Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia
The purpose of this research study is to assess the response rate of perifosine in patients with relapsed or refractory Waldenstrom's macroglobulinemia. Perifosine is a newly discovered compound that kills Waldenstrom cells in laboratory experiments. Perifosine has been used in research for the treatment of other types of cancer such as multiple myeloma.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia|
- Response Rate Defined as Minimal, Partial or Complete Response in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia Receiving Daily Perifosine at 150mg Orally. [ Time Frame: participants were followed for response for the duration of the study, approximately 2 years ] [ Designated as safety issue: No ]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.
- To Evaluate the Toxicity of Perifosine in Patients With WM [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
- to Evaluate the Time to Progression in Patients With WM [ Time Frame: 5 years ] [ Designated as safety issue: No ]
- to Obtain Correlative Data in Patients With WM Treated With Perifosine [ Time Frame: 5 years ] [ Designated as safety issue: No ]
- to Assess PFS and Duration of Response. [ Time Frame: 5 years ] [ Designated as safety issue: No ]
|Study Start Date:||September 2006|
|Study Completion Date:||November 2012|
|Primary Completion Date:||June 2009 (Final data collection date for primary outcome measure)|
This is a one armed study, all participants receive perifosine.
Taken orally every night for 6 back to back 28 day cycles. Participant may continue on treatment if there is no disease progression at the discretion of the treating investigator
- 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 side effects.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00422656
|United States, Massachusetts|
|Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02115|
|Principal Investigator:||Irene Ghobrial, MD||Dana-Farber Cancer Institute|