Trial Outcomes & Findings for Melphalan and Autologous Stem Cell Transplant Followed By Bortezomib and Dexamethasone in Treating Patients With Previously Untreated Systemic Amyloidosis (NCT NCT00458822)
NCT ID: NCT00458822
Last Updated: 2016-08-10
Results Overview
patients will be assessed for hematologic response (the response of the clonal plasma cell disease). If the plasma cell disease persists, then they will receive 6 cycles of adjuvant therapy with bortezomib and dexamethasone; patients with peripheral neuropathy will receive dexamethasone alone because of the risk of neuropathy associated with bortezomib. Symptomatic organ involvement with amyloid as defined below. Patients must have symptomatic involvement of no more than 2 of the following 4 visceral organ-systems: kidneys, liver/GI, peripheral/autonomic nervous system, and heart.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
40 participants
Primary outcome timeframe
2-3 months post transplant
Results posted on
2016-08-10
Participant Flow
Participant milestones
| Measure |
All Patients
All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis
|
|---|---|
|
Overall Study
STARTED
|
40
|
|
Overall Study
COMPLETED
|
40
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Melphalan and Autologous Stem Cell Transplant Followed By Bortezomib and Dexamethasone in Treating Patients With Previously Untreated Systemic Amyloidosis
Baseline characteristics by cohort
| Measure |
All Patients
n=40 Participants
All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis
|
|---|---|
|
Age, Continuous
|
57 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
23 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2-3 months post transplantpatients will be assessed for hematologic response (the response of the clonal plasma cell disease). If the plasma cell disease persists, then they will receive 6 cycles of adjuvant therapy with bortezomib and dexamethasone; patients with peripheral neuropathy will receive dexamethasone alone because of the risk of neuropathy associated with bortezomib. Symptomatic organ involvement with amyloid as defined below. Patients must have symptomatic involvement of no more than 2 of the following 4 visceral organ-systems: kidneys, liver/GI, peripheral/autonomic nervous system, and heart.
Outcome measures
| Measure |
All Patients
n=40 Participants
All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis
|
|---|---|
|
Hematologic and Organ Response
Complete Hematologic Response
|
11 participants
|
|
Hematologic and Organ Response
Partial Response
|
7 participants
|
|
Hematologic and Organ Response
Stable Disease
|
18 participants
|
Adverse Events
All Patients
Serious events: 20 serious events
Other events: 40 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Patients
n=40 participants at risk
All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis
|
|---|---|
|
General disorders
Arthritis (non-septic)
|
2.5%
1/40 • Number of events 1
|
|
Cardiac disorders
Atrial fibrillation
|
2.5%
1/40 • Number of events 1
|
|
Nervous system disorders
CNS cerebrovascular ischemia
|
2.5%
1/40 • Number of events 1
|
|
Cardiac disorders
Cardiac Arrhythmia, other
|
2.5%
1/40 • Number of events 1
|
|
Cardiac disorders
Cardiac General, other
|
15.0%
6/40 • Number of events 6
|
|
Blood and lymphatic system disorders
Coagulation, other
|
5.0%
2/40 • Number of events 2
|
|
General disorders
Conduction Abnormality NOS
|
2.5%
1/40 • Number of events 1
|
|
General disorders
Death not associated with CTCAE term- Death NOS
|
7.5%
3/40 • Number of events 3
|
|
Gastrointestinal disorders
Dehydration
|
2.5%
1/40 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
7.5%
3/40 • Number of events 4
|
|
General disorders
Febrile neutropenia
|
2.5%
1/40 • Number of events 1
|
|
General disorders
Fever (in the absence of neutropenia)
|
2.5%
1/40 • Number of events 1
|
|
Gastrointestinal disorders
Hemorrhage, Upper GI NOS
|
2.5%
1/40 • Number of events 1
|
|
General disorders
Hemorrhage/Bleeding, other
|
2.5%
1/40 • Number of events 1
|
|
Cardiac disorders
Hypotension
|
10.0%
4/40 • Number of events 5
|
|
General disorders
Hypoxia
|
2.5%
1/40 • Number of events 1
|
|
Infections and infestations
Neutrophil-Pneumonia (lung)
|
5.0%
2/40 • Number of events 2
|
|
Infections and infestations
Neutrophil-Up airway NOS
|
2.5%
