Pacritinib + Standard Care for Blood Cancers

Overseen By Anna Halpern, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Washington

Must be taking: Hypomethylating agents

Must not be taking: Strong CYP3A4 inhibitors

Disqualifiers: Active infection, Pregnancy, 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: 7

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether adding the drug pacritinib to standard treatments, azacitidine or decitabine, can help more patients with certain aggressive blood cancers (myeloproliferative neoplasms) qualify for a stem cell transplant. Pacritinib may block enzymes that cancer cells need to grow, while azacitidine and decitabine assist the body in producing normal blood cells. This study may suit individuals diagnosed with myeloproliferative neoplasms that have progressed to a more severe stage (with 5% or more abnormal cells in the blood or bone marrow) and are considered potential candidates for a stem cell transplant. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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

The trial requires that you do not use strong CYP3A4 inhibitors or inducers when you start the study drugs, and you may need to stop these medications at least 5 half-lives before starting. Other medications are not specifically mentioned, so it's best to discuss your current medications with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that pacritinib has been studied for its safety in treating blood cancer. One study found that serious tumors occurred at similar rates in patients using pacritinib and those using other treatments, but pacritinib resulted in fewer cases of non-melanoma skin cancers. Another study found that more than half of the side effects with pacritinib occurred within the first month of treatment. These side effects included common issues like stomach problems and blood-related issues. The FDA has already approved pacritinib for other conditions, suggesting it is generally safe. This ongoing research is crucial to ensure its safety and effectiveness when combined with standard treatments like azacitidine or decitabine for blood cancers. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about pacritinib for blood cancers because it targets a specific enzyme called JAK2, which plays a crucial role in the growth of these cancer cells. Unlike standard treatments like azacitidine or decitabine, which generally aim to kill rapidly dividing cells, pacritinib works by selectively inhibiting this enzyme, potentially leading to fewer side effects. Additionally, pacritinib is taken orally, making it a more convenient option compared to intravenous treatments, enhancing patient comfort and adherence.

What evidence suggests that this trial's treatments could be effective for blood cancers?

Research has shown that pacritinib can help treat certain blood cancers. Studies have found it can reduce spleen size and improve symptoms in people with myelofibrosis, a type of blood cancer. Specifically, 23% of patients experienced a significant decrease in spleen size, and 31% reported a major improvement in symptoms. Pacritinib blocks enzymes that cancer cells need to grow, helping to manage the disease. This trial tests pacritinib in combination with standard care HMA bridge therapy to determine if it can enable more patients to receive stem cell transplants, potentially leading to better outcomes. ^{6 7 8 9 10}

Who Is on the Research Team?

Anna Halpern, MD

Principal Investigator

Fred Hutch/University of Washington Cancer Consortium

Are You a Good Fit for This Trial?

This trial is for patients with advanced myeloproliferative neoplasms, specifically those in accelerated or blast phase, who are candidates for a stem cell transplant. Participants must be able to receive standard treatments azacitidine or decitabine.

Inclusion Criteria

I am 18 years old or older.

Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) < 3 x upper limits of normal (exceptions for Gilbert's disease, hemolysis, or MPN disease) (assessed within 14 days of study day 1)

Women of child-bearing potential and men must agree to use a highly effective method of contraception

Exclusion Criteria

I am not pregnant or breastfeeding.

Known hypersensitivity to any study drug

Treatment with any other anti-MDS/leukemia investigational agent within 2 weeks of start of study drugs

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 orally twice daily on days 1-28 of each cycle, starting 7 days before or 30 days after standard of care hypomethylating agent bridge therapy. Cycles repeat every 28 days for 6 cycles.

24 weeks

6 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment completion, with periodic follow-up for up to 5 years.

5 years

Periodic visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Azacitidine
• Decitabine
• Pacritinib

Trial Overview The study is testing if adding pacritinib to usual treatments (azacitidine or decitabine) helps more patients qualify for a stem cell transplant. It examines the effects of enzyme-blocking on cancer cells and how well these drugs help produce healthy blood cells.

