Multiple MyelomaPhase 2NCT00075608
2nd Autologous Stem Cell Transplant in Patients With Persistent/Recurrent (AL) Amyloidosis
A Phase 2 study testing filgrastim, melphalan and autologous stem cell transplantation in people with Multiple Myeloma and Plasma Cell Neoplasm, run by Boston Medical Center 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 stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Multiple Myeloma, Plasma Cell Neoplasm
- What is being tested?
- filgrastim, melphalan and autologous stem cell transplantation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2001to Oct 2011
- 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 AL amyloidosis
- Persistent or recurrent disease after 1 course of prior high-dose chemotherapy
- Previously treated with autologous stem cell transplantation
- Significant initial improvement in organ function after prior high-dose melphalan, defined by at least 1 of the following:
- Complete hematologic remission (e.g., absence of monoclonal spike by immunofixation in serum and urine AND less then 5% plasma cells in bone marrow with no clonal predominance) OR partial hematologic response (e.g., any decrease in serum or urine monoclonal protein OR decrease in bone marrow plasmacytosis)
Show all 42Show fewer
- Greater than 50% reduction in proteinuria with preservation of creatinine clearance
- Greater than 50% reduction in alkaline phosphatase OR at least 2 cm decrease in liver size by physical exam
- Subjective neurologic improvement, as confirmed by neurologist
- Cardiac stabilization of disease confirmed by echocardiography defined as less than 2 mm increase in mean wall thickness and/or less than 20 g increase in left ventricular mass
- Improvement in performance status* NOTE: *This criteria alone does not constitute significant improvement in organ function
- Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg
- PRIOR CONCURRENT THERAPY:
- Biologic therapy
- See Disease Characteristics
- Chemotherapy
- See Disease Characteristics
- No chemotherapy after first transplantation
- Endocrine therapy
- Not specified
- Radiotherapy
- Not specified
- Surgery
- Not specified
- PATIENT CHARACTERISTICS:
- Age
- 18 to 65
- Performance status
- Southwest Oncology Group- 0-2
- Life expectancy
- More than 6 months
- Hematopoietic
- See Disease Characteristics
- Hepatic
- See Disease Characteristics
- Renal
- See Disease Characteristics
- Cardiovascular
- See Disease Characteristics
- Left ventricular ejection fraction ≥ 45% by multiple gated acquisition scan or echocardiogram
- Pulmonary
- diffusing capacity of lung for carbon monoxide ≥ 50%
Who cannot join
- No myelodysplastic syndromes
- No abnormal bone marrow cytogenetics
- Other
- Not pregnant or nursing
- Fertile patients must use effective contraception
- Acceptable toxicity from first transplantation, confirmed by the transplant team
- HIV negative
- No other concurrent malignancy except treated skin cancer
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 Second Autologous Transplantation in AL Amyloidosis
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 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 autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.
Read the detailed description
OBJECTIVES:
- Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.
- Determine the response rate and durability of response in patients treated with this regimen.
- Determine immune reconstitution in patients treated with this regimen.
OUTLINE:
- Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily beginning before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection.
- Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2.
- Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.
Patients are followed at 6 months, 1 year, and then annually thereafter.
PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.
- Conditions
- Multiple Myeloma, Plasma Cell Neoplasm
- Being tested
- filgrastim, melphalan, autologous stem cell transplantation, stem cell infusion
- Keywords
- 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
Also called G-CSF
16mcg/kg IV daily beginning three days prior to stem cell collection through last day of stem cell collection
melphalanDrug
Also called alkeran
140-200 mcg/kg IV over two days
autologous stem cell transplantationProcedure or surgery
infusion of previously collected stem cells on Day 0
stem cell infusionProcedure or surgery
infusion of previously collected stem cells on Day 0
| Study group | Role | What they receive |
|---|---|---|
| 2nd Stem Cell Transplant | Experimental | Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion Receives: Biological: filgrastim; Drug: melphalan; Procedure: autologous stem cell transplantation; Procedure: stem cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility and Tolerability
Feasibility and tolerability will be evaluated based on participants completing second transplant with tolerable adverse events
Measured: 3 months after treatment and annually
Response and Durability of Response
Response and durability of response will be based on hematologic Complete Response or Partial Response and date of relapse or death
Measured: 3 months after treatment and annually
Evaluate Immune Reconstitution
Evaluate immune reconstitution based on time to engraftment
Measured: 3 months after treatment and annually
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 2001
- Primary completionOct 2011
- Study completionOct 2011
Why it stopped: poor accrual
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
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Karen Quillen, Boston Medical Center
- Investigators
- Karen Quillen, MD, Principal investigator, Boston Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00075608
- Sponsor's protocol ID
- CDR0000347379
- Other IDs
- H-22603
- Status last verified
- Dec 2016
- First submitted
- 9 Jan 2004
- Last updated
- 27 Jan 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 27 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Second Autologous Transplantation in AL Amyloidosis
- ClinicalTrials.gov ID
- NCT00075608
- Acronym / sponsor ID
- CDR0000347379
- Conditions
- Multiple Myeloma, Plasma Cell Neoplasm
- Keywords
- primary systemic amyloidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: poor accrual
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Boston Medical CenterUniversity, hospital or other
- Study start
- Aug 2001
- Primary completion
- Oct 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 12 Jan 2004 (estimated)
- Results
- Posted 27 Jan 2017
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 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 autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.
