Multiple MyelomaPhase 2BuMelMCRN001NCT01702831
Busulfan & Melphalan Conditioning for Autologous Stem Cell Transplant (ASCT) and Lenalidomide Maintenance
A Phase 2 study testing Busulfan, Melphalan and Lenalidomide in people with Multiple Myeloma, run by University Health Network, Toronto at 10 sites in Canada.
Trial summary
A number of strategies have been proposed to improve the outcome of ASCT. The three main strategies are to incorporate novel agents into the induction regimen, using maintenance therapy following ASCT and the final strategy is to enhance conditioning regimens. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- Busulfan, Melphalan and Lenalidomide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 10 sitesCanada
- Study dates
- 1 Oct 2013to 31 Jul 2022
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 to 75 years, inclusive.
- Study participants must have a diagnosis of symptomatic multiple myeloma requiring systemic therapy and are eligible for the planned ASCT.
- Untreated bone marrow sample was shipped to Princess Margaret Hospital for MRD assay.
- Must have been treated with a velcade-based induction regimen. No limit to the number of cycles of induction.
- Study participants in whom the minimum stem cell dose of 2.0 x 106 cluster of differentiation (CD)34+ cells/kg has been collected.
- Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2.
- Negative beta-human chorionic gonadotropin (β-HCG) pregnancy test in all females of child-bearing potential (FOCBP).
- Ability to provide written informed consent prior to initiation of any study-related procedures, and ability, in the opinion of the Principal Investigator, to comply with all requirements of the study.
Who cannot join
- Myeloma progression at any time since starting initial induction therapy for multiple myeloma. Changes to or additions to the existing induction therapy are allowed as long as disease progression has not been confirmed.
- Prior treatment history of ASCT for any medical reason.
- Prior treatment history of high-dose chemotherapy with stem cell rescue for any medical reason, not limited to myeloma treatment.
- Prior treatment with busulfan or gemtuzumab ozogamicin for any reason.
- Systemic amyloidosis.
- Left ventricular ejection fraction (LVEF) < 45% as measured by either multi-gated acquisition scan (MUGA) or echocardiogram (ECHO) performed within 75 days prior to day of busulfan dose. If cyclophosphamide was used for stem cell harvest, an ECHO or MUGA must be done after the stem cell collection and prior to enrollment to confirm adequate cardiac function.
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- Uncontrolled arrhythmia or symptomatic cardiac disease at the time of screening.
- Symptomatic pulmonary disease, based on Forced Expiratory Volume in 1 Second (FEV1), Forced Vital Capacity (FVC) or Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) < 50% of predicted (corrected for hemoglobin) measured within 75 days prior to day of busulfan dose.
- Aspartate transaminase (AST)/alanine transaminase (ALT) ≥ 3 x the upper limit of normal (ULN).
- History of elevated total serum bilirubin >2 mg/dL that had been caused by previous chemotherapy at any point, or total bilirubin > 2.0 mg/dL at the time of screening with the exception of Gilbert's disease.
- Hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time as International Normalized Ratio (INR) ≥ 2.0 at the time of screening.
- Any previous history of fulminant liver failure, cirrhosis, alcoholic hepatitis, esophageal varices, hepatic encephalopathy, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, and symptomatic biliary disease.
- Prior total body irradiation therapy, or radiation therapy directly applied to the liver.
- Patients with a known history of hepatitis B or hepatitis C should be on appropriate anti-viral therapy. Even so, these cases must be discussed with the sponsor and approval obtained prior to screening.
- Known history of or current HIV infection, or active hepatitis B or c infection or any uncontrolled active infection of any kind at the time busulfan administration.
- Serum creatinine >177 umol/L at the time of screening.
- Women who are pregnant or lactating.
- Current or history of drug and/or alcohol abuse.
- Use of other investigational therapies within 30 days of enrollment in this study. Use of investigational therapies, other than the ones given as part of this protocol therapy, is not allowed during the study participation.
- Clinically significant abnormality in medical history or upon examination that might interfere with the outcomes of the study in the opinion of the investigator.
- Any patient, who in the opinion of the investigator, should not participate in this study.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
Canada10 sites
Cross Cancer Institute 11560 University Ave
Edmonton, Alberta, T6G-1Z2
Queen Elizabeth II Health Sciences Centre.
