P=N/A, N=61, Completed, CStone Pharmaceuticals | Active, not recruiting --> Completed | Trial completion date: Oct 2025 --> Sep 2024

P1/2, N=380, Not yet recruiting, Shanghai Runshi Pharmaceutical Technology Co., Ltd

P1, N=60, Not yet recruiting, The First Affiliated Hospital of Henan University of Science and Technology; The First Affiliated Hospital of Henan University of Science and Technolo

P2, N=6, Terminated, Medical College of Wisconsin | Trial completion date: May 2027 --> Oct 2024 | Active, not recruiting --> Terminated; After 6 patients were enrolled, it was determined that only patients with STAT5 activation were having a biochemical response to pacritinib treatment.

P1, N=19, Completed, Shanghai Runshi Pharmaceutical Technology Co., Ltd | Not yet recruiting --> Completed

P=N/A, N=39, Recruiting, Jinling Hospital, China

P2, N=36, Recruiting, Deciphera Pharmaceuticals, LLC | Not yet recruiting --> Recruiting

P1, N=10, Not yet recruiting, City of Hope Medical Center

Collectively, our study showed PLX-3397-related efficacy in ameliorating pain linked to the reduction of microglia and neuroinflammation in mice. Furthermore, our research provided new proof-of-concept data supporting the promise of testing PLX-3397 as an analgesic.

P1/2, N=52, Recruiting, Uma Borate | Not yet recruiting --> Recruiting

P2, N=49, Not yet recruiting, University of Miami

P2, N=9, Suspended, University of Utah | Recruiting --> Suspended

P3, N=450, Not yet recruiting, Shanghai Runshi Pharmaceutical Technology Co., Ltd

These results underscore an additional pharmacological benefit of edicotinib against ABCG2 activity, suggesting its potential incorporation into combination therapies for patients with ABCG2-overexpressing tumors. Further research is warranted to validate these findings and explore their clinical implications.

P1, N=24, Enrolling by invitation, Abbisko Therapeutics Co, Ltd | Not yet recruiting --> Enrolling by invitation

In patients with myelofibrosis and platelets ≤ 100 × 109/L, achieving SVR on pacritinib, but not BAT (including ruxolitinib), was associated with significant OS benefit, suggesting that pacritinib may offer a unique survival advantage in patients with myelofibrosis and thrombocytopenia who achieve any SVR. Trial Registration: ClinicalTrials.gov number: NCT02055781.

This literature review, along with clinical expertise and opinion, was used to develop this concise and clinically relevant consensus paper to harmonize the knowledge and clinical practice on GIST management across Canada. The content presented here will help guide healthcare providers, especially in Canada, in terms of approaching and managing GIST.

P2, N=241, Active, not recruiting, Syndax Pharmaceuticals | Trial completion date: Dec 2024 --> Sep 2027

Interestingly, pacritinib, but not other JAK inhibitors such as ruxolitinib, upadacitinib, or filgotinib, acted synergistically with lenvatinib in HCC cells. Immunohistochemical analysis of tumor sections revealed that pacritinib reduced Ki67 staining and phosphorylated IRAK1. Our findings suggest that pacritinib may be a promising therapeutic option for the treatment of advanced HCC, particularly in patients who have developed resistance to lenvatinib.

P1/2, N=88, Completed, Vanderbilt-Ingram Cancer Center | Active, not recruiting --> Completed | Trial completion date: Jul 2025 --> Apr 2024

P2, N=36, Not yet recruiting, Deciphera Pharmaceuticals, LLC

P2, N=15, Not yet recruiting, M.D. Anderson Cancer Center

In this work, additional effort was applied to design new BIBR1532-based analogues with potential inhibitory activity against telomerase and acting as multitarget antitumor candidates to overcome the resistance problem...Compound 4l represented a very promising JAK1 inhibitory potential with a 0.46-fold change, compared to that of pacritinib reference standard (0.33-fold change). Besides, it showed a superior STAT3-inhibitory potential with a 0.22-fold change compared to sorafenib (0.33-fold change). Additionally, compound 4l downregulated TLR4 protein expression by 0.81-fold change compared to that of resatorvid (0.29-fold change)...Remarkably, compound 4l led to prominent reductions in tumor size and mass. Concurrent enhancements in biochemical, hematologic, histopathologic, and immunohistochemical parameters further confirmed the suppression of angiogenesis and inflammation, elucidating additional mechanisms by which compound 4l exerts its anticancer effects.

