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

March 12, 2018 updated by: AEterna Zentaris
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

37

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Dana-Farber Cancer Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Perifosine
Patients will receive perifosine orally at 150 mg daily after food for 28-d cycles.
Drug: Perifosine
150 mg daily (100 mg daily in case of dose reduction)
Other Names:
  • D-21266
  • KRX-0401

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Response rate
Time Frame: Every 4 weeks
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.
Every 4 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety
Time Frame: Every 4 weeks
Adverse events will be assessed at each visit and graded according to the National Cancer Institute Common Toxicity Criteria (version 3.0) from the first dose until 30 d after the last dose of perifosine.
Every 4 weeks
Time to progression
Time Frame: Every 4 weeks
This will be calculated using Kaplan-Meier methodology.
Every 4 weeks
Progression free survival
Time Frame: Every 4 weeks
This will be calculated using Kaplan-Meier methodology.
Every 4 weeks
Duration of response
Time Frame: Every 4 weeks
This will be reported among responding patients.
Every 4 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AEterna Zentaris

Collaborators

Dana-Farber Cancer Institute

Investigators

  • Study Chair: Irene M Ghobrial, MD, Dana-Farber Cancer Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2006

Primary Completion (Actual)

August 1, 2011

Study Completion (Actual)

October 1, 2011

Study Registration Dates

First Submitted

November 9, 2006

First Submitted That Met QC Criteria

November 9, 2006

First Posted (Estimate)

November 14, 2006

Study Record Updates

Last Update Posted (Actual)

March 15, 2018

Last Update Submitted That Met QC Criteria

March 12, 2018

Last Verified

November 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Perifosine 221

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Waldenström's Macroglobulinemia

Clinical Trials on Perifosine

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Iran

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe