Trial Outcomes & Findings for Bortezomib and Rituximab for Patients With Waldenstrom's Macroglobulinemia (NCT NCT00492050)
NCT ID: NCT00492050
Last Updated: 2025-09-02
Results Overview
Partial response (PR) defined as at least \> 50 % reduction of serum monoclonal IgM concentration determined by protein electrophoresis, \> 50% decrease in adenopathy / organomegaly on physical examination or on CT scan, and no new symptoms or signs of active disease. Complete response (CR) defined as a disappearance of serum and urine monoclonal protein determined by immunofixation, absence of malignant cells in bone marrow determined by histologic evaluation, resolution of adenopathy/organomegaly (confirmed by computed tomography \[CT\] scan), and no signs or symptoms attributable to WM. Stable disease defined as neither grown nor shrunk; the amount of disease has not changed.Participants will be followed for \< 6 months after receiving bortezomib/rituximab or rituximab-modified hyper-CVAD to be considered unresponsive to therapy in accordance with the guidelines of the Consensus Panel of the Third International Workshop on Waldenstrom's Macroglobulinemia.
ACTIVE_NOT_RECRUITING
PHASE2
46 participants
After 2 (35 day) cycles of treatment
2025-09-02
Participant Flow
Recruitment Period: August 2006- March 2013
46 Participants consented, 9 screen failures.
Participant milestones
| Measure |
Bortezomib + Rituximab
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
Overall Study
STARTED
|
37
|
|
Overall Study
Inadequate Response to First Two Cycles
|
25
|
|
Overall Study
Inadequate Response to First 3 Cycles
|
12
|
|
Overall Study
If Adequate Response to First Two Cycles-participants Proceed to Stem Cell Collection
|
30
|
|
Overall Study
COMPLETED
|
28
|
|
Overall Study
NOT COMPLETED
|
9
|
Reasons for withdrawal
| Measure |
Bortezomib + Rituximab
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Lack of Efficacy
|
1
|
|
Overall Study
Withdrawal by Subject
|
3
|
|
Overall Study
Unable to collect SCT
|
2
|
|
Overall Study
Progressive Disease
|
2
|
Baseline Characteristics
Bortezomib and Rituximab for Patients With Waldenstrom's Macroglobulinemia
Baseline characteristics by cohort
| Measure |
All Participants
n=37 Participants
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
28 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
9 Participants
n=5 Participants
|
|
Age, Continuous
|
61 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
35 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
37 participants
n=5 Participants
|
|
Newly diagnosed patient with Waldenstrom Macroglobulinemia with no prior treatment
|
37 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: After 2 (35 day) cycles of treatmentPopulation: 1 subject didn't complete 2 cycles of therapy and isn't evaluable for response
Partial response (PR) defined as at least \> 50 % reduction of serum monoclonal IgM concentration determined by protein electrophoresis, \> 50% decrease in adenopathy / organomegaly on physical examination or on CT scan, and no new symptoms or signs of active disease. Complete response (CR) defined as a disappearance of serum and urine monoclonal protein determined by immunofixation, absence of malignant cells in bone marrow determined by histologic evaluation, resolution of adenopathy/organomegaly (confirmed by computed tomography \[CT\] scan), and no signs or symptoms attributable to WM. Stable disease defined as neither grown nor shrunk; the amount of disease has not changed.Participants will be followed for \< 6 months after receiving bortezomib/rituximab or rituximab-modified hyper-CVAD to be considered unresponsive to therapy in accordance with the guidelines of the Consensus Panel of the Third International Workshop on Waldenstrom's Macroglobulinemia.
