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A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia
This study is ongoing, but not recruiting participants.
First Received: November 9, 2006   Last Updated: April 4, 2008   History of Changes
Sponsor: Keryx / AOI Pharmaceuticals, Inc.
Collaborator: Dana-Farber Cancer Institute
Information provided by: Keryx / AOI Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00398710
  Purpose

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.


Condition Intervention Phase
Waldenström's Macroglobulinemia
Drug: Perifosine
Phase II

Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study
Official Title: A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia

Resource links provided by NLM:


Further study details as provided by Keryx / AOI Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Response rate [ Time Frame: Every 4 weeks ]

Secondary Outcome Measures:
  • Toxicities [ Time Frame: Every 4 weeks ]
  • Time to progression [ Time Frame: Every 4 weeks ]
  • Progression free survival [ Time Frame: Every 4 weeks ]
  • Duration of response [ Time Frame: Every 4 weeks ]

Estimated Enrollment: 37
Study Start Date: October 2006
Estimated Study Completion Date: March 2009
Estimated Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00398710

Locations
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Keryx / AOI Pharmaceuticals, Inc.
Dana-Farber Cancer Institute
Investigators
Study Chair: Irene M Ghobrial, MD Dana-Farber Cancer Institute
  More Information

No publications provided

Study ID Numbers: Perifosine 221
Study First Received: November 9, 2006
Last Updated: April 4, 2008
ClinicalTrials.gov Identifier: NCT00398710     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by Keryx / AOI Pharmaceuticals, Inc.:
Waldenström's Macroglobulinemia
Perifosine

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

ClinicalTrials.gov processed this record on November 27, 2009