Clinical trial · Interventional
A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Waldenström's Macroglobulinemia | Waldenstrom Macroglobulinemia | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Perifosine | Drug | Perifosine | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Perifosine
- description
- Patients will receive perifosine orally at 150 mg daily after food for 28-d cycles.
- interventionNames
- Drug: Perifosine
Primary outcomes (1)
- measure
- Response rate
- timeFrame
- Every 4 weeks
- description
- Response will include complete remission, partial remission (PR), and minimal response (MR) using serum protein electrophoresis. Response will also be assessed by IgM using nephelometry.
Secondary outcomes (4)
- measure
- Safety
- timeFrame
- Every 4 weeks
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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.
References
Publications (1)
- RESULTGhobrial IM, Roccaro A, Hong F, Weller E, Rubin N, Leduc R, Rourke M, Chuma S, Sacco A, Jia X, Azab F, Azab AK, Rodig S, Warren D, Harris B, Varticovski L, Sportelli P, Leleu X, Anderson KC, Richardson PG. Clinical and translational studies of a phase II trial of the novel oral Akt inhibitor perifosine in relapsed or relapsed/refractory Waldenstrom's macroglobulinemia. Clin Cancer Res. 2010 Feb 1;16(3):1033-41. doi: 10.1158/1078-0432.CCR-09-1837. Epub 2010 Jan 26. PMID 20103671