Trial Outcomes & Findings for Carfilzomib With or Without Rituximab in the Treatment of Waldenstrom Macroglobulinemia or Marginal Zone Lymphoma (NCT NCT03269552)
NCT ID: NCT03269552
Last Updated: 2020-01-18
Results Overview
Descriptive statistics will be used for baseline characteristics, and responses to treatment.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
Up to 1 year
Results posted on
2020-01-18
Participant Flow
4 participants signed informed consent, met eligibility and continued on to study treatment.
Participant milestones
| Measure |
Treatment (Carfilzomib, Rituximab)
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Treatment (Carfilzomib, Rituximab)
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
Baseline Characteristics
Carfilzomib With or Without Rituximab in the Treatment of Waldenstrom Macroglobulinemia or Marginal Zone Lymphoma
Baseline characteristics by cohort
| Measure |
Treatment (Carfilzomib, Rituximab)
n=4 Participants
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib, receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=99 Participants
|
|
Age, Continuous
|
64.25 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Up to 1 yearPopulation: Participants who were evaluated after cycle 2 day 15 and prior to cycle 3 day 1.
Descriptive statistics will be used for baseline characteristics, and responses to treatment.
Outcome measures
| Measure |
Treatment (Carfilzomib, Rituximab)
n=4 Participants
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Overall Response Rate
Partial response
|
2 Participants
|
|
Overall Response Rate
Minor response
|
1 Participants
|
|
Overall Response Rate
Complete response
|
0 Participants
|
|
Overall Response Rate
Very good partial response
|
0 Participants
|
|
Overall Response Rate
Stable disease
|
1 Participants
|
|
Overall Response Rate
Progressive disease
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearEstimated using Kaplan-Meier analysis.
Outcome measures
| Measure |
Treatment (Carfilzomib, Rituximab)
n=4 Participants
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Overall Survival
|
4 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearEstimated using Kaplan-Meier analysis.
Outcome measures
| Measure |
Treatment (Carfilzomib, Rituximab)
n=3 Participants
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Time to Best Response
|
2 Months
Interval 0.5 to 3.0
|
SECONDARY outcome
Timeframe: Up to 1 yearEstimated using Kaplan-Meier analysis.
Outcome measures
| Measure |
Treatment (Carfilzomib, Rituximab)
n=4 Participants
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Time to Progression
|
8 Months
Standard Error 0.05
|
Adverse Events
Treatment (Carfilzomib, Rituximab)
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Carfilzomib, Rituximab)
n=4 participants at risk
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Blood and lymphatic system disorders
Microangiopathic Hemolytic Anemia
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
Other adverse events
| Measure |
Treatment (Carfilzomib, Rituximab)
n=4 participants at risk
Patients receive carfilzomib IV over 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who fail to achieve at least 25% M-protein reduction for Waldenstrom's macroglobulinemia or partial response for marginal zone lymphoma after 2 courses of carfilzomib will continue to receive carfolzomib for 4 more courses. In addition, they will also receive rituximab IV weekly on days 1, 8, 15, and 22 of course 3 and then monthly on day 1 of courses 4-6 in the absence of disease progression or unacceptable toxicity.
Carfilzomib: Given IV
Laboratory Biomarker Analysis: Correlative studies
Rituximab: Given IV
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Gastrointestinal disorders
Constipation
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
General disorders
Chills
|
50.0%
2/4 • Number of events 3 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
General disorders
Cough
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
General disorders
Fatigue
|
50.0%
2/4 • Number of events 2 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Gastrointestinal disorders
Flatulance
|
50.0%
2/4 • Number of events 3 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
General disorders
Flu Like Symptoms
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
General disorders
Fluid Retention
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
General disorders
Warm Sensation
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • Number of events 2 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Vascular disorders
Hypertension
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Psychiatric disorders
Insomnia
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Gastrointestinal disorders
Nausea
|
50.0%
2/4 • Number of events 2 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Nervous system disorders
Increased Cold Sensitivity
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Investigations
Neutrophil Count Decreased
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
General disorders
Non-Cardiac Chest Pain
|
50.0%
2/4 • Number of events 2 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Investigations
Platelet Count Decreased
|
50.0%
2/4 • Number of events 3 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Renal and urinary disorders
Decreased Urination
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Renal and urinary disorders
Renal Failure
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
1/4 • Number of events 2 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Investigations
White Blood Cell Count Decreased
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
|
Endocrine disorders
Hypothyroidism
|
25.0%
1/4 • Number of events 1 • Adverse events are reported from the time the subject receives their first dose of study drug through 30 days post-last dose of study drug or initiation of a new anti-cancer therapy, whichever occurs first.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place