other:
- No history of serious coagulopathy, hemorrhage, or bleeding
- No active infection
- No other serious comorbid disease (e.g., poorly controlled diabetes)
- No pregnant women
- Adequate contraception required of fertile women
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Not specified
Chemotherapy:
- More than 12 monthly cycles of prior alkylating agent chemotherapy discouraged
Endocrine therapy:
- Corticosteroids discontinued at least 6 weeks prior to transplantation
Radiotherapy:
- No prior radiotherapy
Surgery:
- Not specified
Trial locations and contacts
1 site in 1 country.
United States1 site
Fox Chase-Temple Cancer Center CCOP Research Base
Philadelphia, Pennsylvania, 19111-2442
About this study
Official title: Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.
PURPOSE: This phase II trial is studying how well giving high-dose melphalan together with peripheral stem cell transplant works in treating patients with primary amyloidosis or amyloidosis associated with multiple myeloma.
Read the detailed description
OBJECTIVES:
- Assess overall and progression-free survival following high-dose melphalan and autologous peripheral blood stem cell transplantation in patients with primary amyloidosis.
- Evaluate the toxic effects associated with this treatment regimen.
- Evaluate the function of involved organs, especially the heart, lungs, and nervous system, before and after treatment with this regimen.
OUTLINE: Peripheral blood stem cells (PBSC) are mobilized with granulocyte colony-stimulating factor (G-CSF) for 5 days and then collected by leukapheresis. Patients receive high-dose melphalan on 2 consecutive days, followed by 1 day of rest, then by PBSC transplantation. G-CSF is given from 1 day after transplantation until the neutrophil count is greater than 1,500 for 3 consecutive days.
Patients are followed at 100 days and 1 year post-transplant.
PROJECTED ACCRUAL: A very small number of patients are expected to be accrued over 5-10 years.
- Being tested
- filgrastim, melphalan, bone marrow ablation with stem cell support, peripheral blood stem cell transplantation
- Keywords
- refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, primary systemic amyloidosis
- Medical subject headings
- Multiple Myeloma, Neoplasms, Plasma Cell, Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
melphalanDrug
bone marrow ablation with stem cell supportProcedure or surgery
peripheral blood stem cell transplantationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Time to clinical progression of amyloid symptoms
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startMay 1996
- Primary completionMay 2006
- Study completionMay 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple Myeloma and Plasma Cell NeoplasmStudies of the same condition, open and under way.
- 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
- Single group
- Masking
- None (open label)
Sponsor and investigators
- Lead sponsor
- Temple University
- Sponsor type
- University, hospital or other
- Investigators
- Kenneth F. Mangan, MD, FACP, Study chair, Fox Chase Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00002810
- Sponsor's protocol ID
- CDR0000064938
- Other IDs
- TUHSC-2797, NCI-V96-0951
- Status last verified
- Sep 2010
- First submitted
- 1 Nov 1999
- Last updated
- 1 Oct 2010
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)
- ClinicalTrials.gov ID
- NCT00002810
- Acronym / sponsor ID
- CDR0000064938
- Keywords
- refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, primary systemic amyloidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- Temple UniversityUniversity, hospital or other
- Study start
- May 1996
- Primary completion
- May 2006 (actual)
- Study completion
- May 2006 (actual)
- First posted
- 27 Jan 2003 (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: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.
PURPOSE: This phase II trial is studying how well giving high-dose melphalan together with peripheral stem cell transplant works in treating patients with primary amyloidosis or amyloidosis associated with multiple myeloma.
Detailed description
OBJECTIVES:
- Assess overall and progression-free survival following high-dose melphalan and autologous peripheral blood stem cell transplantation in patients with primary amyloidosis.
- Evaluate the toxic effects associated with this treatment regimen.
- Evaluate the function of involved organs, especially the heart, lungs, and nervous system, before and after treatment with this regimen.
OUTLINE: Peripheral blood stem cells (PBSC) are mobilized with granulocyte colony-stimulating factor (G-CSF) for 5 days and then collected by leukapheresis. Patients receive high-dose melphalan on 2 consecutive days, followed by 1 day of rest, then by PBSC transplantation. G-CSF is given from 1 day after transplantation until the neutrophil count is greater than 1,500 for 3 consecutive days.
Patients are followed at 100 days and 1 year post-transplant.
PROJECTED ACCRUAL: A very small number of patients are expected to be accrued over 5-10 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| filgrastim | Biological | – |
| melphalan | Drug | – |
| bone marrow ablation with stem cell support | Procedure or surgery | – |
| peripheral blood stem cell transplantation | Procedure or surgery | – |
Primary outcome
Overall survival
Time to clinical progression of amyloid symptoms
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 1 Oct 2010.
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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.
Kenneth F. Mangan, MD, FACP study chair
Fox Chase Cancer 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 bone marrow ablation with stem cell support in people with Multiple Myeloma and Plasma Cell Neoplasm, run by Temple University 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?
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 1 Oct 2010. 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?
Overall survival. The study lists 2 primary outcomes in all. 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 1 Oct 2010.