FMR1 (Fragile X Messenger Ribonucleoprotein 1)

P2/3, N=163, Completed, Tetra Discovery Partners | Active, not recruiting --> Completed | Trial completion date: Dec 2025 --> Sep 2025

P2, N=125, Completed, University of Alberta | Active, not recruiting --> Completed

Knockdown or pharmacological inhibition of METTL3 sensitized HCC cells to elesclomol-Cu-induced cuproptosis and effectively suppressed HCC xenograft growth in vivo. Clinically, elevated METTL3 expression was associated with poor HCC prognosis, and the protein expression of METTL3 and FDX1 was negatively correlated in HCC tissues. In conclusion, our findings identify an METTL3-mediated m6A regulatory mechanism controlling cuproptosis sensitivity, revealing METTL3 inhibition as a promising therapeutic strategy for HCC.

P2, N=45, Active, not recruiting, Children's Hospital Medical Center, Cincinnati | Recruiting --> Active, not recruiting | Trial completion date: Jun 2025 --> Mar 2026 | Trial primary completion date: Jun 2025 --> Oct 2025

P3, N=215, Completed, Zynerba Pharmaceuticals, Inc. | Trial completion date: May 2025 --> Aug 2025 | Trial primary completion date: May 2025 --> Aug 2025 | Active, not recruiting --> Completed

Comprehensive characterization confirmed normal chromosomal integrity, robust expression of pluripotency-associated markers, and tri-lineage differentiation potential as evidenced by teratoma formation assays. This FXS patient-derived iPSC line provides a unique platform for investigating neurodevelopmental pathophysiology and screening potential therapeutic interventions for intellectual disability associated with FMR1 dysfunction.

Our findings demonstrate that FMR1 promotes gastric cancer cell proliferation, migration, and invasion through c-MYC signaling, suggesting that FMR1 may serve as a potential prognostic biomarker and therapeutic target for gastric cancer.

P=N/A, N=1200, Recruiting, The International Peace Maternity and Child Health Hospital; The International Peace Maternity and Child Health Hospital

P2, N=125, Active, not recruiting, University of Alberta | Recruiting --> Active, not recruiting | Trial completion date: Jun 2026 --> Dec 2025 | Trial primary completion date: Jun 2026 --> Dec 2025

Furthermore, the combination with PD-1 antibodies achieves approximately 80 % suppression of tumor growth. These findings provide new insights into tumor-targeted siRNA delivery and immunotherapy strategies involving FMRP targeting and PD-1 antibodies.

P3, N=171, Completed, Tetra Discovery Partners | Active, not recruiting --> Completed

Recent studies have uncovered novel links between FMR1, metabolic regulation, and tumorigenesis. This review discusses the role of FMR1 in cellular metabolism and its potential involvement in cancer, focusing on glycolysis, mitochondrial metabolism, lipid metabolism, immune cell metabolism, and tumor immune evasion, and as a potential target to enhance immunotherapy, and highlights future research directions to elucidate its mechanistic roles in cancer.