CompletedNCT01731886Updated Feb 5, 2020Results posted

Lenalidomide and Dexamethasone With/Without Stem Cell Transplant in Patients With Multiple Myeloma

A Phase 4 interventional study of Autologous peripheral blood stem cell transplant and Lenalidomide in Multiple Myeloma, sponsored by Columbia University. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-02-05.

Sponsored by Columbia University · Phase 4, Interventional, and Treatment

Phase: Phase 4
Study type: Interventional
Enrollment: 60
Allocation: Randomized
Ages: 18 Years and older
Sex: All

Study summary

The study is being done to compare the combination of lenalidomide and dexamethasone followed by autologous peripheral blood stem cell transplant (PBSCT) and lenalidomide and dexamethasone without PBSCT in patients with untreated multiple myeloma. This comparison will include how the subjects respond to each study treatment combination, and what side effects are caused by each combination.

Read the detailed description

Multiple myeloma is a malignant plasma cell proliferative disorder responsible for 11, 000 deaths each year in the United States. Approximately one third of myeloma patients develop hypercalcemia and about two thirds present with anemia. As the second most common hematologic malignancy, myeloma remains incurable. In the last forty years, options for therapy have included melphalan-prednisone, anthracyclines, and vinca alkaloids; however, relapse with those regimens continues to be inevitable with a median survival of 3 years.

Conditions studied

Multiple Myeloma

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Keywords

Stem cell transplant
plasma cell myeloma
multiple myeloma

In context

Multiple Myeloma

3,632 studies on the registry are indexed under Multiple Myeloma; 745 are open to participants now.

This study's enrollment of 60 is above the median of 41 across 2,907 interventional studies indexed under Multiple Myeloma.

Browse Multiple Myeloma studies →

Lead sponsor

Columbia University is the lead sponsor of 1,103 studies on the registry; 193 are open to participants now.

Of its 172 completed or terminated interventional studies of FDA-regulated products, 142 (83%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Histologically or cytologically confirmed Multiple Myeloma, Salmon-Durie Stage II or III or International Staging System II or III that has not been previously treated.
Bone marrow plasmacytosis with > or = 10% plasma cells, or sheets of plasma cells or a biopsy-proven plasmacytoma.
Measurable levels of monoclonal protein (M protein): 1 g/dL Immunoglobulin G (IgG) or .5 g/dL Immunoglobulin A (IgA) on serum protein electrophoresis or > 200 mg of monoclonal light chain on a 24 hour urine protein electrophoresis.
Age > or = 18 years.
Life expectancy of greater than 12 months.
Eastern Cooperative Oncology Group (ECOG) performance status \< or = 2 (Karnofsky > or = 60%).
Adequate organ and marrow function as defined below:
- Hgb > or = 9 g/dL
- Absolute Neutrophil Count > or = 1,500/ ml
- Platelets > or = 50,000/mm3
- Total Bilirubin \< or = 1.5 mg/dL
- Aspartate aminotransferase (AST)(SGOT) / alanine aminotransferase (ALT)(SGPT) \< or = 2.5 X upper limit of normal (ULN)
- Creatinine \< 2.0 mg/dL
- Creatinine Clearance > or = 50 ml/min
Registered into the mandatory Revlimid REMS® program, and be willing and able to comply with the requirements of the REMS® program.
Females of reproductive potential must adhere to the scheduled pregnancy testing as required in the Revlimid REMS® program.
Ability to understand and the willingness to sign a written informed consent document.
Subjects with a history of prior malignancy are eligible provided there is no active malignancy and a low expectation of recurrence within 6 months.
Must be willing and able to take prophylaxis with either aspirin at 81 mg/day or alternative prophylaxis with either low molecular weight heparin or warfarin as recommended.
Eligible for transplant with an age up to and including 75 years.
Subjects in Arm A who are refusing transplant can go onto Arm B and will be evaluated separately.
Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 50 milli-international units per millilitre (mIU/mL) within 10 - 14 days prior to and again within 24 hours of prescribing lenalidomide and must either commit to continued abstinence or 2 acceptable methods of birth control. FCBP must also agree to ongoing pregnancy testing. Males must agree to use a latex condom.

Exclusion criteria

Exclusion Criteria:

Have had chemotherapy or radiotherapy for multiple myeloma within 4 weeks of baseline.
Receiving any other investigational agents or therapy within 28 days of baseline.
Brain metastases.
Subjects who are pregnant or breast feeding.
History of previous deep vein thrombosis or pulmonary embolism must be on anticoagulation therapy with low molecular weight heparin or warfarin at therapeutic dosages (e.g. International Normalized Ratio (INR) 2-3).
If a subject is on full-dose anticoagulants, the following criteria should be met for enrollment:
- Must not have active bleeding or pathological conditions that carry high risk of bleeding (e.g. tumor involving major vessels, known varices).
- Must not have thrombocytopenia requiring transfusion.
- Must have a platelet count > 50,000.
- Must have stable INR between 2-3.
Smoldering myeloma or monoclonal gammopathy of undetermined significance.
Active, uncontrolled infection.
Active, uncontrolled seizure disorder (seizures in the last 6 months).
Concurrent use of other anti-cancer agents or treatments.
Positive for HIV or infectious hepatitis, type B or C.
Hypersensitivity to thalidomide.
Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk.

