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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests pacritinib, a kinase inhibitor, to determine its safety and effectiveness for treating Waldenström macroglobulinemia (WM), a rare blood cancer. The trial targets individuals with symptoms such as fatigue, night sweats, or swollen lymph nodes due to WM. Ideal candidates have WM, have undergone at least two previous treatments, and experience symptoms that disrupt daily life. Participants will take pacritinib twice daily and undergo regular check-ups, including bone marrow tests. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant medical advancements.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but you cannot take certain drugs that affect liver enzymes within 14 days before starting the study drug. It's best to discuss your current medications with the trial team.

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

Previous studies have shown that pacritinib is safe for humans. This medicine, a kinase inhibitor, blocks certain enzymes that help cancer grow.

Participants using pacritinib for various conditions have generally tolerated it well. However, some side effects have been reported. Common ones include diarrhea, nausea, and low blood cell counts. Serious side effects, though less common, might include bleeding or heart problems.

This trial is in Phase 2, indicating that pacritinib has already passed initial safety checks in earlier studies. This suggests it is reasonably safe for further testing, but more information is needed to fully understand its safety. Prospective participants should discuss potential risks with healthcare professionals before joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Waldenström's macroglobulinemia?

Most treatments for Waldenström's Macroglobulinemia, like rituximab and bendamustine, target the cancerous cells more generally. Pacritinib is unique because it specifically inhibits enzymes called Janus kinases (JAKs), which play a key role in the signaling pathways that drive the growth of these cancer cells. This targeted action could lead to more effective control of the disease with potentially fewer side effects. Researchers are excited about Pacritinib because it offers a novel approach that might enhance treatment effectiveness and improve the quality of life for patients with this condition.

What evidence suggests that pacritinib might be an effective treatment for Waldenström macroglobulinemia?

Research has shown that pacritinib, the investigational treatment in this trial, may help treat Waldenström's macroglobulinemia (WM). Studies have found that pacritinib blocks key signals that aid cancer cell survival, leading to increased cancer cell death, especially in those with the MYD88 mutation. Early results suggest that pacritinib effectively targets the mechanisms that allow these cancer cells to survive and grow. This makes it a promising option for people with WM, particularly those whose cancer has these mutations. ^{1 2 3 6 7}

Who Is on the Research Team?

Shayna Sarosiek, MD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06986174

NCT06986174 | A Phase 2 Study to Evaluate the Safety ... This study is being done to examine the safety and effectiveness of pacritinib as a possible treatment for participants with Waldenström macroglobulinemia ...

2. dana-farber.org dana-farber.org/clinical-trials/24-660

A Phase 2 Study to evaluate the safety and efficacy of ... This study is being done to examine the safety and effectiveness of pacritinib as a possible treatment for participants with Waldenström macroglobulinemia ...

3. centerwatch.com centerwatch.com/clinical-trials/listings/NCT06986174/a-phase-2-study-to-evaluate-the-safety-and-efficacy-of-pacritinib-in-relapsed-or-refractory-waldenstrom-macroglobulinemia

A Phase 2 Study to Evaluate the Safety and Efficacy of ... This study is being done to examine the safety and effectiveness of pacritinib as a possible treatment for participants with Waldenström ...

4. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/5830/527699/Pacritinib-Effectively-Inhibits-Pro-Survival

Pacritinib Effectively Inhibits Pro-Survival Signaling and ... Our studies provide a framework for the investigation of pacritinib in MYD88-mutated lymphomas. Based on these findings, a phase II clinical ...

5. waldenstroms.com waldenstroms.com/images/news/ASH-2023/SLiu.pdf

Pacritinib Blocks Key Pro-survival Signaling Related to ... Pacritinib demonstrates higher levels of apoptotic activity for patient bone marrow malignant B cells. Our studies provide a framework for investigating.

6. withpower.com withpower.com/trial/pacritinib-for-waldenstrms-macroglobulinemia-2e7ee

Pacritinib for Waldenström's Macroglobulinemia This study is being done to examine the safety and effectiveness of pacritinib as a possible treatment for participants with Waldenström macroglobulinemia (WM).

7. go.drugbank.com go.drugbank.com/indications/DBCOND0031072/clinical_trials/DB11697?phase=2&status=not_yet_recruiting

Lymphoplasmacytic Lymphoma Not Yet Recruiting Phase 2 ... A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia, Treatment. Pacritinib (DB11697) ...