Pomalidomide for Relapse/Refractory Waldenstrom's

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: NCT01198067
Recruitment Status : Active, not recruiting
First Posted : September 9, 2010
Last Update Posted : April 9, 2018
Celgene Corporation
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:

The goal of this clinical research study is to find the highest tolerable dose of pomalidomide that can be given to patients with relapsed or refractory WM. The safety of this drug will also be studied.

This is an investigational study. Pomalidomide is FDA approved and commercially available for the treatment of certain types of MM. Its use in this study is investigational.

Up to 30 patients will take part in this study. All will be enrolled at MD Anderson.

Condition or disease Intervention/treatment Phase
Lymphoma Myeloma Drug: Pomalidomide Phase 1

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Pomalidomide in Relapsed or Refractory Waldenstrom Macroglobulinemia
Actual Study Start Date : October 2010
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : October 2020

Arm Intervention/treatment
Experimental: Pomalidomide Schedule A
Starting dose 1 mg orally for 28 days
Drug: Pomalidomide
Starting dose level 1 mg by mouth on days 1-28 of a 28 day cycle.
Other Names:
  • Actimid
  • CC-4047
Experimental: Pomalidomide Schedule B
Starting dose level 1 mg orally for 21 days then 7 days rest
Drug: Pomalidomide
Starting dose level 1 mg by mouth on days 1-21 of a 28 day cycle.
Other Names:
  • Actimid
  • CC-4047

Primary Outcome Measures :
  1. Maximum Tolerated Dose (MTD) of Pomalidomide in Participants with Symptomatic Waldenström Macroglobulinemia (WM) [ Time Frame: 28 days ]
    MTD is defined as the highest dose level in which 6 patients have been treated with less than 2 instances of dose limiting toxicity (DLT) defined as any grade >/= 3 toxicity occurring during the first cycle (28 days).

Secondary Outcome Measures :
  1. Efficacy of Pomalidomide in Participants with Symptomatic Waldenström Macroglobulinemia (WM) [ Time Frame: 30 days after last dose of study drug given. ]
    Efficacy of pomalidomide measured as best response to therapy. Results of blood, urine, and CT and/or MRI scan of the chest, abdomen, and pelvis assessed at end of treatment.

Information from the National Library of Medicine

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

Inclusion Criteria:

  1. Understand and voluntarily sign an informed consent form
  2. Age >/= 18 years at the time of signing the informed consent form
  3. Able to adhere to the study visit schedule and other protocol requirements
  4. Waldenström's Macroglobulinemia that has relapsed and/or is refractory to at least one prior line of therapy
  5. All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study
  6. ECOG performance status of </= 2 at study entry
  7. Laboratory test results within these ranges: Serum creatinine </= 2.0 mg/dL; Creatinine clearance >/= 45 ml/min; Total bilirubin </= 3 x Upper Limit of Normal (ULN) or Direct Bilirubin </= 2 x ULN; AST (SGOT) and ALT (SGPT) </= 2 x ULN; Platelet count >/= 20 K/microL; Absolute neutrophil count >/= 500 K/microL
  8. Disease free of prior malignancies for >/= 5 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix or breast
  9. Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 25 mIU/mL within 10 to 14 days prior to and again within 24 hours of starting pomalidomide and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking pomalidomide
  10. Continued from Inclusion #9: FCBP must also agree to ongoing pregnancy testing. Men must agree to practice complete abstinence or agree use a latex condom during sexual contact with a FCBP while participating in the study, during dose interruptions and for at least 90 days following study drug discontinuation, even if they have had a successful vasectomy. All patients must be counseled at a minimum of every 28 days about pregnancy precautions and risks of fetal exposure
  11. Able to take aspirin (325mg) daily as prophylactic anticoagulation (patients intolerant to ASA may use therapeutic dose warfarin or low molecular weight heparin).
  12. All study participants must be registered into the mandatory POMALYST REMS program, and be willing and able to comply with the requirements of the POMALYST REMS program.

Exclusion Criteria:

  1. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form
  2. Pregnant or breast feeding females. (Lactating females must agree not to breast feed while taking pomalidomide or for 28 days after stopping pomalidomide)
  3. Any medical or psychiatric 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
  4. Use of any other experimental drug or therapy within 28 days of the first dose of study drug
  5. Known hypersensitivity to thalidomide or lenalidomide
  6. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs
  7. Any prior use of pomalidomide
  8. Concurrent use of other anti-cancer agents or treatments
  9. Known positive for HIV or acute hepatitis A or acute or chronic active hepatitis B or C
  10. Grade >2 peripheral neuropathy
  11. Neutrophil count <1000K/microL and/or platelet count <100K/microL unless infiltration by Waldenström's Macroglobulinemia equals or exceed 60% of bone marrow cellularity.

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): NCT01198067

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Celgene Corporation
Principal Investigator: Sheeba K. Thomas, MD M.D. Anderson Cancer Center

Additional Information:
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01198067     History of Changes
Other Study ID Numbers: 2009-0972
NCI-2012-01882 ( Registry Identifier: NCI CTRP )
First Posted: September 9, 2010    Key Record Dates
Last Update Posted: April 9, 2018
Last Verified: April 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by M.D. Anderson Cancer Center:
Waldenstrom Macroglobulinemia
B-cell lymphoproliferative disorder
Bone marrow
Lymphoplasmacytic cells
Monoclonal gammopathy

Additional relevant MeSH terms:
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
Immunologic Factors
Physiological Effects of Drugs
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents
Immunosuppressive Agents
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents