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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests pomalidomide, a drug that might help the immune system combat Waldenstrom's macroglobulinemia, a type of blood cancer. The study aims to determine the optimal dose and understand any side effects, specifically for patients whose condition has relapsed or is refractory to previous therapies. Ideal candidates for this trial are those with Waldenstrom's macroglobulinemia not improving with current treatment. Participants will take pomalidomide in cycles and must adhere to a specific visit schedule and take aspirin daily. As a Phase 1 trial, this research seeks to understand how pomalidomide works in people, offering participants the chance to be among the first to receive this treatment.

Will I have to stop taking my current medications?

The trial requires that all previous cancer treatments, including radiation, hormonal therapy, and surgery, must be stopped at least 4 weeks before starting the study. However, it does not specify about other medications, so it's best to discuss your current medications with the trial team.

Is there any evidence suggesting that pomalidomide is likely to be safe for humans?

Research has shown that pomalidomide has been tested for safety in various situations. In past studies, patients generally tolerated pomalidomide well. Common side effects include tiredness, low blood cell counts, and constipation, which can be managed with proper care.

Pomalidomide is already approved for treating multiple myeloma, a type of blood cancer. This approval indicates a certain level of safety for its use in people, although different conditions may respond differently.

While more information is needed to fully understand its safety for Waldenstrom's macroglobulinemia, early studies suggest it can be used safely with careful monitoring. As this trial is in its early stages, it aims to find the best dose and closely monitor any side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Pomalidomide is unique because it offers a fresh approach to treating Waldenstrom's Macroglobulinemia by modulating the immune system. Unlike the standard treatments, such as chemotherapy or monoclonal antibodies, pomalidomide works by enhancing immune responses and directly affecting the tumor environment. Researchers are excited about its potential to improve outcomes with potentially fewer side effects, offering a new hope for patients with this rare type of cancer.

What evidence suggests that pomalidomide might be an effective treatment for Waldenstrom macroglobulinemia?

Research has shown that pomalidomide, the treatment under study in this trial, might benefit patients with relapsed or hard-to-treat Waldenstrom's macroglobulinemia (WM). Early results suggest it can enhance the immune system and inhibit cancer cell growth. In studies, pomalidomide has controlled the disease in patients unresponsive to other treatments. Some patients experienced a decrease in cancer symptoms, indicating that pomalidomide could be a helpful option. While more research is needed to confirm its effectiveness, these early findings offer promise for those with challenging cases of WM. ^{2 6 7 8 9}

Who Is on the Research Team?

Sheeba Thomas, MD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT01198067

Study Details | NCT01198067 | Pomalidomide in Treating ... To evaluate the efficacy of pomalidomide in patients with relapsed or refractory Waldenstrom macroglobulinemia. OUTLINE: This is a dose ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12234261/

Phase I study of pomalidomide in relapsed or refractory ... Data from all subjects who received at least one cycle of therapy were used in the analysis of drug efficacy. All subjects who took at least one dose of ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT01198067?term=AREA%5BBasicSearch%5D(waldenstrom)&rank=2

Pomalidomide in Treating Patients With Relapsed or Refractory ... Submission of false or misleading clinical trial information; Failure to submit primary and secondary outcomes. Correction: Shown when the FDA confirms that the ...

4. researchgate.net researchgate.net/publication/391708767_Phase_I_study_of_pomalidomide_in_relapsed_or_refractory_Waldenstrom_macroglobulinaemia

Phase I study of pomalidomide in relapsed or refractory ... The authors report the final results from a multicenter, prospective study examining FCR in Waldenstrom macroglobulinemia (WM). Forty-three ...

5. iwmf.com iwmf.com/wp-content/uploads/2020/10/Martin_2014.pdf

Next Generation Treatments Trials with pomalidomide. • Pomalidomide for relapsed/refractory WM. • MDACC. • Active, not recruiting. • Pomalidomide, dexamethasone, rituximab for previously ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT01198067

Study Details | NCT01198067 | Pomalidomide in Treating ... This phase I trial studies the side effects and best dose of pomalidomide in treating patients with Waldenstrom macroglobulinemia that has ...

7. packageinserts.bms.com packageinserts.bms.com/sds/sds_pomalyst.pdf

Pomalyst Safety Data Sheet Although SPM rates were low overall, the data suggest that a potential for pomalidomide to increase neoplasms cannot be excluded if accidental exposure occurs.

8. dana-farber.org dana-farber.org/clinical-trials/10-007

Phase I Study of Pomalidomide, Dexamethasone and ... Pomalidomide is a newly discovered drug that may stop cancer cells from growing abnormally. Pomalidomide may also stimulate the immune system to fight the ...

9. go.drugbank.com go.drugbank.com/drugs/DB08910

Pomalidomide: Uses, Interactions, Mechanism of Action Pomalidomide is indicated for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and have demonstrated disease ...