1/40 • Number of events 1
|
|
Infections and infestations
Infection, other
|
12.5%
5/40 • Number of events 5
|
|
Gastrointestinal disorders
Nausea
|
2.5%
1/40 • Number of events 1
|
|
General disorders
Ocular/Visual - Other
|
2.5%
1/40 • Number of events 1
|
|
Blood and lymphatic system disorders
Platelets
|
10.0%
4/40 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Pulm/upper respiratory - Other
|
2.5%
1/40 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
2.5%
1/40 • Number of events 1
|
|
Renal and urinary disorders
Renal failure
|
7.5%
3/40 • Number of events 3
|
|
Cardiac disorders
Restrictive cardiomyopathy
|
2.5%
1/40 • Number of events 2
|
|
Metabolism and nutrition disorders
Sodium, low (hyponatremia)
|
2.5%
1/40 • Number of events 1
|
|
Cardiac disorders
Supraventricular tachycardia
|
2.5%
1/40 • Number of events 1
|
|
Nervous system disorders
Syncope (fainting)
|
10.0%
4/40 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Thrombosis/thrombus/embolism
|
5.0%
2/40 • Number of events 2
|
|
Nervous system disorders
Vasovagal episode
|
5.0%
2/40 • Number of events 2
|
|
Cardiac disorders
Ventricular arrhythmia- Ventricular tachycardia
|
2.5%
1/40 • Number of events 1
|
Other adverse events
| Measure |
All Patients
n=40 participants at risk
All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis
|
|---|---|
|
Metabolism and nutrition disorders
Albumin, low (hypoalbuminemia)
|
77.5%
31/40
|
|
Metabolism and nutrition disorders
Alkaline phosphatase
|
22.5%
9/40
|
|
Blood and lymphatic system disorders
ALT, SGPT
|
27.5%
11/40
|
|
Blood and lymphatic system disorders
AST, SGOT
|
15.0%
6/40
|
|
Metabolism and nutrition disorders
Bicarbonate, serum-low
|
12.5%
5/40
|
|
Metabolism and nutrition disorders
Bilirubin (hyperbilirubinemia)
|
15.0%
6/40
|
|
General disorders
Cholesterol,high(hypercholestremia)
|
15.0%
6/40
|
|
Gastrointestinal disorders
Constipation
|
22.5%
9/40
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.5%
3/40
|
|
Metabolism and nutrition disorders
Creatinine
|
55.0%
22/40
|
|
General disorders
Cushingoid appearance
|
5.0%
2/40
|
|
Gastrointestinal disorders
Dehydration
|
5.0%
2/40
|
|
Gastrointestinal disorders
Diarrhea
|
7.5%
3/40
|
|
Gastrointestinal disorders
Distension/bloating, abdominal
|
7.5%
3/40
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
10.0%
4/40
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
37.5%
15/40
|
|
Metabolism and nutrition disorders
Glucose, high (hyperglycemia)
|
67.5%
27/40
|
|
Metabolism and nutrition disorders
Glucose, low (hypoglycemia)
|
30.0%
12/40
|
|
Blood and lymphatic system disorders
Hemoglobin
|
92.5%
37/40
|
|
Infections and infestations
Infection, other
|
5.0%
2/40
|
|
Blood and lymphatic system disorders
Prothrombin Time international normalized ratio
|
25.0%
10/40
|
|
Blood and lymphatic system disorders
Leukocytes (total WBC)
|
95.0%
38/40
|
|
General disorders
Lipase
|
5.0%
2/40
|
|
Blood and lymphatic system disorders
Lymphopenia
|
97.5%
39/40
|
|
Metabolism and nutrition disorders
Magnesium, high (hypermagnesemia)
|
12.5%
5/40
|
|
Metabolism and nutrition disorders
Magnesium, low (hypomagnesemia)
|
5.0%
2/40
|
|
Gastrointestinal disorders
Nausea
|
10.0%
4/40
|
|
Nervous system disorders
Neuropathy: sensory
|
25.0%
10/40
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes
|
90.0%
36/40
|
|
Nervous system disorders
Pain - Neuralgia/peripheral nerve
|
17.5%
7/40
|
|
Metabolism and nutrition disorders
Phosphate, low (hypophosphatemia)
|
45.0%
18/40
|
|
Blood and lymphatic system disorders
Platelets
|
97.5%
39/40
|
|
Metabolism and nutrition disorders
Potassium, high (hyperkalemia)
|
22.5%
9/40
|
|
Metabolism and nutrition disorders
Potassium, low (hypokalemia)
|
35.0%
14/40
|
|
Skin and subcutaneous tissue disorders
Pruritus/itching
|
5.0%
2/40
|
|
Blood and lymphatic system disorders
Partial thromboplastin time
|
12.5%
5/40
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
5.0%
2/40
|
|
Renal and urinary disorders
Renal failure
|
5.0%
2/40
|
|
Metabolism and nutrition disorders
Sodium, low (hyponatremia)
|
30.0%
12/40
|
|
Nervous system disorders
Syncope (fainting)
|
7.5%
3/40
|
|
General disorders
Weight gain
|
5.0%
2/40
|
Additional Information
Dr. Heather Landau
Memorial Sloan Kettering Cancer Center
Phone: 212-639-8808
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place