Halifax, Nova Scotia, B3H 2Y9
London Regional Cancer Program 790 Commissioners Road East
London, Ontario, N6A 4L6
Hôpital Maisonneuve-Rosemont, 5415, boul. de l'Assomption
Montreal, Quebec, H1T 2M4
Royal Victoria Hospital, MUHC Glen Site, Cedars Cancer Centre
Montreal, Quebec, H4A 3J1
The Ottawa Hospital
Ottawa, Ontario, K1H 8L6
Saint John Regional Hospital, 5DN Research Department, 400 University Ave
Saint John, New Brunswick, E2L 4L2
Saskatoon Cancer Centre 20 Campus Drive
Saskatoon, Saskatchewan, S7N 4H4
Princess Margaret Cancer Centre
Toronto, Ontario, M5G 2M9
Vancouver General Hospital, Centennial Pavilion, 6th Floor
Vancouver, British Columbia, V5Z 1M9
About this study
Official title: A Phase II Study of Busulfan & Melphalan as Conditioning Regimen for ASCT in Patients Who Received Bortezomib Based Induction for Newly Diagnosed Multiple Myeloma Followed by Lenalidomide Maintenance Until Progression.
A number of strategies have been proposed to improve the outcome of ASCT. The three main strategies are to incorporate novel agents into the induction regimen, using maintenance therapy following ASCT and the final strategy is to enhance conditioning regimens.
Investigators would like to explore all these three strategies in this study: Investigators propose to take patients who have had standard novel agent (bortezomib) based induction regimens into this study and then use a dose-adjusted combination of busulfan and melphalan as conditioning regimen and finally Investigators would like to incorporate lenalidomide maintenance post ASCT until disease progression.
Read the detailed description
STUDY RATIONALE AND PURPOSE:
A number of strategies have been proposed to improve the outcome of ASCT. The three main strategies are to incorporate novel agents into the induction regimen, using maintenance therapy following ASCT and the final strategy is to enhance conditioning regimens.
Investigators would like to explore all these three strategies in this study: Investigators propose to take patients who have had standard novel agent (bortezomib) based induction regimens into this study and then use a dose-adjusted combination of busulfan and melphalan as conditioning regimen and finally Investigators would like to incorporate lenalidomide maintenance post ASCT until disease progression.
The conventional immunological markers used to define myeloma disease status have also been a subject of debate recently with some reports suggesting that more accurate disease assessment tools are needed to better decide on management of this disease. One of the most promising assays which is increasingly accepted as a more sensitive indicator of myeloma disease status is the Minimal Residual Disease (MRD) analysis. Therefore, Investigators plan to use MRD analysis as a disease assessment tool throughout this study and will correlate it with conventional myeloma disease assessment tools. Investigators would also like to incorporate a newly developed assay - Heavy lite (HevyLite) Chain assay and to explore the feasibility of using optional cell free DNA (cfDNA) to detect and monitor response assessments in multiple myeloma, - which will be done at the same time points as the other disease assessments thereby allowing us to explore the viability of these assays in clinical practice.
INTERVENTIONS:
Conditioning Regimen IV Busulfan 3.2mg/kg or equivalent pharmacokinetics directed dose once daily as a 3-hour infusion on days -5, -4 and -3 (option 1) or on days -6, -5, -4 (option 2).IV Melphalan 140mg/m2 once on day -2 (for option 1) or day -3 ( for option 2) Maintenance Regimen Oral Lenalidomide 10 mg once daily for 28 days of a 28 days cycle for first three cycles and then dose escalation to 15 mg daily if clinically appropriate to do so.
STUDY ENDPOINTS
Primary:
- MRD negativity at day 100 post ASCT
Secondary:
- To determine the pattern of MRD analysis during lenalidomide maintenance.
- To determine the response rate using conventional immunoglobulin-based markers at day 100 post ASCT and best response using lenalidomide maintenance.
- To determine the effectiveness of using the Heavy lite (HevyLite) Chain assay to assess anti-tumour response at day 100 post ASCT and during lenalidomide maintenance.
- To determine the toxicity of busulfan and melphalan when used as a high-dose conditioning therapy for ASCT.
- To determine the toxicity of lenalidomide maintenance post busulfan and melphalan conditioning ASCT.
- To determine the progression free survival (PFS) and overall survival (OS) of this program.
- To determine, through whole exome sequencing in individual Multiple Myeloma (MM) patients, the type and frequencies of somatic abnormalities (point mutations, indels, and copy number abnormalities) and their evolution overtime as the clinical disease progresses.
- To explore the feasibility of using optional cell free DNA (cfDNA) to detect and monitor response assessments in multiple myeloma.
