Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia
The purpose of this research study is to determine the safety of RAD001(Everolimus) and the highest dose of this drug that can be given to people safely. RAD001(Everolimus) is a drug that works by preventing cells in the body from growing and dividing. Information from basic and Phase I clinical research studies suggests that RAD001 also may help to prevent tumor growth in people with relapsed or refractory lymphoma.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study of Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia|
- Overall Response Rate of RAD001 in Patients With Previously Untreated WM [ Time Frame: 2 years ] [ Designated as safety issue: No ]
Overall Response = Complete Response + Near Complete Response + Very Good Partial Response + Partial Response + Minor Response Complete Response: resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. A near CR (nCR) is defined as fulfilling all CR criteria in the presence of positive immunofixation test for an IgM paraprotein.
Very Good Partial Response: > 90% reduction in serum IgM levels. Partial Response: > 50% reduction in serum IgM levels. Minor Response: 25-49% reduction in serum IgM levels Progressive Disease: greater than 25% increase in serum IgM level occurs from the lowest attained response value or progression of clinically significant disease related symptom(s).
Stable Disease: < 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
- Safety and Tolerability of RAD001 in Symptomatic Patients With Previously Untreated WM. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
- Time to Progression and Time to Next Therapy With Single Agent RAD001 Therapy in Previously Untreated WM. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
|Study Start Date:||November 2009|
|Estimated Study Completion Date:||July 2018|
|Estimated Primary Completion Date:||July 2016 (Final data collection date for primary outcome measure)|
RAD001, oral, 10 mg, daily
Taken orally once a day
Other Name: Everolimus
- Participants will take RAD001 orally once a day in the morning. Each treatment cycle lasts for four weeks. Participants will receive up to 72 cycles of treatment.
- During each cycle, participants will be asked to visit the clinic for scheduled tests and exams. They will visit the clinic on the first day of each of the first three cycles, and then once every 3 cycles. During the visits, participants will have a physical exam and blood tests.. Participants may also have CT scans of the chest, abdomen and pelvis as well as a bone marrow aspirate and biopsy.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00976248
|United States, Massachusetts|
|Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02115|
|Principal Investigator:||Steven Treon, MD||Dana-Farber Cancer Institute|