Pomalidomide for Waldenstrom's Macroglobulinemia

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: M.D. Anderson Cancer Center

Must be taking: Aspirin

Must not be taking: Thalidomide, Lenalidomide

Disqualifiers: HIV, Hepatitis B, Hepatitis C, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 21-28 days per course

15 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Sheeba Thomas, MD

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for adults with Waldenstrom macroglobulinemia that has come back or isn't responding to treatment. They must be fairly active (ECOG <=2), have normal liver function, and not have had cancer treatments in the last 4 weeks. People can't join if they're allergic to similar drugs, pregnant, breastfeeding, HIV positive, or have certain other health issues.

Inclusion Criteria

My Waldenstrom's macroglobulinemia has returned or didn't respond to my first treatment.

Understand and voluntarily sign an informed consent form

I can take aspirin daily or, if allergic, I can use an alternative like warfarin or heparin.

Exclusion Criteria

I have previously used pomalidomide.

I am known to be positive for HIV or hepatitis A, B, or C.

Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

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

21-28 days per course

Follow-up

Participants are monitored for safety and effectiveness after treatment

Every 6 months

Treatment Details

Interventions

• Pomalidomide

Trial Overview The trial is testing Pomalidomide's safety and the best dose for treating relapsed/refractory Waldenstrom macroglobulinemia. It aims to see how well it stimulates the immune system to stop cancer growth.

Participant Groups

Group I: Treatment (pomalidomide) Experimental Treatment 1 Intervention

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.

Pomalidomide is already approved in United States, European Union, Canada, Japan for the following indications:

Approved in United States as Pomalyst for:

• Multiple myeloma
• Kaposi's sarcoma

Approved in European Union as Imnovid for:

• Multiple myeloma
• Kaposi's sarcoma

Approved in Canada as Pomalyst for:

• Multiple myeloma
• Kaposi's sarcoma

Approved in Japan as Imnovid for:

• Multiple myeloma

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+