Trial Outcomes & Findings for Bortezomib, Melphalan, and Dexamethasone in Treating Patients With Primary Amyloidosis or Light Chain Deposition Disease (NCT NCT00520767)
NCT ID: NCT00520767
Last Updated: 2023-12-22
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
35 participants
Primary outcome timeframe
Up to 12 months
Results posted on
2023-12-22
Participant Flow
Participant milestones
| Measure |
Melphalan, Dexamethasone, Bortezomib,
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
Overall Study
STARTED
|
35
|
|
Overall Study
COMPLETED
|
33
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Melphalan, Dexamethasone, Bortezomib,
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
Overall Study
Death
|
2
|
Baseline Characteristics
Bortezomib, Melphalan, and Dexamethasone in Treating Patients With Primary Amyloidosis or Light Chain Deposition Disease
Baseline characteristics by cohort
| Measure |
Melphalan, Dexamethasone, Bortezomib,
n=35 Participants
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
Age, Continuous
|
64 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
35 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 12 monthsPopulation: Individuals evaluable for response
Outcome measures
| Measure |
Melphalan, Dexamethasone, Bortezomib,
n=33 Participants
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
Complete Hematologic Response
|
16 participants
|
SECONDARY outcome
Timeframe: time from day of registration until 72 months.time from day of registration until day of death.
Outcome measures
| Measure |
Melphalan, Dexamethasone, Bortezomib,
n=35 Participants
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
Overall Survival
|
31.1 month
Interval 22.1 to 45.5
|
SECONDARY outcome
Timeframe: start of treatment until 72 monthsTime from start of treatment until date of documented disease progression, removal from protocol due to toxicity, or death from any cause.
Outcome measures
| Measure |
Melphalan, Dexamethasone, Bortezomib,
n=35 Participants
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
Time to Treatment Failure (TTF)
|
18.1 month
Interval 10.8 to 34.4
|
SECONDARY outcome
Timeframe: Beginning of cycles 4, 8, 12, 16 and 20, at follow up and end of study.Population: Not collected not analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Beginning of cycles 4, 8, 12, 16 and 20, at follow up and end of study.Population: Not collected not analyzed.
Outcome measures
Outcome data not reported
Adverse Events
Melphalan, Dexamethasone, Bortezomib,
Serious events: 12 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Melphalan, Dexamethasone, Bortezomib,
n=35 participants at risk
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
General disorders
Death
|
5.7%
2/35 • Number of events 2
|
|
Infections and infestations
Infection with Normal ANC-Lung (pneumonia)
|
5.7%
2/35 • Number of events 2
|
|
General disorders
Pain-chest wall
|
2.9%
1/35 • Number of events 1
|
|
Cardiac disorders
Congestive Heart Failure
|
2.9%
1/35 • Number of events 1
|
|
Gastrointestinal disorders
Dehydration
|
5.7%
2/35 • Number of events 2
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
2.9%
1/35 • Number of events 1
|
|
Metabolism and nutrition disorders
Hyponatremia
|
2.9%
1/35 • Number of events 1
|
|
Psychiatric disorders
Mental Status Change
|
5.7%
2/35 • Number of events 2
|
|
Cardiac disorders
Hypotension
|
2.9%
1/35 • Number of events 1
|
|
Gastrointestinal disorders
Nausea
|
2.9%
1/35 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
2.9%
1/35 • Number of events 1
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
5.7%
2/35 • Number of events 2
|
|
Metabolism and nutrition disorders
Creatinine
|
5.7%
2/35 • Number of events 2
|
|
Cardiac disorders
Ventricular Arrhythmia
|
5.7%
2/35 • Number of events 2
|
|
Blood and lymphatic system disorders
Edema
|
5.7%
2/35 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.7%
2/35 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.7%
2/35 • Number of events 2
|
|
Nervous system disorders
syncope
|
2.9%
1/35 • Number of events 1
|
|
Cardiac disorders
Hypertension
|
5.7%
2/35 • Number of events 2
|
|
Nervous system disorders
Pain-Headache
|
5.7%
2/35 • Number of events 2
|
|
Renal and urinary disorders
Renal Failure
|
2.9%
1/35 • Number of events 1
|
|
Infections and infestations
Infection with Normal ANC-Catheter related
|
2.9%
1/35 • Number of events 1
|
|
Infections and infestations
Infection with Normal ANC-Skin (cellulitis)-shingles
|
2.9%
1/35 • Number of events 1
|
|
Cardiac disorders
cardiac Arrhythmia
|
2.9%
1/35 • Number of events 1
|
Other adverse events
| Measure |
Melphalan, Dexamethasone, Bortezomib,
n=35 participants at risk
Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4
bortezomib: Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
dexamethasone: Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
melphalan: Melphalan 9 mg/m2/day days 1-4
microarray analysis: ≤28 days prior to enrollment
flow cytometry: Day 1 of cycles 6, 12, 18 and at end of study.
laboratory biomarker analysis: ≤28 days prior to enrollment
quality-of-life assessment: Start of each cycle
|
|---|---|
|
Gastrointestinal disorders
Constipation
|
5.7%
2/35 • Number of events 2
|
|
Blood and lymphatic system disorders
Hemoglobin
|
5.7%
2/35 • Number of events 2
|
Additional Information
Jeffrey Zonder, M.D.
Barbara Ann Karmanos Cancer Institute
Phone: 313-576-8732
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place