A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia

• Status: Completed
• Trial end date: 2011-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AEterna Zentaris
• Collaborator: Dana-Farber Cancer Institute

Criteria

Inclusion Criteria:

• Age >= 18 years.

• Must have received prior therapy for their WM and have relapsed or refractory WM. Any number of prior therapies is acceptable.

• Measurable disease, defined as presence of immunoglobulin M (IgM) paraprotein with a minimum IgM level of > 2 times the upper limit of each institution's normal value is required and over 10% of lymphoplasmacytic cells in the bone marrow.

• ECOG Performance Status (PS) 0, 1, or 2.

• The following laboratory values obtained 14 days prior to registration

• ANC >= 1 x109/L

• PLT >= 75 x109/L

• Total bilirubin ≤ 2.0 mg/dL (If total is elevated check direct and if normal patient is eligible.)

• AST <= 3 x upper limit of normal (ULN)

• Creatinine <= 2 x ULN

• Ability to provide informed consent.

• Life expectancy >= 12 weeks.

Exclusion Criteria:

• Uncontrolled infection.

• Other active malignancies.

• CNS involvement.

• Cytotoxic chemotherapy ≤ 3 weeks, or biologic therapy ≤ 2 weeks, or corticosteroids ≤ 2 weeks, prior to registration. Patients may be receiving chronic corticosteroids if they are being given for disorders other than WM such as auto-immune diseases. Plasmapheresis is not considered as an active therapy and can be used at the physician's discretion.

• Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy considered investigational.

• Any of the following:

• Pregnant women

• Nursing women

• Men or women of childbearing potential who are unwilling to employ adequate contraception (condoms, diaphragm, birth control pills, injections, intrauterine device (IUD), or abstinence, etc.)

• Known to be HIV positive.

• Radiation therapy ≤ 2 weeks prior to registration.