Study design

Phase: Phase 4
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: None (open label)
Enrollment: 60 participants (actual)

Study arms

Active comparator
Arm A
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by steam cell collection and autologous peripheral blood stem cell transplant. After 90 days, start the maintenance phase (lenalidomide days 1-21 every 28 days for two years or until your disease progresses).
Procedure: Autologous peripheral blood stem cell transplant · Drug: Lenalidomide · Drug: Dexamethasone · Procedure: Stem cell collection · Drug: Melphalan · Drug: G-CSF · Drug: Cyclophosphamide · Drug: Mesna
Active comparator
Arm B
Subjects will receive the new treatment that will be compared with the standard of care. Lenalidomide and dexamethasone for eight 28-day cycles. After four cycles your stem cells will be collected (stem cell collection). After an additional four cycles of lenalidomide (a total of 8 cycles), start the maintenance phase (lenalidomide days 1-21 every 28 days for two years or until your disease progresses).
Drug: Lenalidomide · Drug: Dexamethasone · Procedure: Stem cell collection · Drug: Cyclophosphamide · Drug: Mesna

Interventions

ProcedureAutologous peripheral blood stem cell transplant
Subjects deemed suitable by the principal investigator will undergo autologous peripheral blood stem cell transplantation on day 0.
DrugLenalidomide
Administered orally at a dose 25 mg daily on days 1-21 of each 28-day cycle.
Also known as: CC-5013, Revlimid
DrugDexamethasone
Administered orally at a dose of 40 mg daily on days 1, 8, 15, 22 of each cycle.
Also known as: Decadron, Hexadrol, Dexameth, Dexone, DXM
ProcedureStem cell collection
Peripheral stem cell collection will be performed at marrow recovery, usually when white blood cell (WBC) is \>2500 x 109 cells/liter; platelet count is \>20 x 103/mm3.
Also known as: Stem Cell Mobilization
DrugMelphalan
Subjects undergoing autologous peripheral blood stem cell transplant will receive melphalan 200 mg/m2 intravenously on days -2 and -1 or only on day -2.
Also known as: Evomela
DrugG-CSF
Subjects will receive G-CSF subcutaneously daily beginning on day 5 and until blood counts recover.
Also known as: Granulocyte-colony stimulating factor (CSF), Filgrastim
DrugCyclophosphamide
Subjects may receive up to the maximum recommended high-dose of cyclophosphamide at 4 gm/m2 intravenously.
Also known as: Cytophosphane
DrugMesna
Mesna will be provided with the cyclophosphamide.
Also known as: Mesnex

What researchers measure

Primary outcomes

Complete Response Rate
The primary objective of this study is to determine the complete response rate of lenalidomide and low-dose dexamethasone versus that of lenalidomide and low-dose dexamethasone followed by autologous peripheral blood stem cell transplant in patients with newly diagnosed multiple myeloma (will include unconfirmed complete response (CR), CR and stringent complete response (sCR)).
Time frame: 3 years

Secondary outcomes

Overall Survival Rate (OS)
To compare overall survival in subjects receiving autologous peripheral blood stem cell transplant after undergoing induction therapy with lenalidomide and dexamethasone versus in those receiving only lenalidomide and dexamethasone, followed by lenalidomide maintenance in both arms. Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis.
Time frame: 4 years
Overall Survival Rate (OS)
To compare overall survival in subjects receiving autologous peripheral blood stem cell transplant after undergoing induction therapy with lenalidomide and dexamethasone versus in those receiving only lenalidomide and dexamethasone, followed by lenalidomide maintenance in both arms. Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis.
Time frame: 2 years
Progression Free Survival (PFS)
PFS is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse.
Time frame: 4 years
Progression Free Survival (PFS)
PFS is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse.
Time frame: 2 years

Results

Posted Oct 15, 2019

Participant flow

Participant flow — Overall Study
Milestone	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
Started	31	29
Completed	29	28
Not completed	2	1
Withdrew: Death	1	0
Withdrew: Never started treatment	0	1
Withdrew: Non-compliant	1	0

Outcome measures

PrimaryComplete Response Rate

The primary objective of this study is to determine the complete response rate of lenalidomide and low-dose dexamethasone versus that of lenalidomide and low-dose dexamethasone followed by autologous peripheral blood stem cell transplant in patients with newly diagnosed multiple myeloma (will include unconfirmed complete response (CR), CR and stringent complete response (sCR)).

