Trial Outcomes & Findings for Lenalidomide and Dexamethasone With/Without Stem Cell Transplant in Patients With Multiple Myeloma (NCT NCT01731886)
NCT ID: NCT01731886
Last Updated: 2020-02-05
Results Overview
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)).
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
60 participants
Primary outcome timeframe
3 years
Results posted on
2020-02-05
Participant Flow
Participant milestones
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
Autologous peripheral blood stem cell transplant: Subjects deemed suitable by the principal investigator will undergo autologous peripheral blood stem cell transplantation on day 0.
Lenalidomide: Administered orally at a dose 25 mg daily on days 1-21 of each 28-day cycle.
Dexamethasone: Administered orally at a dose of 40 mg daily on days 1, 8, 15, 22 of each cycle.
Stem 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.
Melphalan: Subjects undergoing autologous peripheral blood stem cell transplant will receive
|
Arm B: Low-dose Dexamethasone
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).
Lenalidomide: Administered orally at a dose 25 mg daily on days 1-21 of each 28-day cycle.
Dexamethasone: Administered orally at a dose of 40 mg daily on days 1, 8, 15, 22 of each cycle.
Stem 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.
Cyclophosphamide: Subjects may receive up to the maximum recommended high-dose of cyclophosphamide at 4 gm/m2 intravenously.
Mesna: Mesna will be provided with the cyclophosphamide.
|
|---|---|---|
|
Overall Study
STARTED
|
31
|
29
|
|
Overall Study
COMPLETED
|
29
|
28
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
Autologous peripheral blood stem cell transplant: Subjects deemed suitable by the principal investigator will undergo autologous peripheral blood stem cell transplantation on day 0.
Lenalidomide: Administered orally at a dose 25 mg daily on days 1-21 of each 28-day cycle.
Dexamethasone: Administered orally at a dose of 40 mg daily on days 1, 8, 15, 22 of each cycle.
Stem 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.
Melphalan: Subjects undergoing autologous peripheral blood stem cell transplant will receive
|
Arm B: Low-dose Dexamethasone
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).
Lenalidomide: Administered orally at a dose 25 mg daily on days 1-21 of each 28-day cycle.
Dexamethasone: Administered orally at a dose of 40 mg daily on days 1, 8, 15, 22 of each cycle.
Stem 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.
Cyclophosphamide: Subjects may receive up to the maximum recommended high-dose of cyclophosphamide at 4 gm/m2 intravenously.
Mesna: Mesna will be provided with the cyclophosphamide.
|
|---|---|---|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Never started treatment
|
0
|
1
|
|
Overall Study
Non-compliant
|
1
|
0
|
Baseline Characteristics
Lenalidomide and Dexamethasone With/Without Stem Cell Transplant in Patients With Multiple Myeloma
Baseline characteristics by cohort
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=31 Participants
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=29 Participants
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).
|
Total
n=60 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
21 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
23 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
ISS Stage
Stage 1(B2M <3.5 mg/L and serum albumin ≥3.5 g/dL)
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
|
ISS Stage
Stage 2(Neither stage I nor stage III)
|
14 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
ISS Stage
Stage 3(B2M ≥5.5 mg/L)
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 yearsPopulation: 1 participant in Arm B never started treatment.
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)).
Outcome measures
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=31 Participants
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=28 Participants
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).
|
|---|---|---|
|
Complete Response Rate
|
7 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: 4 yearsPopulation: Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis.
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.
Outcome measures
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=25 Participants
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=19 Participants
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).
|
|---|---|---|
|
Overall Survival Rate (OS)
|
79.8 percentage of participants
Interval 64.0 to 95.6
|
78.9 percentage of participants
Interval 60.6 to 97.3
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis.
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.
Outcome measures
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=25 Participants
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=19 Participants
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).
|
|---|---|---|
|
Overall Survival Rate (OS)
|
100 percentage of participants
Interval 95.0 to 100.0
|
94.7 percentage of participants
Interval 85.4 to 99.9
|
SECONDARY outcome
Timeframe: 4 yearsPopulation: Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis.
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.
Outcome measures
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=25 Participants
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=19 Participants
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).
|
|---|---|---|
|
Progression Free Survival (PFS)
|
36.0 percentage of participants
Interval 17.2 to 54.8
|
31.6 percentage of participants
Interval 10.7 to 52.5
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis.
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.
Outcome measures
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=25 Participants
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=19 Participants
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).
|
|---|---|---|
|
Progression Free Survival (PFS)
|
52.0 percentage of participants
Interval 32.4 to 71.6
|
47.4 percentage of participants
Interval 24.9 to 69.8
|
Adverse Events
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
Serious events: 7 serious events
Other events: 21 other events
Deaths: 14 deaths
Arm B: Low-dose Dexamethasone
Serious events: 7 serious events
Other events: 19 other events
Deaths: 11 deaths
Serious adverse events
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=31 participants at risk
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=28 participants at risk
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).
