Trial Outcomes & Findings for Simvastatin in Waldenstrom's Macroglobulinemia (NCT NCT00575965)
NCT ID: NCT00575965
Last Updated: 2015-12-16
Results Overview
Objective response is defined as achieving partial response or better on therapy based on the Consensus Panel Recommendations from the 2nd and 3rd International Workshop on WM \[Weber et al, 2003; Kimby et al, 2005\]. Complete Response (CR): Complete disappearance of serum monoclonal (SM) Immunoglobulin (Ig) E (IgE), measured centrally; resolution of adenopathy/organomegaly upon physical exam and computerized tomography (CT) scan; lymph nodes =\<1.5 centimeters; absence of malignant cell by bone marrow histologic examination. Partial Response (PR): a \>=50% reduction from baseline in the SM IgM concentration. Minor Response (MR): \>=25%, but a \<50% reduction of SM IgM from baseline.
TERMINATED
PHASE2
18 participants
Assessed at month 1 and 3 and thereafter every 3 months while on therapy. Median duration on treatment was 6 months (range 1-24 months).
2015-12-16
Participant Flow
Outpatient Clinic at Dana Farber Cancer Institute
Patients with progressive nonsymptomatic disease on watch and wait were enrolled.
Participant milestones
| Measure |
Simvastatin
Single arm, phase II study. All 18 patients received study drug.
|
|---|---|
|
Overall Study
STARTED
|
18
|
|
Overall Study
COMPLETED
|
18
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Simvastatin in Waldenstrom's Macroglobulinemia
Baseline characteristics by cohort
| Measure |
Simvastatin
n=18 Participants
Single arm, phase II study. All 18 patients received study drug.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=5 Participants
|
|
Age, Continuous
|
66 years
STANDARD_DEVIATION 13 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
18 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Assessed at month 1 and 3 and thereafter every 3 months while on therapy. Median duration on treatment was 6 months (range 1-24 months).Population: The analysis dataset is comprised of all evaluable patients. One patient was lost to follow-up within 3 weeks of enrollment and was unevaluable.
Objective response is defined as achieving partial response or better on therapy based on the Consensus Panel Recommendations from the 2nd and 3rd International Workshop on WM \[Weber et al, 2003; Kimby et al, 2005\]. Complete Response (CR): Complete disappearance of serum monoclonal (SM) Immunoglobulin (Ig) E (IgE), measured centrally; resolution of adenopathy/organomegaly upon physical exam and computerized tomography (CT) scan; lymph nodes =\<1.5 centimeters; absence of malignant cell by bone marrow histologic examination. Partial Response (PR): a \>=50% reduction from baseline in the SM IgM concentration. Minor Response (MR): \>=25%, but a \<50% reduction of SM IgM from baseline.
Outcome measures
| Measure |
Simvastatin
n=17 Participants
Simvastatin at 20 mg daily for the first week, then dose escalated weekly by 20 mg a day to a maximum of 80 mg daily by week 4. Patients were maintained on therapy until progression or up to 2 years.
|
|---|---|
|
Objective Response Rate
|
0.00 proportion of patients
|
PRIMARY outcome
Timeframe: Assessed at month 1 and 3 and thereafter every 3 months while on therapy; Assessed every 6 months for up to 2 years of follow-up. Median follow-up in this study cohort was 6 months (range 2-18 months).Population: The analysis dataset is comprised of all evaluable patients. One patient was lost to follow-up within 3 weeks of enrollment and was unevaluable.
Progression-free survival is the defined as the time from study entry to disease progression (PD) or death based on Kaplan-Meier estimates. Patients alibe without PD are censored at the date of last disease evaluation. PD is defined as a greater than 25% increase in serum IgM monoclonal protein levels from the lowest attained response value as determined by serum electrophoresis, confirmed by at least one other investigation, or progression of clinically significant disease related symptom(s). \[Consensus panel criteria: Weber et al, 2003; Kimby et al, 2005\].
Outcome measures
| Measure |
Simvastatin
n=17 Participants
Simvastatin at 20 mg daily for the first week, then dose escalated weekly by 20 mg a day to a maximum of 80 mg daily by week 4. Patients were maintained on therapy until progression or up to 2 years.
|
|---|---|
|
Progression-Free Survival
|
6 months
Interval 2.45 to 9.55
|
Adverse Events
Simvastatin
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Simvastatin
n=17 participants at risk
Simvastatin at 20 mg daily for the first week, then dose escalated weekly by 20 mg a day to a maximum of 80 mg daily by week 4. Patients were maintained on therapy until progression or up to 2 years.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Elevated CPK
|
5.9%
1/17 • Number of events 1 • Assessed every 3 months on therapy up to day 30 post-therapy. Median duration on treatment was 6 months (range 1-24 months).
The analysis dataset is comprised of all evaluable patients. One patient was lost to follow-up within 3 weeks of enrollment and was unevaluable.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
17.6%
3/17 • Number of events 3 • Assessed every 3 months on therapy up to day 30 post-therapy. Median duration on treatment was 6 months (range 1-24 months).
The analysis dataset is comprised of all evaluable patients. One patient was lost to follow-up within 3 weeks of enrollment and was unevaluable.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place