- Conditions
- Multiple Myeloma
- Being tested
- Busulfan, Melphalan, Lenalidomide
- Keywords
- Newly Diagnosed multiple myeloma
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Busulfex
Once daily intravenous (IV) busulfan at a dose of 3.2 mg/kg or equivalent pharmacokinetics directed dose for three consecutive days (days -5 to -3), option 1 OR Once daily intravenous (IV) busulfan at a dose of 3.2mg/kg or equivalent pharmacokinetics directed dose for three consecutive days (days -6 to -4), option 2.
MelphalanDrug
Also called Alkeran
I.V. reduced dose of melphalan (140mg/m2) on day -2, followed by an autologous stem cell transplant on day 0 (day -1 will be a rest day) - this is referred to as "Option 1" dosing schema OR I.V. reduced dose of melphalan (140mg/m2) on day -3 followed by autologous stem cell transplant on day 0 (days -2 and -1 will be rest days). This is referred to as "Option 2"
LenalidomideDrug
Also called Revlimid
Oral lenalidomide 10mg per day (on all 28 days of a 28 day cycle) for the first three cycles and then escalated to 15 mg daily if clinically appropriate to do so. The lenalidomide maintenance will start on day 100 post ASCT and continue till disease progression.
| Study group | Role | What they receive |
|---|---|---|
| BuMel + lenalidomide Maintenance | Experimental | I.V. Busulfan + I.V. Melphalan for conditioning prior ASCT, followed by Lenalidomide maintenance at day 100 after ASCT. Receives: Drug: Busulfan; Drug: Melphalan; Drug: Lenalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
• Minimal Residual Disease (MRD) negativity at day 100 post ASCT
The conventional immunological markers used to define myeloma disease status have also been a subject of debate recently with some reports suggesting that more accurate disease assessment tools are needed to better decide on management of this disease. One of the most promising assays which is increasingly accepted as a more sensitive indicator of myeloma disease status is the Minimal Residual Disease (MRD) analysis. Therefore, Investigators plan to use MRD analysis as a disease assessment tool throughout this study and will correlate it with conventional myeloma disease assessment tools. Investigators would also like to incorporate a newly developed assay - Heavy lite (HevyLite) Chain assay - which will be done at the same time points as the other disease assessments thereby allowing us to explore the viability of this assay in clinical practice.
Measured: day 100 post ASCT
Secondary outcomes (8)
• To determine the pattern, positivity (in terms of percentages) or negativity of MRD analysis during lenalidomide maintenance.
The conventional immunological markers used to define myeloma disease status have also been a subject of debate recently with some reports suggesting that more accurate disease assessment tools are needed to better decide on management of this disease. One of the most promising assays which is increasingly accepted as a more sensitive indicator of myeloma disease status is the Minimal Residual Disease (MRD) analysis. Therefore, Investigators plan to use MRD negativity analysis as a disease assessment tool throughout this study and will correlate it with conventional myeloma disease assessment tools. Investigators would also like to incorporate a newly developed assay - Heavy lite (HevyLite) Chain assay - which will be done at the same time points as the other disease assessments thereby allowing us to explore the viability of this assay in clinical practice.
Measured: After ASCT at day 100, during maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the response rate using conventional immunoglobulin and monoclonal protein-based markers at day 100 post ASCT and best response using lenalidomide maintenance.
The response rate (Complete Response, Very Good Partial Response, Partial Response, Minimal Response and Stable Disease) will be assessed by using the European Group for Blood and Marrow Transplantation modified response criteria,with conventional immunoglobulin and monoclonal protein-based markers at day 100 post ASCT and best response using lenalidomide maintenance.
Measured: After ASCT at day 100, during maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the effectiveness of using the HevyLite Chain assay to assess anti-tumour response at day 100 post ASCT and during lenalidomide maintenance
Immunoglobulin (Ig) A kappa, Immunoglobulin (Ig)A lambda, Immunoglobulin (Ig) A kappa/Immunoglobulin (Ig) A lambda ratio; Immunoglobulin (Ig) G Kappa, Immunoglobulin (Ig) G Lambda,Immunoglobulin (Ig) G kappa/Immunoglobulin (Ig) G lambda ratio; Immunoglobulin (Ig) M Kappa, Immunoglobulin (Ig) M Lambda and Immunoglobulin (Ig) M kappa/Immunoglobulin (Ig) M lambda ratio will be measured. These results will be correlated to standard Free Lite Chain assays performed at the same time points listed above.
