A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia
- Participants will be given study medication-dosing diaries for each treatment cycle.
Each treatment cycle lasts 28 days. Participants will receive 6 cycles of the study
treatment with perifosine and those 6 cycles will be given one after another.
- Medication will be taken orally once a day with food before going to bed and the dose
may be reduced or the schedule changed in the event that the participant experiences
- On Day one of each cycle, routine blood tests and exams will be performed.
- At the end of cycle 2 the following procedures will be performed; history and physical
exam, routine blood tests, bone marrow aspirate and biopsy and CT scan.
- At the end of cycle 6 (end of study) the following procedures will be performed;
routine blood tests, history and physical exam, bone marrow biopsy and aspirate,
research bone marrow and blood samples and CT scan.
- Participants will be in this study for about 6 months and be followed every three years
for 2 more years. They may continue the study treatment indefinitely if there is no
disease progression at the discretion of the treating investigator.
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Response Rate Defined as Minimal, Partial or Complete Response in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia Receiving Daily Perifosine at 150mg Orally.
Participants will be formally evaluated for response after the second cycle and at each subsequent cycle using the criteria from the Second International Workshop on Waldenstrom's Macroglobulinemia. Response was determined through review of routine blood tests, serum protein electrophoresis with quantitative M-spike, quantitative IgM level, and serum free light chain as well as tumor measurements by CT scan and bone marrow aspirate/biopsy.
participants were followed for response for the duration of the study, approximately 2 years
Irene Ghobrial, MD
Dana-Farber Cancer Institute
United States: Institutional Review Board
|Dana-Farber Cancer Institute||Boston, Massachusetts 02115|