Pomalidomide in Treating Patients With Relapsed or Refractory Waldenstrom Macroglobulinemia

NCT01198067 | Terminated Phase 1 Results FDA Drug

• 3 other identifiers: 2009-0972NCI-2012-01882NCI-2010-02076
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This phase I trial studies the side effects and best dose of pomalidomide in treating patients with Waldenstrom macroglobulinemia that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). Pomalidomide may stimulate the immune system in different ways and stop cancer cells from growing.

Conditions

Recurrent Waldenstrom Macroglobulinemia; Refractory Waldenstrom Macroglobulinemia

Outcome Measures

Primary Outcomes (1)

• Number of Patients Experiencing MTD

  Maximum Tolerated Dose

  Participants Experiencing Study Medication Maximum Tolerated Dose through Study Completion (Avg. 1 Year)

Secondary Outcomes (3)

• Cycles Completed

  Study Completion (Avg. 1 Year)

• Changes in Waldestrom Biomarkers

  Through Study Completion (Avg. 1 Year)

• Changes in Waldestrom Biomarkers

  Through Study Completion (Avg. 1 Year)

Study Arms (1)

Treatment (pomalidomide)

EXPERIMENTAL

Patients receive pomalidomide PO on days 1-28 or 1-21. Courses repeat every 21 or 28 days in the absence of disease progression or unacceptable toxicity.

Drug: Pomalidomide

Interventions

Pomalidomide DRUG

Given PO

Also known as: 4-Aminothalidomide, Actimid, CC-4047, Imnovid, Pomalyst

Treatment (pomalidomide)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Understand and voluntarily sign an informed consent form
• Able to adhere to the study visit schedule and other protocol requirements
• Waldenstrom's macroglobulinemia that has relapsed and/or is refractory to at least one prior line of therapy
• All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study
• Eastern Cooperative Oncology Group (ECOG) performance status of =\< 2 at study entry
• 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
• Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2 x ULN
• Platelet count \>= 20 K/microL
• Absolute neutrophil count \>= 500 K/microL
• 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
• 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; 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
• Able to take aspirin (325 mg) daily as prophylactic anticoagulation (patients intolerant to acetylsalicylic acid \[ASA\] may use therapeutic dose warfarin or low molecular weight heparin)
• 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

You may not qualify if:

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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Waldenstrom Macroglobulinemia

Interventions

pomalidomide

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Results Point of Contact

• Title: Sheeba K. Thomas, MD
• Organization: M.D. Anderson Cancer Center

sthomas@mdanderson.org

713-792-2860

Study Officials

• Sheeba K Thomas

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2010
• First Posted: September 9, 2010
• Study Start: October 6, 2010
• Primary Completion: May 2, 2025
• Study Completion: May 2, 2025
• Last Updated: September 4, 2025
• Results First Posted: September 4, 2025

Record last verified: 2025-09

Locations

US (1)