First-in-human and multicenter phase I study of OSCA therapy for knee osteoarthritis

cells/ml for all cohorts, and cartilage acellular matrix was 60 mg (40 mg/ml). The primary end point was safety, assessed by dose-limiting toxicities and TEAEs. Secondary end points included Visual Analog Scale (VAS), International Knee Documentation Committee (IKDC), Magnetic Resonance Observation of Cartilage Repair Tissue (MOCART), Whole-Organ Magnetic Resonance Imaging Score (WORMS), and K&L grading over 24 weeks. OSCA was well tolerated, with no dose-limiting toxicities and mild-to-moderate TEAEs in 25.0% (3/12) of patients. At 24 weeks, mid-dose and high-dose groups showed numerically greater improvement in pain and function than the low-dose group (P < 0.05), with up to 91.3% reduction in VAS (P = 0.002) and 102.7% IKDC (P = 0.005). MRI outcomes showed efficacy signals: MOCART total scores improved in 70% of patients, WORMS cartilage integrity improved in 50%, and K&L grades remained stable in 80%. Overall, these findings support the feasibility and tolerability of a single OSCA injection, with exploratory efficacy in both clinical and MRI outcomes.