A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia
This is a phase II study in relapsed/refractory WM patients treated with perifosine. It is
designed to assess the proportion of overall confirmed responses (CR + PR + MR) using a
two-stage phase II study design to permit early stopping of the trial if there is strong
evidence that the study regimen is inactive. In addition, it will assess toxicity of this
drug in patients with WM. Patients will receive perifosine 150 mg qhs daily. Patients will
be assessed by serum immunoelectrophoresis and IgM level at least every 4 weeks.
Patients will take three 50 mg tablets of perifosine qhs daily (for 28 days cycles) with
food. Patients may need anti-emetics and/or anti-diarrheas. All patients should continue
therapy unless disease progression is documented on two occasions at least 1 week apart.
Patients with progressive disease or who refuse further therapy will be discontinued from
the protocol. Dose modifications for toxicity will be performed.
Standard criteria for evaluation of response in WM recommended by the Second International
WM Workshop will be used in this study.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Every 4 weeks
Irene M Ghobrial, MD
Dana-Farber Cancer Institute
United States: Food and Drug Administration
|Dana-Farber Cancer Institute||Boston, Massachusetts 02115|