P2, N=120, Recruiting, Incyte Corporation | Not yet recruiting --> Recruiting

Avapritinib was effective for molecular biologically positive CBF-AML patients with KIT mutation after allo-HSCT. The main adverse reaction was myelosuppression, which could generally be tolerated.

P1/2, N=37, Active, not recruiting, Blueprint Medicines Corporation | Recruiting --> Active, not recruiting | Trial completion date: Apr 2025 --> Dec 2025

P=N/A, N=60, Not yet recruiting, Cancer Institute and Hospital, Chinese Academy of Medical Sciences

P1/2, N=63, Terminated, Nerviano Medical Sciences | N=200 --> 63 | Trial completion date: Feb 2026 --> Aug 2024 | Recruiting --> Terminated | Trial primary completion date: Dec 2025 --> Aug 2024; Study stopped due to strategic reasons. The decision is not based on specific safety findings as the safety observed in the phase II part of the study is in line with what was reported in the phase I part.

P2, N=21, Active, not recruiting, Daiichi Sankyo Co., Ltd. | Recruiting --> Active, not recruiting | Trial completion date: Nov 2026 --> May 2026

P3, N=240, Not yet recruiting, Incyte Corporation

Inhibition of microglia by pretreatment with minocycline or depletion of microglia by PLX3397 abolished the preventive effect of low-dose LPS pre-injection on mSPS-induced anxiety- and fear-like behavior and neuroinflammatory responses. These results suggest that pre-stimulation of microglia may prevent the development of PTSD-like behaviors by attenuating the development of neuroinflammatory responses. This could help to develop new strategies to prevent the damaging effects of harmful stress on the brain.

P1, N=20, Recruiting, Dana-Farber Cancer Institute | Initiation date: Aug 2025 --> Aug 2024

P2, N=65, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting

P1/2, N=37, Not yet recruiting, Cancer Institute and Hospital, Chinese Academy of Medical Sciences

P1/2, N=252, Not yet recruiting, Shanghai Runshi Pharmaceutical Technology Co., Ltd

P1, N=20, Recruiting, Dana-Farber Cancer Institute | Not yet recruiting --> Recruiting

P1, N=42, Active, not recruiting, 3D Medicines (Beijing) Co., Ltd. | Trial completion date: Jun 2024 --> Dec 2024 | Trial primary completion date: Jun 2024 --> Dec 2024

To manipulate microglia, we used low-dose treatment with a highly selective CSF1R inhibitor called PLX5622 (PLX)...Across treatment groups, MRI-detected anatomical volumes at P19 and P63 were associated with microglia and proliferating microglia densities, respectively. Overall, our study demonstrates that low-dose PLX treatment produces a sex-dependent response in juvenile mice, that manipulation of microglia alters CRT-induced volume changes and that microglia density and MRI-derived volume changes are correlated in this model.

P1, N=24, Not yet recruiting, Abbisko Therapeutics Co, Ltd

P3, N=20, Recruiting, Incyte Biosciences Japan GK | Trial completion date: Dec 2025 --> Mar 2027 | Trial primary completion date: Dec 2025 --> Sep 2025

To our knowledge, this is the first known description of the activity of avapritinib in the treatment of a sporadic mesenteric DT, which is relevant given the limited treatment options for patients with this diagnosis. This clinical finding may be worth exploring in a dedicated clinical trial.

In conclusion, targeting FLT3 as a receptor tyrosine kinase with PLX3397 represents a promising therapeutic strategy for improving outcomes in patients with FLT3-mutated AML. Further clinical investigations are warranted to validate the efficacy and safety of PLX3397 and to optimize treatment strategies for AML patients based on the FLT3 mutational status.