Measured: After ASCT at day 100, during maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the toxicity of busulfan and melphalan when used as a high-dose conditioning therapy for ASCT.
Toxicity of busulfan and melphalan will be determined by tracking occurrence of adverse events, serious adverse events and immediately reportable events based on the definitions listed in the protocol section 18.1 The severity of the toxicity will be graded according to the NCI Common Toxicity Criteria for Adverse Effects(CTCAE) version 3.0
Measured: During conditioning therapy for ASCT. For schema option 1 this will be assessed at days -6, -5, -4, -3 and -2 prior ASCT. For schema option 2 this will be assessed at days -7, -6, -5, -4 and -3 prior ASCT.
• To determine the toxicity of lenalidomide maintenance post busulfan and melphalan conditioning ASCT.
Toxicity of lenalidomide maintenance will be determined by tracking occurrence of adverse events, serious adverse events and immediately reportable events based on the definitions listed in the protocol section 18.1 The severity of the toxicity will be graded according to the NCI CTCAE version 3.0
Measured: After day 100 post ASCT, during the maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the progression free survival (PFS) and overall survival (OS) of this program.
These endpoints will be analyzed as time to event variables, which is defined as the time from transplant to death for OS and the time from transplant to the first occurrence of death or disease progression for PFS. The event free probabilities for these endpoints will be estimated by the product limit Kaplan Meier method. Subjects without events will be censored at the last followup for OS and at the last disease evaluation for PFS.
Measured: From randomization patients will be followed for PFS every 3 months for the first year after ASCT and then every 6 months until disease progression. After they will be followed every year for O/S until death.
• Identification of the type and frequencies of somatic abnormalities (point mutations, indels, and copy number abnormalities) and their evolution overtime.
Whole genome sequencing will be performed centrally at intervals described under the time frame.
Measured: After ASCT at day 100, during maint. therapy q 3 months at month 6 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• Assessment of using optional cell free DNA (cfDNA) in peripheral blood to monitor and correlate response assessments in multiple myeloma.
cell free DNA (cfDNA) will be performed centrally at intervals described under the time frame AND only for study participants for whom genomics whole exome sequencing) samples have been collected.
Measured: After ASCT at day 100, during maint. therapy q 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2012 (estimated)
- Study start1 Oct 2013
- Primary completion31 Jul 2022
- Study completion31 Jul 2022
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 University Health Network, TorontoThe 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
- 78 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Collaborators
- Princess Margaret Hospital, Canada, Otsuka Pharmaceutical Development & Commercialization, Inc., Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- Donna E Reece, MD, Principal investigator, University Health Network-Princess Margaret Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01702831
- Sponsor's protocol ID
- MCRN 001
- Acronym
- BuMelMCRN001
- Status last verified
- Jun 2026
- First submitted
- 29 Aug 2012
- Last updated
- 5 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Un-identified IPD, pertaining to Serious Adverse Events, will be submitted to the DSMB for review. DSMB letters will be provided to the participating sites. Un-identified IPD could also be shared with Sub-investigators conducting correlative studies for this trial; that will be Dr. Suzanne Trudel and Dr. Rodger Tiedemann.
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Busulfan & Melphalan as Conditioning Regimen for ASCT in Patients Who Received Bortezomib Based Induction for Newly Diagnosed Multiple Myeloma Followed by Lenalidomide Maintenance Until Progression.
- ClinicalTrials.gov ID
- NCT01702831
- Acronym / sponsor ID
- BuMelMCRN001 / MCRN 001
- Conditions
- Multiple Myeloma
- Keywords
- Newly Diagnosed multiple myeloma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 78 participants (actual)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Collaborators
- Princess Margaret Hospital, Canada, Otsuka Pharmaceutical Development & Commercialization, Inc., Celgene Corporation
- Study start
- 1 Oct 2013 (actual)
- Primary completion
- 31 Jul 2022 (actual)
- Study completion
- 31 Jul 2022 (actual)
- First posted
- 8 Oct 2012 (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.
A number of strategies have been proposed to improve the outcome of ASCT. The three main strategies are to incorporate novel agents into the induction regimen, using maintenance therapy following ASCT and the final strategy is to enhance conditioning regimens.
Investigators would like to explore all these three strategies in this study: Investigators propose to take patients who have had standard novel agent (bortezomib) based induction regimens into this study and then use a dose-adjusted combination of busulfan and melphalan as conditioning regimen and finally Investigators would like to incorporate lenalidomide maintenance post ASCT until disease progression.
