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A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia

This study has been completed.
Sponsor:
Collaborator:
Dana-Farber Cancer Institute
Information provided by (Responsible Party):
AEterna Zentaris
ClinicalTrials.gov Identifier:
NCT00398710
First received: November 9, 2006
Last updated: March 4, 2013
Last verified: November 2011
  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 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/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 Waldenström's Macroglobulinemia

Resource links provided by NLM:


Further study details as provided by AEterna Zentaris:

Primary Outcome Measures:
  • Response rate [ Time Frame: Every 4 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Toxicities [ Time Frame: Every 4 weeks ] [ Designated as safety issue: No ]
  • Time to progression [ Time Frame: Every 4 weeks ] [ Designated as safety issue: No ]
  • Progression free survival [ Time Frame: Every 4 weeks ] [ Designated as safety issue: No ]
  • Duration of response [ Time Frame: Every 4 weeks ] [ Designated as safety issue: No ]

Enrollment: 37
Study Start Date: October 2006
Study Completion Date: October 2011
Primary Completion Date: August 2011 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Perifosine
    100 - 150 mg daily
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
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: NCT00398710

Locations
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
AEterna Zentaris
Dana-Farber Cancer Institute
Investigators
Study Chair: Irene M Ghobrial, MD Dana-Farber Cancer Institute
  More Information

Publications:
Responsible Party: AEterna Zentaris
ClinicalTrials.gov Identifier: NCT00398710     History of Changes
Other Study ID Numbers: Perifosine 221
Study First Received: November 9, 2006
Last Updated: March 4, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by AEterna Zentaris:
Waldenström's Macroglobulinemia
Perifosine

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

ClinicalTrials.gov processed this record on November 20, 2014