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Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation for Primary (AL) Amyloidosis

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ClinicalTrials.gov Identifier: NCT01083316
Recruitment Status : Active, not recruiting
First Posted : March 9, 2010
Results First Posted : February 6, 2017
Last Update Posted : May 8, 2018
Sponsor:
Collaborator:
Millennium Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Vaishali Sanchorawala, Boston Medical Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Amyloidosis
Interventions Drug: Bortezomib
Drug: Dexamethasone
Drug: Melphalan
Enrollment 35
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Bortezomib and Dexamethasone
Hide Arm/Group Description

Induction:

Bortezomib (Velcade) 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4 Melphalan 70-100 mg/m2/day IV on days -2 and -1

Bortezomib (Velcade) and Dexamethasone: Induction:

Velcade 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4

Melphalan 70-100 mg/m2/day IV on days -2 and -1

Period Title: Overall Study
Started 35
Completed 32
Not Completed 3
Reason Not Completed
Adverse Event             2
Withdrawal by Subject             1
Arm/Group Title Bortezomib and Dexamethasone
Hide Arm/Group Description

Induction:

Bortezomib (Velcade) 1.3 mg/m2/dose intravenous (IV) Days 1, 4, 8, 11 repeated every 21 days Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4 Melphalan 70-100 mg/m2/day IV on days -2 and -1

Bortezomib (Velcade) and Dexamethasone: Induction:

Velcade 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4

Melphalan 70-100 mg/m2/day IV on days -2 and -1

Overall Number of Baseline Participants 35
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
<=18 years
0
   0.0%
Between 18 and 65 years
30
  85.7%
>=65 years
5
  14.3%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants
Female
22
  62.9%
Male
13
  37.1%
1.Primary Outcome
Title Number of Participants With Disease Response
Hide Description Complete response: Normal serum free light chain ratio and Negative serum and urine immunofixation electrophoresis Very good partial response: Difference in serum free light chains less than 40 mg/L Partial Response: >50% Reduction in the difference in serum free light chains
Time Frame One year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bortezomib and Dexamethasone
Hide Arm/Group Description:

Induction:

Bortezomib (Velcade) 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4 Melphalan 70-100 mg/m2/day IV on days -2 and -1

Bortezomib (Velcade) and Dexamethasone: Induction:

Velcade 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4

Melphalan 70-100 mg/m2/day IV on days -2 and -1

Overall Number of Participants Analyzed 26
Measure Type: Count of Participants
Unit of Measure: Participants
20
  76.9%
2.Primary Outcome
Title Number of Participants Surviving at 100 Days Post Transplant
Hide Description [Not Specified]
Time Frame 100 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bortezomib and Dexamethasone
Hide Arm/Group Description:

Induction:

Bortezomib (Velcade) 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4 Melphalan 70-100 mg/m2/day IV on days -2 and -1

Bortezomib (Velcade) and Dexamethasone: Induction:

Velcade 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4

Melphalan 70-100 mg/m2/day IV on days -2 and -1

Overall Number of Participants Analyzed 30
Measure Type: Count of Participants
Unit of Measure: Participants
27
  90.0%
3.Primary Outcome
Title Number of Participants Proceeding to Transplant Following Induction
Hide Description [Not Specified]
Time Frame 2 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bortezomib and Dexamethasone
Hide Arm/Group Description:

Induction:

Bortezomib (Velcade) 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4 Melphalan 70-100 mg/m2/day IV on days -2 and -1

Bortezomib (Velcade) and Dexamethasone: Induction:

Velcade 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4

Melphalan 70-100 mg/m2/day IV on days -2 and -1

Overall Number of Participants Analyzed 35
Measure Type: Count of Participants
Unit of Measure: Participants
30
  85.7%
4.Secondary Outcome
Title Number of Participants Surviving at 5 Years
Hide Description [Not Specified]
Time Frame 5 years
Outcome Measure Data Not Reported
Time Frame 100 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Bortezomib and Dexamethasone
Hide Arm/Group Description

Induction:

Bortezomib (Velcade) 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4 Melphalan 70-100 mg/m2/day IV on days -2 and -1

Bortezomib (Velcade) and Dexamethasone: Induction:

Velcade 1.3 mg/m2/dose IV Days 1, 4, 8, 11 repeated every 21 days

Dexamethasone 20 mg PO/IV Days 1, 4, 8, 11 repeated every 21 days

Conditioning:

Bortezomib 1.0 mg/m2/dose will be administered on Days +6, -3, +1, + 4

Melphalan 70-100 mg/m2/day IV on days -2 and -1

All-Cause Mortality
Bortezomib and Dexamethasone
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Bortezomib and Dexamethasone
Affected / at Risk (%) # Events
Total   35/35 (100.00%)    
Blood and lymphatic system disorders   
neutropenic fever * 1  7/35 (20.00%)  7
Epstein Barr Virus-related Polymorphous Post Transplant Lymphoproliferative disorder * 1  1/35 (2.86%)  1
thrombocytopenia * 1  1/35 (2.86%)  1
Cardiac disorders   
cardiac arrest * 1  1/35 (2.86%)  1
Gastrointestinal disorders   
diarrhea * 1  15/35 (42.86%)  15
gastrointestinal bleed * 1  1/35 (2.86%)  1
hemorrhoidal hemmorhage * 1  1/35 (2.86%)  1
General disorders   
fever * 2  7/35 (20.00%) 
fatigue * 1  18/35 (51.43%)  18
multiorgan failure * 1  1/35 (2.86%)  1
nausea * 1  6/35 (17.14%)  6
Immune system disorders   
autograft versus host disease * 1  2/35 (5.71%)  2
Infections and infestations   
invasive aspirgillosis * 1  1/35 (2.86%)  1
sepsis * 1  4/35 (11.43%)  4
stomatitis * 1  1/35 (2.86%)  1
Investigations   
creatinine increased * 1  1/35 (2.86%)  1
Nervous system disorders   
syncope * 1  8/35 (22.86%)  9
Dizziness * 1  2/35 (5.71%)  2
Renal and urinary disorders   
Acute kidney failure * 1  3/35 (8.57%)  3
Respiratory, thoracic and mediastinal disorders   
Adult Respiratory Distress Syndrome * 1  1/35 (2.86%)  1
epistaxis * 1  1/35 (2.86%)  1
Skin and subcutaneous tissue disorders   
skin rash * 1  3/35 (8.57%)  3
Vascular disorders   
hypotension * 1  11/35 (31.43%)  11
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (Unspecified)
2
Term from vocabulary, CTCAE 4
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Bortezomib and Dexamethasone
Affected / at Risk (%) # Events
Total   35/35 (100.00%)    
Blood and lymphatic system disorders   
drug rash * 1  3/35 (8.57%)  3
petechiae * 1  4/35 (11.43%)  4
Cardiac disorders   
sinus tachycardia * 1  16/35 (45.71%)  16
Ear and labyrinth disorders   
tinnitus * 1  2/35 (5.71%)  2
Endocrine disorders   
adrenal insufficiency * 1  4/35 (11.43%)  4
Eye disorders   
Blurred vision * 1  7/35 (20.00%)  7
Gastrointestinal disorders   
nausea * 1  15/35 (42.86%)  15
diarrhea * 1  14/35 (40.00%)  14
constipation * 1  13/35 (37.14%)  13
anorexia * 1  5/35 (14.29%)  5
abdominal distension * 1  4/35 (11.43%)  4
Abdominal pain * 1  3/35 (8.57%)  3
oral mucositis * 1  3/35 (8.57%)  3
General disorders   
peripheral edema * 1  5/35 (14.29%)  5
Investigations   
alkaline phosphatase elevated * 1  4/35 (11.43%)  4
Musculoskeletal and connective tissue disorders   
muscle weakness * 1  5/35 (14.29%)  5
Bone pain * 1  12/35 (34.29%)  14
Nervous system disorders   
dizziness * 1  4/35 (11.43%)  6
anxiety * 1  7/35 (20.00%)  12
Respiratory, thoracic and mediastinal disorders   
dyspnea * 1  10/35 (28.57%)  10
cough * 1  6/35 (17.14%)  7
Skin and subcutaneous tissue disorders   
alopecia * 1  14/35 (40.00%)  14
rash * 1  7/35 (20.00%)  7
Vascular disorders   
hypotension * 1  8/35 (22.86%)  8
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (Unspecified)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Vaishali Sanchorawala
Organization: Boston Medical Center
Phone: 6176386521
Responsible Party: Vaishali Sanchorawala, Boston Medical Center
ClinicalTrials.gov Identifier: NCT01083316     History of Changes
Other Study ID Numbers: H-28441
X05292 ( Other Identifier: Millennium )
First Submitted: November 2, 2009
First Posted: March 9, 2010
Results First Submitted: December 13, 2016
Results First Posted: February 6, 2017
Last Update Posted: May 8, 2018