Detailed description
STUDY RATIONALE AND PURPOSE:
A number of strategies have been proposed to improve the outcome of ASCT. The three main strategies are to incorporate novel agents into the induction regimen, using maintenance therapy following ASCT and the final strategy is to enhance conditioning regimens.
Investigators would like to explore all these three strategies in this study: Investigators propose to take patients who have had standard novel agent (bortezomib) based induction regimens into this study and then use a dose-adjusted combination of busulfan and melphalan as conditioning regimen and finally Investigators would like to incorporate lenalidomide maintenance post ASCT until disease progression.
The conventional immunological markers used to define myeloma disease status have also been a subject of debate recently with some reports suggesting that more accurate disease assessment tools are needed to better decide on management of this disease. One of the most promising assays which is increasingly accepted as a more sensitive indicator of myeloma disease status is the Minimal Residual Disease (MRD) analysis. Therefore, Investigators plan to use MRD analysis as a disease assessment tool throughout this study and will correlate it with conventional myeloma disease assessment tools. Investigators would also like to incorporate a newly developed assay - Heavy lite (HevyLite) Chain assay and to explore the feasibility of using optional cell free DNA (cfDNA) to detect and monitor response assessments in multiple myeloma, - which will be done at the same time points as the other disease assessments thereby allowing us to explore the viability of these assays in clinical practice.
INTERVENTIONS:
Conditioning Regimen IV Busulfan 3.2mg/kg or equivalent pharmacokinetics directed dose once daily as a 3-hour infusion on days -5, -4 and -3 (option 1) or on days -6, -5, -4 (option 2).IV Melphalan 140mg/m2 once on day -2 (for option 1) or day -3 ( for option 2) Maintenance Regimen Oral Lenalidomide 10 mg once daily for 28 days of a 28 days cycle for first three cycles and then dose escalation to 15 mg daily if clinically appropriate to do so.
STUDY ENDPOINTS
Primary:
- MRD negativity at day 100 post ASCT
Secondary:
- To determine the pattern of MRD analysis during lenalidomide maintenance.
- To determine the response rate using conventional immunoglobulin-based markers at day 100 post ASCT and best response using lenalidomide maintenance.
- To determine the effectiveness of using the Heavy lite (HevyLite) Chain assay to assess anti-tumour response at day 100 post ASCT and during lenalidomide maintenance.
- To determine the toxicity of busulfan and melphalan when used as a high-dose conditioning therapy for ASCT.
- To determine the toxicity of lenalidomide maintenance post busulfan and melphalan conditioning ASCT.
- To determine the progression free survival (PFS) and overall survival (OS) of this program.
- To determine, through whole exome sequencing in individual Multiple Myeloma (MM) patients, the type and frequencies of somatic abnormalities (point mutations, indels, and copy number abnormalities) and their evolution overtime as the clinical disease progresses.
- To explore the feasibility of using optional cell free DNA (cfDNA) to detect and monitor response assessments in multiple myeloma.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BuMel + lenalidomide Maintenance | Experimental | Drug: Busulfan, Drug: Melphalan, Drug: LenalidomideI.V. Busulfan + I.V. Melphalan for conditioning prior ASCT, followed by Lenalidomide maintenance at day 100 after ASCT. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age 18 to 75 years, inclusive.
- Study participants must have a diagnosis of symptomatic multiple myeloma requiring systemic therapy and are eligible for the planned ASCT.
- Untreated bone marrow sample was shipped to Princess Margaret Hospital for MRD assay.
- Must have been treated with a velcade-based induction regimen. No limit to the number of cycles of induction.
- Study participants in whom the minimum stem cell dose of 2.0 x 106 cluster of differentiation (CD)34+ cells/kg has been collected.
Key exclusion criteria (21 listed)
- Myeloma progression at any time since starting initial induction therapy for multiple myeloma. Changes to or additions to the existing induction therapy are allowed as long as disease progression has not been confirmed.
- Prior treatment history of ASCT for any medical reason.
- Prior treatment history of high-dose chemotherapy with stem cell rescue for any medical reason, not limited to myeloma treatment.
- Prior treatment with busulfan or gemtuzumab ozogamicin for any reason.
- Systemic amyloidosis.