Time frame:: 3 years
Reported as:: Count of participants · Participants

Complete Response Rate
Participants	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
Complete Response Rate	7	7

SecondaryOverall Survival Rate (OS)

To compare overall survival in subjects receiving autologous peripheral blood stem cell transplant after undergoing induction therapy with lenalidomide and dexamethasone versus in those receiving only lenalidomide and dexamethasone, followed by lenalidomide maintenance in both arms. Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis.

Time frame:: 4 years
Reported as:: Number · percentage of participants

Overall Survival Rate (OS)
percentage of participants	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
Overall Survival Rate (OS)	79.8 (64.0 to 95.6)	78.9 (60.6 to 97.3)

SecondaryOverall Survival Rate (OS)

Time frame:: 2 years
Reported as:: Number · percentage of participants

Overall Survival Rate (OS)
percentage of participants	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
Overall Survival Rate (OS)	100 (95 to 100)	94.7 (85.4 to 99.9)

SecondaryProgression Free Survival (PFS)

PFS is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse.

Time frame:: 4 years
Reported as:: Number · percentage of participants

Progression Free Survival (PFS)
percentage of participants	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
Progression Free Survival (PFS)	36.0 (17.2 to 54.8)	31.6 (10.7 to 52.5)

SecondaryProgression Free Survival (PFS)

PFS is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse.

Time frame:: 2 years
Reported as:: Number · percentage of participants

Progression Free Survival (PFS)
percentage of participants	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
Progression Free Survival (PFS)	52.0 (32.4 to 71.6)	47.4 (24.9 to 69.8)

Adverse events

Collected over 3 years. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	14/31 (45.2%)	7/31 (22.6%)	21/31 (67.7%)
Arm B: Low-dose Dexamethasone	11/28 (39.3%)	7/28 (25%)	19/28 (67.9%)

Most frequent serious events

Showing 10 of 15

Most frequent serious events
Event	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
PresyncopeCardiac disorders	0/31	1/28
Bone PainMusculoskeletal and connective tissue disorders	1/31	1/28
Infection with Grade 3 or 4 NeutrophilsInfections and infestations	1/31	1/28
InfectionInfections and infestations	0/31	1/28
Myelodysplastic SyndromeBlood and lymphatic system disorders	0/31	1/28
Pain of skinSkin and subcutaneous tissue disorders	0/31	1/28
Abdominal PainGeneral disorders	0/31	1/28
Back PainGeneral disorders	0/31	1/28
Thromboembolic EventVascular disorders	0/31	1/28
Febrile NeutropeniaInfections and infestations	1/31	0/28

Most frequent other events

Showing 10 of 25

Most frequent other events
Event	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone
Decreased Neutrophil CountBlood and lymphatic system disorders	8/31	4/28
Abnormal Neutrophils/Granulocytes (ANC/AGC)Blood and lymphatic system disorders	5/31	7/28
Decreased WBC CountBlood and lymphatic system disorders	6/31	3/28
DiarrheaGastrointestinal disorders	3/31	5/28
InsomniaGeneral disorders	3/31	5/28
InfectionInfections and infestations	3/31	5/28
Decreased Platelet CountBlood and lymphatic system disorders	5/31	1/28
AnemiaBlood and lymphatic system disorders	2/31	4/28
Abnormal Platelet CountBlood and lymphatic system disorders	2/31	4/28
CoughGeneral disorders	4/31	3/28

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone	Total
<=18 years	0	0	0
Between 18 and 65 years	21	18	39
>=65 years	10	11	21

Sex: Female, Male
Sex: Female, Male(Participants)	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone	Total
Female	15	12	27
Male	16	17	33

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone	Total
American Indian or Alaska Native	0	0	0
Asian	0	0	0
Native Hawaiian or Other Pacific Islander	0	0	0
Black or African American	4	6	10
White	23	20	43
More than one race	1	0	1
Unknown or Not Reported	3	3	6

ISS Stage
ISS Stage(Participants)	Arm A: Low-dose Dexamethasone + Stem Cell Transplantation	Arm B: Low-dose Dexamethasone	Total
Stage 1(B2M <3.5 mg/L and serum albumin ≥3.5 g/dL)	11	11	22
Stage 2(Neither stage I nor stage III)	14	14	28
Stage 3(B2M ≥5.5 mg/L)	6	4	10

Study locations

1 site

Columbia University
New York, New York 10032, United States

References and documents

Study documents

Protocol and statistical analysis plan · Jun 19, 2015

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Individual participant data (IPD) will be coded and shared with the University of Pittsburgh at the end of the trial.

Supporting information: Study protocol, Sap

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Feb 5, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT01731886
Lead sponsor: Columbia University
Responsible party: Suzanne Lentzsch, MD (Associate Clinical Professor of Clinical Medicine, Columbia University) — Principal investigator
First posted: Nov 22, 2012
Start date: Sep 2012
Primary completion: Apr 11, 2017
Completion: Apr 11, 2017
Results posted: Oct 15, 2019
Last update: Feb 5, 2020

Study contacts

Suzanne Lentzsch, MD

principal investigator · Columbia University

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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