Trial Outcomes & Findings for A Study of PAD Followed by Autologous Stem Cell Transplantation (ASCT) to Treat Newly Diagnosed Multiple Myeloma (NCT NCT01852799)
NCT ID: NCT01852799
Last Updated: 2018-08-10
Results Overview
The bone formation marker- bone alkaline phosphatase(bALP) and osteoblast inhibitor- Dickkopf-1(DKK-1）are measured on serum samples by ELISA methodology at baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative multiple myeloma(MM) treatment, if earlier.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
18 participants
Primary outcome timeframe
Up to Cycle 4 with 28 days per cycle
Results posted on
2018-08-10
Participant Flow
Participant milestones
| Measure |
PAD Followed by ASCT
Drug: Bortezomib, Adriamycin (Doxorubicin) /Epidoxorubicin(EPI), Dexamethasone.
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
PAD Followed by ASCT: Drug: Bortezomib, Bortezomib (1.3mg/m2, iv, on day 1, 4, 8, 11, of each 28 day cycle)
Drug: Adriamycin (Doxorubicin) /EPI, Adriamycin (Doxorubicin) (9 mg/m2, iv, on days 1-4 of each 28 day cycle) or EPI (15mg/m2, iv, on days 1-4 of each 28 day cycle)
Drug: Dexamethasone, Dexamethasone (20mg, iv, on days 1-4, 8-11of each 28 day cycle)
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
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|---|---|
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Overall Study
STARTED
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18
|
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Overall Study
COMPLETED
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0
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Overall Study
NOT COMPLETED
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18
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of PAD Followed by Autologous Stem Cell Transplantation (ASCT) to Treat Newly Diagnosed Multiple Myeloma
Baseline characteristics by cohort
| Measure |
PAD Followed by ASCT
n=18 Participants
Drug: Bortezomib, Adriamycin (Doxorubicin) /Epidoxorubicin(EPI), Dexamethasone.
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
PAD Followed by ASCT: Drug: Bortezomib, Bortezomib (1.3mg/m2, iv, on day 1, 4, 8, 11, of each 28 day cycle)
Drug: Adriamycin (Doxorubicin) /EPI, Adriamycin (Doxorubicin) (9 mg/m2, iv, on days 1-4 of each 28 day cycle) or EPI (15mg/m2, iv, on days 1-4 of each 28 day cycle)
Drug: Dexamethasone, Dexamethasone (20mg, iv, on days 1-4, 8-11of each 28 day cycle)
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
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|---|---|
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Age, Categorical
<=18 years
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0 Participants
n=5 Participants
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Age, Categorical
Between 18 and 65 years
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18 Participants
n=5 Participants
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Age, Categorical
>=65 years
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0 Participants
n=5 Participants
|
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Age, Continuous
|
52.1 years
STANDARD_DEVIATION 6.8 • n=5 Participants
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Sex: Female, Male
Female
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6 Participants
n=5 Participants
|
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Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
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Region of Enrollment
China
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18 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Up to Cycle 4 with 28 days per cycleThe bone formation marker- bone alkaline phosphatase(bALP) and osteoblast inhibitor- Dickkopf-1(DKK-1）are measured on serum samples by ELISA methodology at baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative multiple myeloma(MM) treatment, if earlier.
Outcome measures
| Measure |
PAD Followed by ASCT
n=18 Participants
Drug: Bortezomib, Adriamycin (Doxorubicin) /Epidoxorubicin(EPI), Dexamethasone.
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
PAD Followed by ASCT: Drug: Bortezomib, Bortezomib (1.3mg/m2, iv, on day 1, 4, 8, 11, of each 28 day cycle)
Drug: Adriamycin (Doxorubicin) /EPI, Adriamycin (Doxorubicin) (9 mg/m2, iv, on days 1-4 of each 28 day cycle) or EPI (15mg/m2, iv, on days 1-4 of each 28 day cycle)
Drug: Dexamethasone, Dexamethasone (20mg, iv, on days 1-4, 8-11of each 28 day cycle)
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
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|---|---|
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Bone Formation Markers Measurement
bALP on baseline
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21.87 U/L
Standard Deviation 14.11
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Bone Formation Markers Measurement
bALP after cycle 1
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23.2 U/L
Standard Deviation 12.89
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Bone Formation Markers Measurement
bALP changes from baseline to cycle 1
|
1.33 U/L
Standard Deviation 1.02
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|
Bone Formation Markers Measurement
bALP after cycle 4
|
28.75 U/L
Standard Deviation 13.12
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Bone Formation Markers Measurement
bALP changes from baseline to cycle 4
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6.88 U/L
Standard Deviation 3.20
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Bone Formation Markers Measurement
DKK-1 on baseline
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4765 U/L
Standard Deviation 159.5
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Bone Formation Markers Measurement
DKK-1 aftercycle 1
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2653 U/L
Standard Deviation 135.2
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Bone Formation Markers Measurement
DKK-1 changes from baseline to cycle 1
|
2653 U/L
Standard Deviation 135.2
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Bone Formation Markers Measurement
DKK-1 after cycle 4
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1589 U/L
Standard Deviation 128.2
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Bone Formation Markers Measurement
DKK-1 changes from baseline to cycle 4
|
3176 U/L
Standard Deviation 123.8
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SECONDARY outcome
Timeframe: At baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatmentPopulation: Data were not collected.