How Is the Trial Designed?

Group I: Treatment (pacritinib and HMA bridge therapy) Experimental Treatment 8 Interventions

Patients receive pacritinib PO BID on days 1-28 of each cycle, starting 7 days before or 30 days after standard of care HMA bridge therapy. Cycles repeat every 28 days for 6 cycles. Patients receive HMA bridge therapy per treating physician's standard institutional practice with azacitidine IV or SC, or decitabine IV or cedazuridine/decitabine PO per standard of care. Treatment is given in the absence of unacceptable toxicity. Patents also undergo bone marrow aspiration and/or biopsy and blood sample collection during screening and as clinically indicated throughout the study.

Who Is Running the Clinical Trial?

University of Washington

Lead Sponsor

Trials

1,858

Recruited

2,023,000+

Swedish Orphan Biovitrum

Industry Sponsor

Trials

103

Recruited

13,300+

Dr. Guido Oelkers

Swedish Orphan Biovitrum

Chief Executive Officer since 2017

PhD in Economics

Dr. Lydia Abad-Franch

Swedish Orphan Biovitrum

Chief Medical Officer since 2023

MD, MBA

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9244834/

Retrospective analysis of pacritinib in patients with ... In this retrospective analysis, the pacritinib group was associated with improved SVR and symptom response compared to BAT in patients with MF and severe ...

2. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/6649/528574/Real-Word-Effectiveness-of-Pacritinib-in-Patients

Real-Word Effectiveness of Pacritinib in Patients with ... Recent post-hoc data from the phase 3 PERSIST-2 study have shown that PAC is associated with spleen volume reduction, symptom benefit, and ...

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.6578

Efficacy of pacritinib in patients with myelofibrosis who ... Conclusions: PAC at full dose demonstrates efficacy for spleen, symptoms, and transfusion response in pts with MF and both thrombocytopenia and ...

4. cancernursingtoday.com cancernursingtoday.com/post/pacritinib-more-effective-than-best-available-treatment-for-myelofibrosis-and-severe-thrombocytopenia

Pacritinib More Effective Than Best Available Treatment for ... A significantly greater percentage of patients treated with pacritinib met efficacy endpoints for SVR35 (23%) compared to patients receiving BAT ...

5. bloodcancerstoday.com bloodcancerstoday.com/post/pacritinib-improves-symptom-relief-in-myelofibrosis-patients-requiring-rbc-transfusions

Pacritinib Improves Symptom Relief in Patients With ... Results showed a significant improvement in symptom response, with 31% of patients on pacritinib achieving ≥50% TSS response compared with 9% ...

6. ascopubs.org ascopubs.org/doi/10.1200/JCO.2022.40.16_suppl.7058

Risk-adjusted safety analysis of pacritinib (PAC) in patients ... Malignant neoplasms occurred at similar rates on PAC and BAT, though rate of non-melanoma skin cancers was lower in pooled PAC (3/100 pt-yrs) ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12218468/

Indirect treatment comparisons of momelotinib vs pacritinib ... These results show that momelotinib had lower odds of key gastrointestinal and hematologic AEs, including grade 3/4 anemia and thrombocytopenia.

8. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1618267/full

a real-world study based on FDA Adverse Event Reporting ... As shown in Figure 2, more than half of the AEs occurred within the first month (n=244, 51.91%) after starting pacritinib treatment. The ...

9. haematologica.org haematologica.org/article/view/haematol.2021.279415

Retrospective analysis of pacritinib in patients with ... These results indicate that pacritinib is a promising treatment for patients with MF who lack safe and effective therapeutic options due to severe ...

10. targetedonc.com targetedonc.com/view/rossetti-reviews-myelofibrosis-risk-stratification-and-outcome-data-for-pacritinib

Rossetti Reviews Myelofibrosis Risk Stratification and ... Rossetti Reviews Myelofibrosis Risk Stratification and Outcome Data for Pacritinib · The patient is a 62-year-old man in Florida. · He presented ...