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Bortezomib in Treating Patients With Waldenstrom's Macroglobulinemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00045695
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : May 17, 2013
National Cancer Institute (NCI)
Eastern Cooperative Oncology Group
Information provided by (Responsible Party):
National Cancer Institute (NCI) ( NCIC Clinical Trials Group )

Brief Summary:

RATIONALE: Bortezomib may stop the growth of cancer by blocking the enzymes necessary for tumor cell growth.

PURPOSE: Phase II trial to study the effectiveness of bortezomib in treating patients who have untreated or relapsed Waldenstrom's macroglobulinemia.

Condition or disease Intervention/treatment Phase
Lymphoma Drug: bortezomib Phase 2

Detailed Description:


  • Determine the efficacy of bortezomib, in terms of response rate, in patients with previously untreated or relapsed Waldenstrom's macroglobulinemia.
  • Determine the toxicity of this drug in these patients.
  • Determine the time to progression, stable disease duration, and response duration in patients treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive bortezomib IV over 3-5 seconds on days 1, 4, 8, and 11. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Patients are followed at 4 weeks. Patients with complete or partial response or stable disease are followed every 3 months thereafter.

PROJECTED ACCRUAL: A total of 15-25 patients will be accrued for this study within 1.5-2 years.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study Of PS-341 (NSC 681239) In Patients With Untreated Or Relapsed Waldenstrom's Macroglobulinemia
Study Start Date : August 2002
Actual Primary Completion Date : March 2006
Actual Study Completion Date : December 2009

Intervention Details:
  • Drug: bortezomib
    PS-341 bolus intravenous injection twice weekly* for 2 out of every 3 weeks

Primary Outcome Measures :
  1. Response rate [ Time Frame: 4 years ]
    To assess the efficacy (response rate) of PS-341 given as a bolus intravenous injection twice weekly for two out of every 3 weeks in the treatment of a population of patients with previously untreated or relapsed Waldenström's Macroglobulinemia

Secondary Outcome Measures :
  1. Toxicity [ Time Frame: 4 years ]
    To assess the toxicity of PS-341 in patients with Waldenström's Macroglobulinemia as well as time to progression, stable disease duration and, if responses are observed, response duration.

  2. Cytogenetics and genome profiling [ Time Frame: 4 years ]
    To assess bone marrow and peripheral blood for cytogenetics and genome profiling by microarray in patients with Waldenstrom's macroglobulinemia.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of Waldenstrom's macroglobulinemia confirmed by immunofixation or immunoelectrophoresis

    • Newly diagnosed or untreated with IgM ≥ 20 g/L OR
    • Previously treated with IgM ≥ 5 g/L
  • Non-refractory, defined as no disease progression during prior therapy or within 4 weeks of the last dose of most recent prior therapy (12 weeks for rituximab)
  • Must have 1 or more of the following:

    • Symptomatic lymphadenopathy
    • Hepatomegaly and/or splenomegaly
    • Anemia (i.e., hemoglobin < 11.0 g/dL)
    • Hyperviscosity syndrome
  • No other lymphoproliferative disease including transformed aggressive lymphoma



  • 18 and over

Performance status

  • ECOG 0-2

Life expectancy

  • At least 12 weeks


  • See Disease Characteristics
  • Absolute granulocyte count ≥ 1,000/mm^3
  • Platelet count ≥ 50,000/mm^3


  • Bilirubin ≤ 1.5 times upper limit of normal (ULN)
  • AST or ALT ≤ 2.5 times ULN


  • Creatinine ≤ 1.5 times ULN


  • No uncontrolled bacterial, fungal, or viral infection
  • No pre-existing sensory or motor neurotoxicity grade 2 or greater
  • No other prior malignancy except adequately treated nonmelanoma skin cancer, curatively treated carcinoma in situ of the cervix, or other curatively treated solid tumor for which patient has been disease free for at least 5 years
  • No other serious illness or medical condition that would preclude study participation
  • No unreasonable geographical limitations
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception


Biologic therapy

  • See Chemotherapy
  • See Disease Characteristics
  • At least 12 weeks since prior rituximab (for patients who have progressed)
  • At least 24 weeks since prior rituximab (for patients who have not progressed)
  • No prior high-dose chemotherapy and stem cell transplantation
  • No prior radioactive monoclonal antibodies


  • See Disease Characteristics
  • See Biologic therapy
  • At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas or mitomycin)
  • No more than 2 prior chemotherapy regimens

    • The same chemotherapy combination given for first-line and second-line therapy is considered 2 regimens
    • Single-agent rituximab not considered 1 prior regimen
  • No concurrent cytotoxic chemotherapy

Endocrine therapy

  • No concurrent corticosteroids


  • At least 4 weeks since prior radiotherapy (except for low-dose, non- myelosuppressive radiotherapy) and recovered
  • No prior radiotherapy to more than 25% of bone marrow


  • At least 4 weeks since prior major surgery


  • At least 4 weeks since prior plasmapheresis
  • At least 4 weeks since prior investigational anticancer therapy
  • No other concurrent investigational anticancer agents or therapies

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00045695

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United States, Illinois
Hinsdale Hematology Oncology Associates
Hinsdale, Illinois, United States, 60521
United States, Pennsylvania
Abramson Cancer Center at the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104-4283
Canada, Alberta
Tom Baker Cancer Centre - Calgary
Calgary, Alberta, Canada, T2N 4N2
Cross Cancer Institute
Edmonton, Alberta, Canada, T6G 1Z2
Canada, Manitoba
CancerCare Manitoba
Winnipeg, Manitoba, Canada, R3E 0V9
Canada, Nova Scotia
Nova Scotia Cancer Centre at Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, Canada, B3H 1V7
Canada, Ontario
Margaret and Charles Juravinski Cancer Centre
Hamilton, Ontario, Canada, L8V 5C2
Cancer Care Ontario-London Regional Cancer Centre
London, Ontario, Canada, N6A 4L6
Toronto Sunnybrook Regional Cancer Centre
Toronto, Ontario, Canada, M4N 3M5
Princess Margaret Hospital
Toronto, Ontario, Canada, M5G 2M9
Canada, Quebec
Maisonneuve-Rosemont Hospital
Montreal, Quebec, Canada, H1T 2M4
Canada, Saskatchewan
Saskatoon Cancer Centre
Saskatoon, Saskatchewan, Canada, S7N 4H4
Sponsors and Collaborators
NCIC Clinical Trials Group
National Cancer Institute (NCI)
Eastern Cooperative Oncology Group
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Study Chair: Christine I. Chen, MD Princess Margaret Hospital, Canada
Publications of Results:
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Responsible Party: NCIC Clinical Trials Group Identifier: NCT00045695    
Other Study ID Numbers: I152
CDR0000257042 ( Other Identifier: PDQ )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: May 17, 2013
Last Verified: September 2011
Keywords provided by National Cancer Institute (NCI) ( NCIC Clinical Trials Group ):
Waldenstrom macroglobulinemia
Additional relevant MeSH terms:
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Waldenstrom Macroglobulinemia
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Antineoplastic Agents