Brief Title
A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia
Official Title
A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia
Brief 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.
Detailed Description
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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Response rate
Secondary Outcome
Safety
Condition
Waldenström's Macroglobulinemia
Intervention
Perifosine
Study Arms / Comparison Groups
Perifosine
Description: Patients will receive perifosine orally at 150 mg daily after food for 28-d cycles.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
37
Start Date
October 2006
Completion Date
October 2011
Primary Completion Date
August 2011
Eligibility 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.
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Irene M Ghobrial, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00398710
Organization ID
Perifosine 221
Responsible Party
Sponsor
Study Sponsor
AEterna Zentaris
Collaborators
Dana-Farber Cancer Institute
Study Sponsor
Irene M Ghobrial, MD, Study Chair, Dana-Farber Cancer Institute
Verification Date
November 2011