P2, N=24, Recruiting, Medical University of South Carolina | Trial completion date: Jun 2026 --> Dec 2027 | Trial primary completion date: Feb 2026 --> Dec 2027

PK/PD-guided screening on the NOCS prioritized Paxalisib, which, when combined with the exon skipping approach, demonstrated synergistic anticancer efficacy in patient-derived tumor models. These findings establish a clinically relevant framework that integrates multi-organ PK/PD modeling with genotype-driven therapeutic strategies, highlighting the potential of combining targeted gene correction with small-molecule therapy for personalized treatment. This platform offers broad applicability in precision oncology and drug development across diverse genetic contexts.

P2, N=29, Not yet recruiting, Sabine Mueller, MD, PhD

In the present study, we investigated whether combining reduced-dose 177Lu-CD25 Ab radioimmunotherapy (RIT) with molecularly targeted inhibitors of ALK (crizotinib) or mTOR (everolimus) could enhance antitumor efficacy while minimizing systemic toxicity. These findings demonstrate that targeted inhibition of ALK or mTOR synergizes with CD25-directed 177Lu RIT to enhance therapeutic efficacy without increasing toxicity. This combinatorial approach enables radiation-dose reduction while preserving antitumor potency, supporting further preclinical and translational development of 177Lu-CD25-based combination RIT for ALCL and other CD25-expressing malignancies.

P2, N=46, Active, not recruiting, Emory University | Trial completion date: Jan 2026 --> Jun 2026 | Trial primary completion date: Jan 2026 --> Jun 2026

These pro-tumor effects were partially reversed by pharmacological inhibitor BEZ235 for PI3K/mTOR. In vivo validation using a mouse xenograft model confirmed that PFOS exposure promotes tumor growth and upregulates the same pathway and effector molecules. This study provides integrated clinical and experimental evidence that PFOS exposure at environmentally relevant concentrations promotes PTC progression by inducing PI3K/AKT/mTOR-mediated EMT and associated enzyme secretion, providing crucial experimental evidence for the toxic role of PFOS as an environmental contaminant in thyroid tumors and underscoring the urgent need for enhanced environmental health risk assessment and regulation.

Furthermore, HS inhalation induced an elevation in the concentrations of mammalian targets of rapamycin and nuclear factor erythroid-derived 2-like 2 (Nrf2) expression in the heart, and this effect was significantly potentiated in the OTC + HS group. Despite these molecular and biochemical alterations, no detectable differences in cardiac histology were observed among the experimental groups. Collectively, these findings demonstrate that OTC mitigates HS-induced cardiac injury by reducing oxidative stress, inflammation, DNA damage, and apoptosis through mechanisms that involve inhibition of NLRP3 inflammasome activation and NF-κB signaling, together with activation of sirtuin-1 and Nrf2 pathways.

P=N/A, N=410, Not yet recruiting, Cook Research Incorporated

P1/2, N=38, Not yet recruiting, Second Affiliated Hospital, School of Medicine, Zhejiang University

P2, N=55, Recruiting, Emory University | Trial completion date: Nov 2026 --> Feb 2029 | Trial primary completion date: Nov 2026 --> Feb 2029

P2, N=30, Not yet recruiting, AFT Pharmaceuticals, Ltd.

PRRT combined with everolimus-based immunosuppression has demonstrated controllable safety and preliminary antitumor activity in patients with relapsed NETLM after LT who have been strictly screened, but myelosuppression requires close monitoring and timely symptomatic treatment.