Detailed description
OBJECTIVES:
- Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.
- Determine the response rate and durability of response in patients treated with this regimen.
- Determine immune reconstitution in patients treated with this regimen.
OUTLINE:
- Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily beginning before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection.
- Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2.
- Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.
Patients are followed at 6 months, 1 year, and then annually thereafter.
PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 2nd Stem Cell Transplant | Experimental | Biological: filgrastim, Drug: melphalan, Procedure: autologous stem cell transplantation, Procedure: stem cell infusionMobilization with filgrastim autologous stem cell transplantation with melphalan conditioning 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 (42 listed)
- Histologically confirmed AL amyloidosis
- Previously treated with autologous stem cell transplantation
- Significant initial improvement in organ function after prior high-dose melphalan, defined by at least 1 of the following:
- Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg
- Biologic therapy
Key exclusion criteria (8 listed)
- No myelodysplastic syndromes
- No abnormal bone marrow cytogenetics
- Other
- Not pregnant or nursing
- Fertile patients must use effective contraception
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: G-CSF16mcg/kg IV daily beginning three days prior to stem cell collection through last day of stem cell collection | Biological | 2nd Stem Cell Transplant |
| melphalanAlso: alkeran140-200 mcg/kg IV over two days | Drug | 2nd Stem Cell Transplant |
| autologous stem cell transplantationinfusion of previously collected stem cells on Day 0 | Procedure or surgery | 2nd Stem Cell Transplant |
| stem cell infusioninfusion of previously collected stem cells on Day 0 | Procedure or surgery | 2nd Stem Cell Transplant |
Primary outcome
Feasibility and Tolerability
Time frame: 3 months after treatment and annually
Feasibility and tolerability will be evaluated based on participants completing second transplant with tolerable adverse events
Response and Durability of Response
Time frame: 3 months after treatment and annually
Response and durability of response will be based on hematologic Complete Response or Partial Response and date of relapse or death
Evaluate Immune Reconstitution
Time frame: 3 months after treatment and annually
Evaluate immune reconstitution based on time to engraftment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Multiple Myeloma
Not yet recruitingPhase 2
Study of YKST02 in Adults With Relapsed or Refractory Multiple Myeloma
Relapsed or Refractory Multiple Myeloma (RRMM)
RecruitingPhase 2
Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)
Multiple Myeloma
Active, not recruitingPhase 3
A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
Multiple Myeloma
Active, not recruitingPhase 1/2
A Window of Opportunity Trial to Learn if Linvoseltamab is Safe and Well Tolerated, and How Well it Works in Adult Participants With Recently Diagnosed Multiple Myeloma Who Have Not Already Received Treatment
Multiple Myeloma
Active, not recruitingPhase 2
Testing AZD5363 as a Potential Targeted Treatment in Cancers With AKT Genetic Changes (MATCH-Subprotocol Y)
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
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.
Karen Quillen, 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 stem cell transplantation in people with Multiple Myeloma and Plasma Cell Neoplasm, 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: stopped early, as of the sponsor's last update on 27 Jan 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 27 Jan 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?
Feasibility and Tolerability (time frame: 3 months after treatment and annually). The study lists 3 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 27 Jan 2017.