The effect on bone mineral density（BMD） will be measured by quantitative analysis of qCT scans of the intra-individual same region \[lumbar spine and hip\] at baseline, on day 28 of cycles 4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment, if earlier.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At baseline, on day 28 of cycles 4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatmentPopulation: Data were not collected.
Skeletal survey of the skeleton using plain radiography will be performed at baseline, on day 28 of cycle 4, and after 6, 12 and 18 months of follow-up or until start of alternative MM treatment, if earlier. SREs, such as pathological fractures, need for radiation therapy or surgery will be recorded at the time of the event.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: At baseline, on day 28 of cycles 4, and after 4, 6, 12 and 18 months of follow-upEvaluation of responses was performed every cycle. Responses were assessed according to the european group for blood and marrow transplantation（EBMT） criteria (An attempt will be made to collect data for the assessment of stringent complete response (sCR) if these data are available.). Other efficacy parameters including PFS and 1-year overall survival rate and overall survival will be evaluated by normal methodology. According to EBMT criteria, responses were assessed by changes in the level of the serum paraprotein and/or urinary light chain excretion.The specific assessment rules may refer to EBMT criteria for MM.
Outcome measures
| Measure |
PAD Followed by ASCT
n=18 Participants
Drug: Bortezomib, Adriamycin (Doxorubicin) /Epidoxorubicin(EPI), Dexamethasone.
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
PAD Followed by ASCT: Drug: Bortezomib, Bortezomib (1.3mg/m2, iv, on day 1, 4, 8, 11, of each 28 day cycle)
Drug: Adriamycin (Doxorubicin) /EPI, Adriamycin (Doxorubicin) (9 mg/m2, iv, on days 1-4 of each 28 day cycle) or EPI (15mg/m2, iv, on days 1-4 of each 28 day cycle)
Drug: Dexamethasone, Dexamethasone (20mg, iv, on days 1-4, 8-11of each 28 day cycle)
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
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|---|---|
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Evaluation of Responses
After 4-month PAD treatment · MR
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1 Participants
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Evaluation of Responses
After 4-month PAD treatment · NC
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1 Participants
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Evaluation of Responses
After 4-month PAD treatment · PD
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0 Participants
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Evaluation of Responses
After ASCT · sCR
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4 Participants
|
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Evaluation of Responses
After ASCT · CR
|
0 Participants
|
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Evaluation of Responses
After ASCT · PR
|
12 Participants
|
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Evaluation of Responses
After ASCT · MR
|
1 Participants
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Evaluation of Responses
After ASCT · NC
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1 Participants
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Evaluation of Responses
After ASCT · PD
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0 Participants
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Evaluation of Responses
End of study · sCR
|
2 Participants
|
|
Evaluation of Responses
End of study · CR
|
1 Participants
|
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Evaluation of Responses
End of study · PR
|
9 Participants
|
|
Evaluation of Responses
End of study · MR
|
1 Participants
|
|
Evaluation of Responses
End of study · NC
|
1 Participants
|
|
Evaluation of Responses
End of study · PD
|
4 Participants
|
|
Evaluation of Responses
After 4-month PAD treatment · sCR
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4 Participants
|
|
Evaluation of Responses
After 4-month PAD treatment · CR
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0 Participants
|
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Evaluation of Responses
After 4-month PAD treatment · PR
|
12 Participants
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OTHER_PRE_SPECIFIED outcome
Timeframe: Starting with informed consent signature through study completion, an average of 1 yearSafety evaluations will be based on scheduled physical examinations, Eastern Cooperative Oncology Group(ECOG) scores, vital signs (blood pressure, heart rate) and clinical laboratory tests.
Outcome measures
| Measure |
PAD Followed by ASCT
n=18 Participants
Drug: Bortezomib, Adriamycin (Doxorubicin) /Epidoxorubicin(EPI), Dexamethasone.
