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Pomalidomide, Dexamethasone and Rituximab in 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: NCT01078974
Recruitment Status : Terminated (Safety concerns (IgM Flare))
First Posted : March 2, 2010
Results First Posted : July 25, 2016
Last Update Posted : July 25, 2016
Celgene Corporation
Information provided by (Responsible Party):
Steven P. Treon, MD, PhD, Dana-Farber/Brigham and Women's Cancer Center

Brief Summary:
Pomalidomide is a newly discovered drug that may stop cancer cells from growing abnormally. Pomalidomide may also stimulate the immune system to fight the cancer cells and possibly improve the effectiveness of dexamethasone and rituximab to fight the Waldenstrom's Macroglobulinemia (WM) cancer cells. This drug have been used in multiple myeloma and information from these other research studies suggests that Pomalidomide may help to reduce or prevent the growth of cancer cells.

Condition or disease Intervention/treatment Phase
Waldenstrom's Macroglobulinemia Drug: pomalidomide Drug: dexamethasone Drug: rituximab Phase 1

Detailed Description:
  • Participants will be given a study drug-dosing calendar for each treatment cycle. Each treatment cycle lasts 28 days during which time participants will take Pomalidomide orally once a day. Dexamethasone and rituximab will be administered intravenously on weeks 1, 2, 3, 4 and on weeks 12, 13, 14, 15.
  • Since we are looking for the highest dose of Pomalidomide in combination with dexamethasone and rituximab which can be administered safely without severe or unmanageable side effects, not everyone who participates will receive the same dose of the study drug. The dose participants will get will depend on the number of participants who have been enrolled in the study and how well they have tolerated their doses.
  • As long as there is no evidence that the participant's Waldenstrom's Macroglobulinemia has progressed, they can continue to receive Pomalidomide for up to 52 weeks. Participants will be asked to return to the clinic for follow-up tests at least every three months for four years.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Pomalidomide, Dexamethasone and Rituximab (PDR) in Relapsed or Refractory Waldenstrom's Macroglobulinemia
Study Start Date : May 2010
Actual Primary Completion Date : September 2015
Actual Study Completion Date : March 2016

Arm Intervention/treatment
Experimental: pomalidomide, dexamethasone, rituximab

Drug: pomalidomide Taken orally once a day

Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15

Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15

Drug: pomalidomide
Taken orally once a day
Other Names:
  • CC-4047
  • Pomalyst

Drug: dexamethasone
Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Other Name: Decadron

Drug: rituximab
Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Other Name: Rituxan

Primary Outcome Measures :
  1. Maximum Tolerated Dose of Pomalidomide [ Time Frame: 2 years ]
    To determine the MTD of pomalidomide administered orally in patients with Waldenstrom's Macroglobulinemia in combination with dexamethasone and rituximab. Because maximum tolerated dose was not determined due to study termination, the highest dose of pomalidomide administered is presented below.

  2. Tolerability of Pomalidomide [ Time Frame: 2 years ]
    Number of participants with dose limiting toxicities which resulted in being removed from pomalidomide therapy

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

Inclusion Criteria:

  • 18 years of age or older
  • Able to adhere to the study visit schedule and other protocol requirements
  • Clinicopathological diagnosis of Waldenstrom's macroglobulinemia using consensus panel criteria
  • CD20 positive based on any previous performed bone marrow immunohistochemistry or flow cytometric analysis
  • Meet criteria to treat based on consensus panel criteria
  • Patient must have received at least one previous therapy for WM
  • All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study
  • Measurable disease, defined as presence of immunoglobulin M (IgM) paraprotein with a minimum IgM level of 2 times (or greater) the upper limit of each institution's normal value is required
  • ECOG Performance status of 0, 1 or 2
  • Laboratory tests within ranges outlined in the protocol
  • Disease free of prior malignancies for 5 years or more with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix or breast
  • Screening of patients at high risk of HBV or HCV infection
  • Willing and able to take aspirin or alternate prophylactic anticoagulants

Exclusion Criteria:

  • Any serious medical condition, laboratory abnormality, or psychiatric illness
  • Pregnant or lactating females
  • Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
  • Resistance or intolerance to prior rituximab therapy
  • Previous therapy with thalidomide or lenalidomide
  • Known hypersensitivity to thalidomide, lenalidomide or pomalidomide
  • The development of erythema nodosum if characterized by a desquamating rash while taking similar drugs
  • Concurrent use of other anti-cancer agents or treatments
  • History of non-compliance to medical regimens
  • Patients unwilling to or unable to comply with the protocol
  • Known positive for HIV or hepatitis infection
  • Any history of CVA (Cerebral Vascular Accident/stroke) or clots
  • Active DVT or PE that has not been therapeutically anticoagulated
  • NYHA classification III and greater heart failure
  • Any patient that is unable to ingest or process pomalidomide

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): NCT01078974

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United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Steven P. Treon, MD, PhD
Celgene Corporation
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Principal Investigator: Steven P. Treon, MD Dana-Farber Cancer Institute
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Responsible Party: Steven P. Treon, MD, PhD, Principal Investigator, Dana-Farber/Brigham and Women's Cancer Center Identifier: NCT01078974    
Other Study ID Numbers: 10-007
PO-WM-PI-0005 ( Other Identifier: Celgene )
First Posted: March 2, 2010    Key Record Dates
Results First Posted: July 25, 2016
Last Update Posted: July 25, 2016
Last Verified: June 2016
Keywords provided by Steven P. Treon, MD, PhD, Dana-Farber/Brigham and Women's Cancer Center:
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
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Antineoplastic Agents, Immunological