Trial Outcomes & Findings for CC-5013 (Lenalidomide) and Rituximab in Waldenstrom's Macroglobulinemia (NCT NCT00142168)

NCT ID: NCT00142168

Last Updated: 2016-04-21

Results Overview

Time to progression is measured as the length in time in months from starting therapy until progression, defined as 25% increase in serum IgM from nadir.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

34.3 months

Results posted on

2016-04-21

Participant Flow

Outpatient clinic at DFCI

Symptomatic WM patients requiring therapy

Participant milestones

Participant milestones
Measure
Lenalidomide and Rituximab
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Overall Study
STARTED
16
Overall Study
COMPLETED
16
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

CC-5013 (Lenalidomide) and Rituximab in Waldenstrom's Macroglobulinemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Lenalidomide and Rituximab
n=16 Participants
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Age, Categorical
<=18 years
0 Participants
n=99 Participants
Age, Categorical
Between 18 and 65 years
10 Participants
n=99 Participants
Age, Categorical
>=65 years
6 Participants
n=99 Participants
Age, Continuous
59 years
STANDARD_DEVIATION 9 • n=99 Participants
Sex: Female, Male
Female
4 Participants
n=99 Participants
Sex: Female, Male
Male
12 Participants
n=99 Participants
Region of Enrollment
United States
16 participants
n=99 Participants

PRIMARY outcome

Timeframe: 34.3 months

Population: All enrolled patients

Time to progression is measured as the length in time in months from starting therapy until progression, defined as 25% increase in serum IgM from nadir.

Outcome measures

Outcome measures
Measure
Lenalidomide and Rituximab
n=16 Participants
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Time to Progression
17.1 months
Interval 2.0 to 34.3

PRIMARY outcome

Timeframe: 34.3 months

Population: Evaluable patients. 4 participants were withdrawn for adverse events and were unevaluable.

Overall response is the total number of participants who respond to therapy. Patients achieving a complete response (CR) will be defined as having achieved resolution of all symptoms, normalization of their serum IgM levels with complete disappearance of their IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly during any point while in this study and normal bone marrow biopsy. Patients achieving a partial response (PR) and a minor response (MR) will be defined as achieving a \> 50% and \> 25% reduction in serum IgM levels, respectively, during any point while in this study. Patients with stable disease (SD) will be defined as having \< 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WMduring any point while in this study.

Outcome measures

Outcome measures
Measure
Lenalidomide and Rituximab
n=12 Participants
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Overall Response
8 participants

SECONDARY outcome

Timeframe: 34.3 months

Population: Evaluable patients. 4 participants were withdrawn for adverse events and were unevaluable.

Major response rate is the number of participants who achieve at a PR or better. A PR or better will be defined as achieving a \>50% reduction in serum IgM levels.

Outcome measures

Outcome measures
Measure
Lenalidomide and Rituximab
n=12 Participants
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Major Response Rate
4 participants

SECONDARY outcome

Timeframe: 34.3 months

Population: Evaluable patients. 4 participants were withdrawn for adverse events and were unevaluable.

A minor response is defined as having achieved \>25% but less than 50% reduction in serum IgM levels.

Outcome measures

Outcome measures
Measure
Lenalidomide and Rituximab
n=12 Participants
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Minor Response Rate
4 participants

Adverse Events

Lenalidomide and Rituximab

Serious events: 11 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Lenalidomide and Rituximab
n=16 participants at risk
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Blood and lymphatic system disorders
Neutropenia
31.2%
5/16 • Number of events 5
Blood and lymphatic system disorders
Anemia
6.2%
1/16 • Number of events 1
Cardiac disorders
arrythmia
6.2%
1/16 • Number of events 1
Cardiac disorders
chest pain
6.2%
1/16 • Number of events 1
Respiratory, thoracic and mediastinal disorders
pulmonitis
6.2%
1/16 • Number of events 1
Blood and lymphatic system disorders
Thrombocytopenia
6.2%
1/16 • Number of events 1
Respiratory, thoracic and mediastinal disorders
pleural effusion
6.2%
1/16 • Number of events 1

Other adverse events

Other adverse events
Measure
Lenalidomide and Rituximab
n=16 participants at risk
Intended therapy consisted of 48 weeks of lenalidomide (25 mg/d for 3 weeks and then 1 week off) along with rituximab (375 mg/m(2)/wk) dosed on weeks 2 to 5 and 13 to 16.
Blood and lymphatic system disorders
Anemia
50.0%
8/16 • Number of events 8

Additional Information

Steven P. Treon MD PhD

DFCI

Phone: 617 632 2681

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place