Trial Outcomes & Findings for Combination Bortezomib and Rituximab in Patients With Waldenstrom's Macroglobulinemia (WM) (NCT NCT00422799)

Last Updated: 2020-10-27

Results Overview

Overall Response Rate= Minor response (\>25%-50% reduction in monoclonal IgM from baseline + Partial Response (\>50-90% reduction in monoclonal IgM from baseline)+ Complete Response (Disappearance of monoclonal protein by immunofixation; no histologic evidence of bone marrow involvement, resolution of any adenopathy/organomegaly (confirmed by CT scan), or signs or symptoms attributable to WM. Reconfirmation of the CR status is required at least 6 weeks apart with a second immunofixation.)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

63 participants

Primary outcome timeframe

2 Years

Results posted on

2020-10-27

Participant Flow

Participant milestones

Participant milestones
Measure	Bortezomib and Rituximab bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Overall Study STARTED	63
Overall Study COMPLETED	50
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Bortezomib and Rituximab bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Overall Study Withdrawal by Subject	6
Overall Study Adverse Event	7

Baseline Characteristics

Combination Bortezomib and Rituximab in Patients With Waldenstrom's Macroglobulinemia (WM)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bortezomib and Rituximab n=63 Participants bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	36 Participants n=5 Participants
Age, Categorical >=65 years	27 Participants n=5 Participants
Age, Continuous	63 years n=5 Participants
Sex: Female, Male Female	22 Participants n=5 Participants
Sex: Female, Male Male	41 Participants n=5 Participants
Region of Enrollment United States	63 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 Years

Population: Patients who had received at least one prior line of therapy

Outcome measures

Outcome measures
Measure	Bortezomib and Rituximab n=37 Participants bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Overall Response Rate of Bortezomib and Rituximab (VR) in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia (WM)	30 Participants

PRIMARY outcome

Timeframe: 2 years

Population: Participants who were previously untreated.

Outcome measures

Outcome measures
Measure	Bortezomib and Rituximab n=26 Participants bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Overall Response Rate of Bortezomib and Rituximab (VR) in Patients With Previously Untreated Waldenstrom's Macroglobulinemia (WM)	23 Participants

SECONDARY outcome

Timeframe: 5 Years

Population: All enrolled participants.

Time to progresion is the defined as the time from study entry to disease progression (PD) or death. Patients without PD are censored at the date of last disease evaluation. PD is defined as a greater than 25% increase in serum monoclonal IgM electrophoresis confirmed by a second measurement at least 2 weeks apart, or progression of clinically significant findings due to disease or symptoms attributable to WM.

Outcome measures

Outcome measures
Measure	Bortezomib and Rituximab n=63 Participants bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Time to Progression in Patients With WM	1.6 years Interval 1.1 to 5.0

SECONDARY outcome

Timeframe: 5 Years

Population: All participants enrolled

Time from documentation of first response to progressive disease.

Outcome measures

Outcome measures
Measure	Bortezomib and Rituximab n=63 Participants bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Duration of Response in Patients With WM	1.4 years Interval 0.6 to 5.0

Adverse Events

Bortezomib and Rituximab

Serious events: 11 serious events

Other events: 63 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Bortezomib and Rituximab n=63 participants at risk bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
Infections and infestations Infection	1.6% 1/63 • Number of events 1
Blood and lymphatic system disorders Anemia	1.6% 1/63 • Number of events 1
Blood and lymphatic system disorders Leukopenia	1.6% 1/63 • Number of events 1
Blood and lymphatic system disorders Neutropenia	6.3% 4/63 • Number of events 4
Blood and lymphatic system disorders Thrombocytopenia	1.6% 1/63 • Number of events 1
Blood and lymphatic system disorders Lymphopenia	4.8% 3/63 • Number of events 3

Other adverse events

Other adverse events
Measure	Bortezomib and Rituximab n=63 participants at risk bortezomib and rituximab Bortezomib: Once weekly for 3 weeks Rituximab: Intravenously once a week for the first and fourth weeks of a cycle
General disorders Edema limb	11.1% 7/63 • Number of events 7
Respiratory, thoracic and mediastinal disorders Cough	6.3% 4/63 • Number of events 4
Respiratory, thoracic and mediastinal disorders Dyspnea	7.9% 5/63 • Number of events 5
Infections and infestations Upper airway infection	6.3% 4/63 • Number of events 4
Musculoskeletal and connective tissue disorders Joint pain	9.5% 6/63 • Number of events 6
Musculoskeletal and connective tissue disorders Muscle pain	7.9% 5/63 • Number of events 5
Immune system disorders Allergic reaction	31.7% 20/63 • Number of events 20
Nervous system disorders Dizziness	11.1% 7/63 • Number of events 7
Nervous system disorders Neuropathy-sensory	50.8% 32/63 • Number of events 32
Investigations ALT elevation	9.5% 6/63 • Number of events 6
Investigations AST elevation	17.5% 11/63 • Number of events 11
Investigations Alkaline phosphatase elevation	11.1% 7/63 • Number of events 7
Metabolism and nutrition disorders Hyperglycemia	44.4% 28/63 • Number of events 28
Metabolism and nutrition disorders Hypoalbuminemia	6.3% 4/63 • Number of events 4
Metabolism and nutrition disorders Hyponatremia	9.5% 6/63 • Number of events 6
Metabolism and nutrition disorders Anorexia	6.3% 4/63 • Number of events 4
Gastrointestinal disorders Constipation	14.3% 9/63 • Number of events 9
Gastrointestinal disorders Diarrhea	39.7% 25/63 • Number of events 25
Gastrointestinal disorders Nausea	39.7% 25/63 • Number of events 25
Gastrointestinal disorders Vomiting	14.3% 9/63 • Number of events 9
General disorders Fatigue	69.8% 44/63 • Number of events 44
General disorders Fever without neutropenia	9.5% 6/63 • Number of events 6
Investigations Weight loss	6.3% 4/63 • Number of events 4
Blood and lymphatic system disorders Anemia	87.3% 55/63 • Number of events 55
Blood and lymphatic system disorders Leukopenia	57.1% 36/63 • Number of events 36
Blood and lymphatic system disorders Neutropenia	44.4% 28/63 • Number of events 28
Blood and lymphatic system disorders Thrombocytopenia	44.4% 28/63 • Number of events 28

Additional Information

Irene M. Ghobrial

Dana-Farber Cancer Institute

Phone: (617) 632-4218

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60