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
PAD Followed by ASCT: Drug: Bortezomib, Bortezomib (1.3mg/m2, iv, on day 1, 4, 8, 11, of each 28 day cycle)
Drug: Adriamycin (Doxorubicin) /EPI, Adriamycin (Doxorubicin) (9 mg/m2, iv, on days 1-4 of each 28 day cycle) or EPI (15mg/m2, iv, on days 1-4 of each 28 day cycle)
Drug: Dexamethasone, Dexamethasone (20mg, iv, on days 1-4, 8-11of each 28 day cycle)
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
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|---|---|
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Safety Evaluation
AE
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15 Participants
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Safety Evaluation
TEAE
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15 Participants
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Safety Evaluation
TEAE related to treatment
|
14 Participants
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Safety Evaluation
TEAE(CTCAE≥3)
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11 Participants
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Safety Evaluation
TEAE related to treatment(CTCAE≥3)
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10 Participants
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Safety Evaluation
SAE
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0 Participants
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OTHER_PRE_SPECIFIED outcome
Timeframe: From date of signing informed consent form until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 monthsEfficacy parameters PFS will be evaluated by normal methodology. PFS =（date of signing informed consent form minus the date of first documented progression or date of death from any cause plus 1）/30.5（months）
Outcome measures
| Measure |
PAD Followed by ASCT
n=18 Participants
Drug: Bortezomib, Adriamycin (Doxorubicin) /Epidoxorubicin(EPI), Dexamethasone.
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
PAD Followed by ASCT: Drug: Bortezomib, Bortezomib (1.3mg/m2, iv, on day 1, 4, 8, 11, of each 28 day cycle)
Drug: Adriamycin (Doxorubicin) /EPI, Adriamycin (Doxorubicin) (9 mg/m2, iv, on days 1-4 of each 28 day cycle) or EPI (15mg/m2, iv, on days 1-4 of each 28 day cycle)
Drug: Dexamethasone, Dexamethasone (20mg, iv, on days 1-4, 8-11of each 28 day cycle)
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
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|---|---|
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Evaluation of Responses(PFS)
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13.7 months
Interval 10.3 to 13.7
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Adverse Events
PAD Followed by ASCT
Serious events: 0 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
PAD Followed by ASCT
n=18 participants at risk
Drug: Bortezomib, Adriamycin (Doxorubicin) /Epidoxorubicin(EPI), Dexamethasone.
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
PAD Followed by ASCT: Drug: Bortezomib, Bortezomib (1.3mg/m2, iv, on day 1, 4, 8, 11, of each 28 day cycle)
Drug: Adriamycin (Doxorubicin) /EPI, Adriamycin (Doxorubicin) (9 mg/m2, iv, on days 1-4 of each 28 day cycle) or EPI (15mg/m2, iv, on days 1-4 of each 28 day cycle)
Drug: Dexamethasone, Dexamethasone (20mg, iv, on days 1-4, 8-11of each 28 day cycle)
After received induction therapy, patients will proceed to receive ASCT based on the willing of the patients and the decision of the investigators.
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|---|---|
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Hepatobiliary disorders
ALT rise
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5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Infections and infestations
Herpes zoster
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16.7%
3/18 • Number of events 3 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Blood and lymphatic system disorders
Leukopenia
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44.4%
8/18 • Number of events 14 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Infections and infestations
Perineural inflammation
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5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Blood and lymphatic system disorders
Thrombocytopenia
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38.9%
7/18 • Number of events 19 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Gastrointestinal disorders
Constipation
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11.1%
2/18 • Number of events 3 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Blood and lymphatic system disorders
Hypokalemia
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5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Gastrointestinal disorders
Nausea
|
16.7%
3/18 • Number of events 3 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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General disorders
Fever
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16.7%
3/18 • Number of events 4 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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|
General disorders
Hypodynamia
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5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Infections and infestations
Pulmonary infection
|
5.6%
1/18 • Number of events 4 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
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Gastrointestinal disorders
Diarrhea
|
16.7%
3/18 • Number of events 3 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
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Blood and lymphatic system disorders
Hyperbilirubinemia
|
5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
|
Blood and lymphatic system disorders
Hyperuricemia
|
5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
|
Blood and lymphatic system disorders
Lymphopenia
|
16.7%
3/18 • Number of events 4 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
|
Blood and lymphatic system disorders
Hypoalbuminemia
|
11.1%
2/18 • Number of events 2 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
2/18 • Number of events 2 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
|
General disorders
Rash
|
5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
|
General disorders
Edema
|
5.6%
1/18 • Number of events 1 • On baseline, on day 28 of cycles 1,4, and after 4, 6, 12 and 18 months of follow-up or until start of alternative MM treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place