Clinical trial NCT00575965: Simvastatin in Waldenstrom's Macroglobulinemia
Official title: Phase II Study of Simvastatin in Waldenstrom's Macroglobulinemia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Waldenstrom's Macroglobulinemia
Keywords: simvastatin
Lead sponsor: Dana-Farber Cancer Institute
Sponsor class: OTHER
First posted: 2007-12-18
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This research study seeks to find new ways to treat people with Waldenstrom's Macroglobulinemia (WM). The study is for participants with slow growing WM who otherwise might not need therapy for at least 3-6 months. Simvastatin is a drug approved by the FDA for lowering cholesterol. In test tube studies the study drug appears to have direct anti-cancer effect against WM tumor cells and mast cells.

Detailed description: OBJECTIVES:

To define objective response, time to progression and safety of Simvastatin in Waldenström's Macroglobulinemia.

STATISTICAL DESIGN:

For this phase II study, a single-stage design is used to evaluate the efficacy of Simvastatin. With a target enrollment of 30 participants, the 95% exact confidence bounds surrounding the response estimate will be no wider than +/- 19%.

Eligibility criteria: Inclusion Criteria:

* 18 years of age or older
* Clinicopathological diagnosis of Waldenstrom's macroglobulinemia
* Measurable disease
* Slowly progressing disease not requiring therapy for at least 3-6 months and who do not meet consensus panel criteria for initiation of therapy
* ECOG Performance status of 0 or 1
* Adequate organ function as defined in the protocol
* Patients should agree to avoid grapefruit juice which is a major inhibitor of CYP 3A4

Exclusion Criteria:

* Patients who have had chemotherapy or radiotherapy within 4 weeks prior to entering the study, or those who have not recovered from adverse events due to agents administered more than four weeks earlier
* Patients who have had rituximab within 3 months prior to entering the study
* Patients who have taken any Statin in the past
* Patients who take cyclosporin, danazol, or gemfibrozil will be excluded
* Prior history of rhabdomyolysis
* Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with the study requirements
* Pregnant or breastfeeding women
* HIV-positive
* Patients who take verapamil will be excluded
* Patients with active or history of liver disease
* Patients who consume more than three alcoholic beverages per day

Primary outcomes:
measure: Objective Response Rate
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).
measure: Progression-Free Survival
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).
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
Study officials:
affiliation: Dana-Farber Cancer Institute
name: Steven Treon, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: