Efficacy and safety of autologous cell therapies for knee cartilage defects (autologous stem cells, chondrocytes or the two): randomized controlled trial design

Abstract

Aim: The main aim of this trial is to test the safety and efficacy of autologous stromal/stem cells, chondrocytes or the two combined in the treatment of knee cartilage defects.

Patients & methods: Patients with symptomatic chondral/osteochondral defects will be randomized to cell therapy treatment with one of three cell populations (1:1:1). The primary efficacy outcome is a functional knee score (Lysholm) at 15 months post-treatment and the primary safety outcome is the incidence of adverse events. Secondary objectives are to analyze repair tissues, quality of life and cost-utility assessments. Exploratory objectives are to identify predictors for success/potency and dose-response relationships.

Results & conclusion: This trial has been carefully designed so that valuable scientific and clinical information can be gathered throughout and in the final analysis.

Keywords: MSCs; biomarkers; cartilage repair; cell potency; cell-based therapy; chondrocytes; clinical trial protocol; functional outcome measures; reverse translational research.