P2, N=180, Active, not recruiting, Central Institute of Mental Health, Mannheim | Recruiting --> Active, not recruiting | Trial completion date: Jun 2026 --> Dec 2027 | Trial primary completion date: Jun 2026 --> Dec 2027

P=N/A, N=104, Not yet recruiting, Huashan Hospital Fudan University; Huashan Hospital Fudan University

P3, N=900, Not yet recruiting, LB Pharmaceuticals Inc.

P3, N=456, Not yet recruiting, LB Pharmaceuticals Inc.

P1, N=100, Not yet recruiting, Stanford University

P1, N=48, Completed, Engrail Therapeutics INC | Recruiting --> Completed

P1, N=10, Completed, Engrail Therapeutics INC

P1, N=42, Recruiting, Engrail Therapeutics INC

Notably, pimozide exhibited anti-tumor effects as a monotherapy and in combination with paclitaxel at clinically relevant doses. While tumor volume reduction in the combination group was not statistically greater than that in the monotherapy group, fluorescence immunohistochemistry revealed a marked decrease in undifferentiated tumor cells, indicating enhanced therapeutic effects of combination treatment. Taken together, these findings indicate that pimozide is a promising candidate for repurposing as a novel therapeutic agent against HNSCC.

P2/3, N=42, Recruiting, M.D. Anderson Cancer Center | N=30 --> 42

Our study supports the clinical potential of USP1 inhibitors as a novel therapy for ATL.

Although FLT3 tyrosine kinase inhibitors (TKIs), such as quizartinib (Quiz) and gilteritinib, have improved clinical outcomes, secondary TKD mutations, particularly the gatekeeper mutation F691L, confer significant resistance. Expression of constitutively active STAT5 partially rescued CPZ-induced growth inhibition. These findings suggest that STAT5 suppression is a key mechanism of CPZ's antileukemic activity and support its potential as a therapeutic strategy for FLT3-ITD-positive AML.