Brief Title
Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia
Official Title
A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia
Brief Summary
Waldenström's Macroglobulinemia (lymphoplasmacytic lymphoma, WM) remains incurable with
limited therapeutic options and notably absent FDA approved therapy with any WM indication.
Therefore, there is a need to identify new therapeutic agents for WM patients both in the
upfront and relapsed/refractory setting. The purpose of this research study is to assess the
efficacy of perifosine in patients with relapsed or refractory WM.
Detailed Description
Perifosine is a drug that in particular inhibits Akt thought to be important for initiation
and progression of malignancies, specifically in lymphomas. In laboratory experiments of WM
and lymphoma cell lines, perifosine has shown to have cytotoxic and anti-proliferative
activity as a single agent. This drug has been used in clinical research studies of other
types of cancers including soft tissue sarcomas, head and neck cancers and prostate cancer.
This study uses a two-stage design to evaluate efficacy of perifosine based on overall
response (OR). The null and alternative OR rates are 20% and 40%. If 4 or more patients
enrolled in the stage one cohort (n=17 patients) achieve OR than accrual will proceed to
stage two (n=20 patients). If fewer than 10 ORs are observed then the regimen will be
considered ineffective.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Overall Response (OR) Rate
Secondary Outcome
Time to Progression (TTP)
Condition
Waldenstrom's Macroglobulinemia
Intervention
Perifosine
Study Arms / Comparison Groups
Perifosine
Description: Patients receive oral perifosine (150 mg) daily each cycle. Cycle duration is 28 days. After cycle 2, response is assessed and patients with stable or responding disease can continue for another 4 cycles or until disease progression (PD). Protocol treatment duration is 6 cycles but patients may receive perifosine maintenance per investigator discretion in absence of PD.
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
September 2006
Completion Date
November 2012
Primary Completion Date
June 2009
Eligibility Criteria
Inclusion Criteria:
- 18 years of age or older
- 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 paraprotein with a minimum
IgM level of equal to or greater than 2 times the ULN and over 10% of
lymphoplasmacytic cells in bone marrow
- ECOG Performance Status 0,1, or 2
- Laboratory values as described in the protocol
- Life expectancy of greater than 12 weeks
Exclusion Criteria:
- Uncontrolled infection
- Other active malignancies
- CNS involvement
- Cytotoxic chemotherapy less than 3 weeks, or biologic therapy less than 2 weeks, or
corticosteroids less than 2 weeks prior to registration.
- Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy
considered investigational
- Pregnant or nursing women
- Known to be HIV positive
- Radiation therapy less than 2 weeks prior to registration
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Irene Ghobrial, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00422656
Organization ID
06-077
Responsible Party
Principal Investigator
Study Sponsor
Dana-Farber Cancer Institute
Study Sponsor
Irene Ghobrial, MD, Principal Investigator, Dana-Farber Cancer Institute
Verification Date
November 2017