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 |
|---|---|---|
| BusulfanAlso: BusulfexOnce daily intravenous (IV) busulfan at a dose of 3.2 mg/kg or equivalent pharmacokinetics directed dose for three consecutive days (days -5 to -3), option 1 OR Once daily intravenous (IV) busulfan at a dose of 3.2mg/kg or equivalent pharmacokinetics directed dose for three consecutive days (days -6 to -4), option 2. | Drug | BuMel + lenalidomide Maintenance |
| MelphalanAlso: AlkeranI.V. reduced dose of melphalan (140mg/m2) on day -2, followed by an autologous stem cell transplant on day 0 (day -1 will be a rest day) - this is referred to as "Option 1" dosing schema OR I.V. reduced dose of melphalan (140mg/m2) on day -3 followed by autologous stem cell transplant on day 0 (days -2 and -1 will be rest days). This is referred to as "Option 2" | Drug | BuMel + lenalidomide Maintenance |
| LenalidomideAlso: RevlimidOral lenalidomide 10mg per day (on all 28 days of a 28 day cycle) for the first three cycles and then escalated to 15 mg daily if clinically appropriate to do so. The lenalidomide maintenance will start on day 100 post ASCT and continue till disease progression. | Drug | BuMel + lenalidomide Maintenance |
Primary outcome
• Minimal Residual Disease (MRD) negativity at day 100 post ASCT
Time frame: day 100 post ASCT
The conventional immunological markers used to define myeloma disease status have also been a subject of debate recently with some reports suggesting that more accurate disease assessment tools are needed to better decide on management of this disease. One of the most promising assays which is increasingly accepted as a more sensitive indicator of myeloma disease status is the Minimal Residual Disease (MRD) analysis. Therefore, Investigators plan to use MRD analysis as a disease assessment tool throughout this study and will correlate it with conventional myeloma disease assessment tools. Investigators would also like to incorporate a newly developed assay - Heavy lite (HevyLite) Chain assay - which will be done at the same time points as the other disease assessments thereby allowing us to explore the viability of this assay in clinical practice.
8 secondary outcomes
• To determine the pattern, positivity (in terms of percentages) or negativity of MRD analysis during lenalidomide maintenance.
Time frame: After ASCT at day 100, during maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the response rate using conventional immunoglobulin and monoclonal protein-based markers at day 100 post ASCT and best response using lenalidomide maintenance.
Time frame: After ASCT at day 100, during maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the effectiveness of using the HevyLite Chain assay to assess anti-tumour response at day 100 post ASCT and during lenalidomide maintenance
Time frame: After ASCT at day 100, during maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the toxicity of busulfan and melphalan when used as a high-dose conditioning therapy for ASCT.
Time frame: During conditioning therapy for ASCT. For schema option 1 this will be assessed at days -6, -5, -4, -3 and -2 prior ASCT. For schema option 2 this will be assessed at days -7, -6, -5, -4 and -3 prior ASCT.
• To determine the toxicity of lenalidomide maintenance post busulfan and melphalan conditioning ASCT.
Time frame: After day 100 post ASCT, during the maintenance therapy every 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• To determine the progression free survival (PFS) and overall survival (OS) of this program.
Time frame: From randomization patients will be followed for PFS every 3 months for the first year after ASCT and then every 6 months until disease progression. After they will be followed every year for O/S until death.
• Identification of the type and frequencies of somatic abnormalities (point mutations, indels, and copy number abnormalities) and their evolution overtime.
Time frame: After ASCT at day 100, during maint. therapy q 3 months at month 6 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
• Assessment of using optional cell free DNA (cfDNA) in peripheral blood to monitor and correlate response assessments in multiple myeloma.
Time frame: After ASCT at day 100, during maint. therapy q 3 months at month 6, 9 and 12 after ASCT; and thereafter every 6 months until disease progression and at discontinuation.
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 5 Jun 2026.
- Individual participant dataSharing planned: Yes. Un-identified IPD, pertaining to Serious Adverse Events, will be submitted to the DSMB for review. DSMB letters will be provided to the participating sites. Un-identified IPD could also be shared with Sub-investigators conducting correlative studies for this trial; that will be Dr. Suzanne Trudel and Dr. Rodger Tiedemann.
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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.
Donna E Reece, MD principal investigator
University Health Network-Princess Margaret Hospital
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Busulfan, Melphalan and Lenalidomide in people with Multiple Myeloma, run by University Health Network, Toronto at 10 sites in Canada.
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 8 inclusion and 21 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 5 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Canada. 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?
• Minimal Residual Disease (MRD) negativity at day 100 post ASCT (time frame: day 100 post ASCT). 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 5 Jun 2026.