Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Irene Ghobrial, MD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier:
NCT00422656
First received: January 12, 2007
Last updated: October 16, 2013
Last verified: October 2013
  Purpose

The purpose of this research study is to assess the response rate of perifosine in patients with relapsed or refractory Waldenstrom's macroglobulinemia. Perifosine is a newly discovered compound that kills Waldenstrom cells in laboratory experiments. Perifosine has been used in research for the treatment of other types of cancer such as multiple myeloma.


Condition Intervention Phase
Waldenstrom's Macroglobulinemia
Drug: Perifosine
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia

Resource links provided by NLM:


Further study details as provided by Dana-Farber Cancer Institute:

Primary Outcome Measures:
  • Response Rate Defined as Minimal, Partial or Complete Response in Patients With Relapsed or Refractory Waldenstrom's Macroglobulinemia Receiving Daily Perifosine at 150mg Orally. [ Time Frame: participants were followed for response for the duration of the study, approximately 2 years ] [ Designated as safety issue: No ]
    Participants will be formally evaluated for response after the second cycle and at each subsequent cycle using the criteria from the Second International Workshop on Waldenstrom's Macroglobulinemia. Response was determined through review of routine blood tests, serum protein electrophoresis with quantitative M-spike, quantitative IgM level, and serum free light chain as well as tumor measurements by CT scan and bone marrow aspirate/biopsy.


Secondary Outcome Measures:
  • To Evaluate the Toxicity of Perifosine in Patients With WM [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
  • to Evaluate the Time to Progression in Patients With WM [ Time Frame: 5 years ] [ Designated as safety issue: No ]
  • to Obtain Correlative Data in Patients With WM Treated With Perifosine [ Time Frame: 5 years ] [ Designated as safety issue: No ]
  • to Assess PFS and Duration of Response. [ Time Frame: 5 years ] [ Designated as safety issue: No ]

Enrollment: 37
Study Start Date: September 2006
Study Completion Date: November 2012
Primary Completion Date: June 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Perifosine
This is a one armed study, all participants receive perifosine.
Drug: Perifosine
Taken orally every night for 6 back to back 28 day cycles. Participant may continue on treatment if there is no disease progression at the discretion of the treating investigator

Detailed Description:
  • Participants will be given study medication-dosing diaries for each treatment cycle. Each treatment cycle lasts 28 days. Participants will receive 6 cycles of the study treatment with perifosine and those 6 cycles will be given one after another.
  • Medication will be taken orally once a day with food before going to bed and the dose may be reduced or the schedule changed in the event that the participant experiences side effects.
  • On Day one of each cycle, routine blood tests and exams will be performed.
  • At the end of cycle 2 the following procedures will be performed; history and physical exam, routine blood tests, bone marrow aspirate and biopsy and CT scan.
  • At the end of cycle 6 (end of study) the following procedures will be performed; routine blood tests, history and physical exam, bone marrow biopsy and aspirate, research bone marrow and blood samples and CT scan.
  • Participants will be in this study for about 6 months and be followed every three years for 2 more years. They may continue the study treatment indefinitely if there is no disease progression at the discretion of the treating investigator.
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00422656

Locations
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Dana-Farber Cancer Institute
Investigators
Principal Investigator: Irene Ghobrial, MD Dana-Farber Cancer Institute
  More Information

Publications:
Responsible Party: Irene Ghobrial, MD, Principal Investigator, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT00422656     History of Changes
Other Study ID Numbers: 06-077
Study First Received: January 12, 2007
Results First Received: June 29, 2011
Last Updated: October 16, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Dana-Farber Cancer Institute:
relapsed Waldenstrom's Macroglobulinemia
refractory Waldenstrom's Macroglobulinemia
WM
perifosine

Additional relevant MeSH terms:
Waldenstrom Macroglobulinemia
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases

ClinicalTrials.gov processed this record on August 28, 2014