Pacritinib for Waldenström's Macroglobulinemia

Overseen By Shayna Sarosiek, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Shayna Sarosiek, MD

Must not be taking: CYP3A inhibitors/inducers

Disqualifiers: HIV, Hepatitis B/C, Liver disease, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 19

Duration: 48 months

30 Participants Needed

Trial Summary

What is the purpose of this trial?

This study is being done to examine the safety and effectiveness of pacritinib as a possible treatment for participants with Waldenström macroglobulinemia (WM). The name of the study drug involved in this study is: -Pacritinib (a type of kinase inhibitor)

Research Team

Shayna Sarosiek, MD

Principal Investigator

Dana-Farber Cancer Institute

Eligibility Criteria

This trial is for individuals with Waldenström macroglobulinemia (WM), a rare type of lymphoma, who have seen their disease return or not respond to treatment. Specific eligibility details are not provided, but typically participants must meet certain health standards and may be excluded based on other medical conditions.

Inclusion Criteria

I can take care of myself but might not be able to do heavy physical work.

I have been diagnosed with Waldenström Macroglobulinemia.

Serum IgM level ≥ 2 times the upper limit of normal

Exclusion Criteria

Participants who are pregnant, breast feeding, or planning to become pregnant within specified timeframes

I cannot swallow pills.

My HIV is not under control.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive pacritinib for up to 4 years, with regular assessments and bone marrow biopsies

48 months

In-clinic visits every cycle, bone marrow biopsies at Cycles 6, 12, and yearly

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

Follow-up every 12 weeks

Treatment Details

Interventions

• Pacritinib

Trial Overview The study is testing the safety and effectiveness of pacritinib, a kinase inhibitor drug, in treating WM. It aims to find out if this medication can help patients whose WM has relapsed or is refractory.

Participant Groups

Group I: Pacritinib Experimental Treatment 1 Intervention

30 participants will complete: * Baseline visit with assessments and ECGs * Cycles 1 through 2 (28 days per cycle): --Day 1: Predetermined dose of Pacritinib 2x daily * Cycle 3, 6, 9, 12, and every 3 cycles for 48 cycles total (28 days per cycle): --Day 1: Predetermined dose of Pacritinib 2x daily * Bone marrow biopsies/aspirations at Cycles 6 and 12 and then yearly * End of Treatment visit with CT scan and bone marrow biopsy/aspiration * Follow up: every 12 weeks for 2 years

Who Is Running the Clinical Trial?

Shayna Sarosiek, MD

Lead Sponsor

Trials

2

Recruited

70+

Sobi, Inc.

Industry Sponsor

Trials

14

Recruited

1,000+