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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 : April 24, 2019
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
Hide 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
Hide 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
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
number of patients that completed at least one cycle of induction therapy
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: Induction:

Velcade 1.3 mg/m2/dose 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

Dexamethasone: Induction:

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

Melphalan: Conditioning:

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
29
  82.9%
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
Layout table for Results Point of Contact information
Name/Title: Vaishali Sanchorawala
Organization: Boston Medical Center
Phone: 6176386521
EMail: vaishali.sanchorawala@bmc.org
Layout table for additonal information
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: April 24, 2019