Trial Outcomes & Findings for Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia (NCT NCT00142116)
NCT ID: NCT00142116
Last Updated: 2014-06-02
Results Overview
Response determinations were made using modified consensus panel criteria from the Third International Workshop on WM, and response rates were determined on an evaluable basis. A complete response was defined as having resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. Patients achieving a partial response and a minor response were defined as achieving a more than or equal to 50% and more than or equal to 25% reduction in serum IgM levels, respectively. Patients with stable disease were defined as having less than 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WM. Progressive disease was defined as a greater than 25% increase in serum IgM level occurred from the lowest attained response value or progression of clinically significant disease-related symptom(s).
COMPLETED
PHASE2
25 participants
3 years
2014-06-02
Participant Flow
Patients with Waldenstrom's Macroglobulinemia
A phase II study using thalidomide and rituximab in symptomatic Waldenstrom's macroglobulinemia (WM) patients naïve to either agent.
Participant milestones
| Measure |
All WM Patients
Waldenstrom's Macroglobulinemia Patients
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
COMPLETED
|
25
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia
Baseline characteristics by cohort
| Measure |
All WM Patients
n=25 Participants
Waldenstrom's Macroglobulinemia Patients
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
14 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=99 Participants
|
|
Age, Continuous
|
63 years
STANDARD_DEVIATION 11.2 • n=99 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
25 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 3 yearsPopulation: Intent-to-treat basis
Response determinations were made using modified consensus panel criteria from the Third International Workshop on WM, and response rates were determined on an evaluable basis. A complete response was defined as having resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. Patients achieving a partial response and a minor response were defined as achieving a more than or equal to 50% and more than or equal to 25% reduction in serum IgM levels, respectively. Patients with stable disease were defined as having less than 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WM. Progressive disease was defined as a greater than 25% increase in serum IgM level occurred from the lowest attained response value or progression of clinically significant disease-related symptom(s).
Outcome measures
| Measure |
All WM Patients
n=25 Participants
Waldenstrom's Macroglobulinemia Patients
|
|---|---|
|
Objective Response Rate
Complete Response
|
1 participants
|
|
Objective Response Rate
Partial Response
|
15 participants
|
|
Objective Response Rate
Minor Response
|
2 participants
|
|
Objective Response Rate
Stable Disease
|
7 participants
|
PRIMARY outcome
Timeframe: 49.1 monthsTime to disease progression (TTP) was calculated from the start of therapy using the Kaplan-Meier method.
Outcome measures
| Measure |
All WM Patients
n=25 Participants
Waldenstrom's Macroglobulinemia Patients
|
|---|---|
|
Time to Progression
TTP for all evaluable patients
|
34.8 months
Interval 1.0 to 49.1
|
|
Time to Progression
TTP for responding patients
|
38.7 months
Interval 10.3 to 49.1
|
|
Time to Progression
TTP for previously treated patients
|
15.25 months
Interval 1.0 to 45.8
|
|
Time to Progression
TTP for previously untreated patients
|
36.04 months
Interval 2.5 to 49.1
|
SECONDARY outcome
Timeframe: 3 yearsOutcome measures
Outcome data not reported
Adverse Events
All WM Patients
Serious adverse events
| Measure |
All WM Patients
n=25 participants at risk
Waldenstrom's Macroglobulinemia Patients
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Death
|
4.0%
1/25 • Number of events 1
|
|
Cardiac disorders
Death
|
4.0%
1/25 • Number of events 1
|
Other adverse events
| Measure |
All WM Patients
n=25 participants at risk
Waldenstrom's Macroglobulinemia Patients
|
|---|---|
|
Nervous system disorders
Peripheral Neuropathy
|
44.0%
11/25 • Number of events 11
|
|
Nervous system disorders
Somnolence
|
12.0%
3/25 • Number of events 3
|
|
Nervous system disorders
Confusion
|
12.0%
3/25 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.0%
2/25 • Number of events 2
|
|
Nervous system disorders
Tremors
|
8.0%
2/25 • Number of events 2
|
|
Cardiac disorders
Bradycardia
|
8.0%
2/25 • Number of events 2
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place