Outcome measures
| Measure |
All Participants
n=36 Participants
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
Response Rate After 2 Cycles of Treatment With Bortezomib and Rituximab
Stable Disease
|
20 Participants
|
|
Response Rate After 2 Cycles of Treatment With Bortezomib and Rituximab
Partial Response
|
11 Participants
|
|
Response Rate After 2 Cycles of Treatment With Bortezomib and Rituximab
Progressive Disease
|
2 Participants
|
|
Response Rate After 2 Cycles of Treatment With Bortezomib and Rituximab
No Response
|
3 Participants
|
PRIMARY outcome
Timeframe: After 3 (35 day) cycles of treatmentPartial response (PR) defined as at least \> 50 % reduction of serum monoclonal IgM concentration determined by protein electrophoresis, \> 50% decrease in adenopathy / organomegaly on physical examination or on CT scan, and no new symptoms or signs of active disease. Complete response (CR) defined as a disappearance of serum and urine monoclonal protein determined by immunofixation, absence of malignant cells in bone marrow determined by histologic evaluation, resolution of adenopathy/organomegaly (confirmed by computed tomography \[CT\] scan), and no signs or symptoms attributable to WM. Stable disease defined as neither grown nor shrunk; the amount of disease has not changed. Participants will be followed for \< 6 months after receiving bortezomib/rituximab or rituximab-modified hyper-CVAD to be considered unresponsive to therapy in accordance with the guidelines of the Consensus Panel of the Third International Workshop on Waldenstrom's Macroglobulinemia.
Outcome measures
| Measure |
All Participants
n=25 Participants
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
Response Rate After 3 Cycles of Treatment With Bortezomib and Rituximab
Stable Disease
|
7 Participants
|
|
Response Rate After 3 Cycles of Treatment With Bortezomib and Rituximab
Partial Response
|
16 Participants
|
|
Response Rate After 3 Cycles of Treatment With Bortezomib and Rituximab
No Response
|
2 Participants
|
PRIMARY outcome
Timeframe: 4 weeksNumber of Participants who were able to collect stem cells successfully or unable to collect after treatment with bortezomib and rituximab.
Outcome measures
| Measure |
All Participants
n=37 Participants
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
Participants Ability to Collect Stem Cells After Treatment With Bortezomib and Rituximab
Participants Collected Successfully
|
30 Participants
|
|
Participants Ability to Collect Stem Cells After Treatment With Bortezomib and Rituximab
Participants Unable to Collect
|
7 Participants
|
Adverse Events
All Participants
Serious adverse events
| Measure |
All Participants
n=37 participants at risk
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
5.4%
2/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Cardiac disorders
Hypotension
|
5.4%
2/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Gastrointestinal disorders
Diarrhea
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Gastrointestinal disorders
Elevated Alkaline Phosphate
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Infections and infestations
Fever
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Infections and infestations
Gram negative bacteremia
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Blood and lymphatic system disorders
Neutropenia
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Infections and infestations
Neutrophenic fever
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary nodules
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Infections and infestations
Spetic shock
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
General disorders
Visual changes
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Blood and lymphatic system disorders
Hyperviscosity
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
Other adverse events
| Measure |
All Participants
n=37 participants at risk
Bortezomib 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22. Rituximab 375 mg/m\^2 IV on Day 8 and 22. Valacyclovir 500 mg orally daily (or acyclovir 200 mg orally twice daily).
Bortezomib: 1.6 mg/m\^2 IV Weekly on Days 1, 8, 15 and 22.
Rituximab: 375 mg/m\^2 IV on Day 8 and 22.
Valacyclovir: 500 mg orally daily (or acyclovir 200 mg orally twice daily)
|
|---|---|
|
General disorders
Fatigue
|
16.2%
6/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Gastrointestinal disorders
Diarrhea
|
8.1%
3/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain (joint)
|
10.8%
4/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain (muscle)
|
10.8%
4/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Blood and lymphatic system disorders
Neutropenia
|
10.8%
4/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Nervous system disorders
Dizziness
|
5.4%
2/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Gastrointestinal disorders
Vomiting
|
5.4%
2/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Renal and urinary disorders
BUN increase
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Renal and urinary disorders
Creatinine increase
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Eye disorders
Dry eye
|
8.1%
3/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.4%
2/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Vascular disorders
Peripheral Edema
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Blood and lymphatic system disorders
Leukopenia
|
8.1%
3/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Nervous system disorders
Neuopathy
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
2.7%
1/37 • From the first dose through 30 days after the last dosage up to 5 years
|
Additional Information
Sheeba Thomas, MD-Professor, Lymphoma-Myeloma
UT MD Anderson Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place