Results of a Phase II Study to Determine the Efficacy and Safety of Genetically Engineered Allogeneic Human Chondrocytes Expressing TGF- β1

J Knee Surg DOI: 10.1055/s-0038-1676803Genetically engineered chondrocytes virally transduced with a transforming growth factor (TGF)-β1 (TG-C [TissueGene-C]) expression vector have been shown to have potential benefits in the nonoperative management of knee osteoarthritis. Previous literature has reported on safe dosages of TG-C. Therefore, the purpose of this study was to evaluate the Phase II results and a 24-month efficacy of this injectable mixture compared with placebo in patients with Kellgren–Lawrence (K–L) grade III knee osteoarthritis. Specifically, we assessed (1) functional outcomes, (2) pain scores, (3) adverse events (AEs), and (4) magnetic resonance imaging (MRIs) findings. We performed a multicenter, double-blinded, placebo-controlled, and randomized study of adults who had K–L grade III knee osteoarthritis. A total of 102 patients were 2:1 randomized to TG-C at a dose of 3.0 × 107 cells, or placebo injections between May 1, 2011 and October 31, 2012. Outcomes analyzed were knee joint function, pain, quality of life, adverse events, and MRI findings using the whole-organ magnetic resonance imaging score (WORMS) system. There were significant improvements in the International Knee Documentation Committee (IKDC) and visual analogue scale (VAS) scores in the TG-C cohort, when compared with the placebo cohort at weeks 12, 52, 72, and 104 (p < 0.05). No severe AEs were observed. Common AEs were arthralgia, joint inflammation, and joint effusion w...
Source: Journal of Knee Surgery - Category: Orthopaedics Authors: Tags: Original Article Source Type: research