Only includes subjects who received drug.
|
|---|---|---|
|
Cardiac disorders
Presyncope
|
0.00%
0/31 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Bone Pain
|
3.2%
1/31 • Number of events 2 • 3 years
|
3.6%
1/28 • Number of events 2 • 3 years
|
|
Infections and infestations
Febrile Neutropenia
|
3.2%
1/31 • Number of events 2 • 3 years
|
0.00%
0/28 • 3 years
|
|
Infections and infestations
Infection with Grade 3 or 4 Neutrophils
|
3.2%
1/31 • Number of events 1 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
Infections and infestations
Infection
|
0.00%
0/31 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
Infections and infestations
Lung Infection
|
3.2%
1/31 • Number of events 3 • 3 years
|
0.00%
0/28 • 3 years
|
|
Blood and lymphatic system disorders
Myelodysplastic Syndrome
|
0.00%
0/31 • 3 years
|
3.6%
1/28 • Number of events 2 • 3 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms
|
3.2%
1/31 • Number of events 2 • 3 years
|
0.00%
0/28 • 3 years
|
|
General disorders
Pain
|
3.2%
1/31 • Number of events 2 • 3 years
|
0.00%
0/28 • 3 years
|
|
Skin and subcutaneous tissue disorders
Pain of skin
|
0.00%
0/31 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
General disorders
Abdominal Pain
|
0.00%
0/31 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
General disorders
Back Pain
|
0.00%
0/31 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
Infections and infestations
Skin Infection
|
3.2%
1/31 • Number of events 1 • 3 years
|
0.00%
0/28 • 3 years
|
|
General disorders
Syncope
|
3.2%
1/31 • Number of events 1 • 3 years
|
0.00%
0/28 • 3 years
|
|
Vascular disorders
Thromboembolic Event
|
0.00%
0/31 • 3 years
|
3.6%
1/28 • Number of events 2 • 3 years
|
Other adverse events
| Measure |
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation
n=31 participants at risk
Subjects will receive the current standard of care treatment. Lenalidomide and dexamethasone for four 28-day cycles followed by stem 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).
|
Arm B: Low-dose Dexamethasone
n=28 participants at risk
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).
Only includes subjects who received drug.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
6.5%
2/31 • Number of events 12 • 3 years
|
14.3%
4/28 • Number of events 14 • 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
9.7%
3/31 • Number of events 7 • 3 years
|
17.9%
5/28 • Number of events 11 • 3 years
|
|
Blood and lymphatic system disorders
Decreased Neutrophil Count
|
25.8%
8/31 • Number of events 22 • 3 years
|
14.3%
4/28 • Number of events 7 • 3 years
|
|
Blood and lymphatic system disorders
Decreased WBC Count
|
19.4%
6/31 • Number of events 13 • 3 years
|
10.7%
3/28 • Number of events 7 • 3 years
|
|
Blood and lymphatic system disorders
Decreased Platelet Count
|
16.1%
5/31 • Number of events 24 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
Blood and lymphatic system disorders
Abnormal Neutrophils/Granulocytes (ANC/AGC)
|
16.1%
5/31 • Number of events 11 • 3 years
|
25.0%
7/28 • Number of events 26 • 3 years
|
|
General disorders
Pain in extremity
|
6.5%
2/31 • Number of events 3 • 3 years
|
3.6%
1/28 • Number of events 6 • 3 years
|
|
Hepatobiliary disorders
Increased Alanine Aminotransferase
|
6.5%
2/31 • Number of events 6 • 3 years
|
7.1%
2/28 • Number of events 3 • 3 years
|
|
General disorders
Cough
|
12.9%
4/31 • Number of events 7 • 3 years
|
10.7%
3/28 • Number of events 3 • 3 years
|
|
General disorders
Insomnia
|
9.7%
3/31 • Number of events 4 • 3 years
|
17.9%
5/28 • Number of events 7 • 3 years
|
|
Infections and infestations
Infection
|
9.7%
3/31 • Number of events 5 • 3 years
|
17.9%
5/28 • Number of events 7 • 3 years
|
|
Blood and lymphatic system disorders
Abnormal Leukocytes
|
6.5%
2/31 • Number of events 3 • 3 years
|
10.7%
3/28 • Number of events 8 • 3 years
|
|
Blood and lymphatic system disorders
Abnormal Platelet Count
|
6.5%
2/31 • Number of events 3 • 3 years
|
14.3%
4/28 • Number of events 12 • 3 years
|
|
Blood and lymphatic system disorders
Abnormal Hemoglobin
|
3.2%
1/31 • Number of events 1 • 3 years
|
10.7%
3/28 • Number of events 8 • 3 years
|
|
Infections and infestations
Upper Respiratory Infection
|
3.2%
1/31 • Number of events 2 • 3 years
|
7.1%
2/28 • Number of events 3 • 3 years
|
|
Infections and infestations
Urinary Tract Infection
|
6.5%
2/31 • Number of events 3 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.5%
2/31 • Number of events 3 • 3 years
|
10.7%
3/28 • Number of events 5 • 3 years
|
|
General disorders
Paresthesia
|
0.00%
0/31 • 3 years
|
10.7%
3/28 • Number of events 4 • 3 years
|
|
General disorders
Nausea
|
6.5%
2/31 • Number of events 3 • 3 years
|
7.1%
2/28 • Number of events 4 • 3 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
6.5%
2/31 • Number of events 3 • 3 years
|
3.6%
1/28 • Number of events 2 • 3 years
|
|
General disorders
Fatigue
|
9.7%
3/31 • Number of events 4 • 3 years
|
10.7%
3/28 • Number of events 5 • 3 years
|
|
General disorders
Abdominal Pain
|
6.5%
2/31 • Number of events 4 • 3 years
|
3.6%
1/28 • Number of events 1 • 3 years
|
|
Infections and infestations
Fever
|
3.2%
1/31 • Number of events 2 • 3 years
|
7.1%
2/28 • Number of events 3 • 3 years
|
|
Blood and lymphatic system disorders
Decreased Lymphocyte Count
|
0.00%
0/31 • 3 years
|
10.7%
3/28 • Number of events 6 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and Connective Tissue Disorder
|
0.00%
0/31 • 3 years
|
10.7%
3/28 • Number of events 4 • 3 years
|
Additional Information
Suzanne Lentzsch, MD, PhD, Professor of Medicine
Columbia University
Phone: 646-317-4840
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place