Efficacy and cost-effectiveness of Stem Cell injections for symptomatic relief and strUctural improvement in people with Tibiofemoral knee OsteoaRthritis: protocol for a randomised placebo-controlled trial (the SCUlpTOR trial)
Leticia A Deveza,
Win Min Oo,
Xia Wang,
Felix Eckstein,
Wolfgang Wirth,
Anthony Harris,
Xiaoqian Liu,
Sarah Robbins,
Sonika Virk,
Benny Antony,
David Humphries,
Christopher Little,
James Linklater,
Karen Schuck,
Kirsty Carmichael
Affiliations
Leticia A Deveza
Institute of Bone and Joint Research, Kolling Institute of Medical Research, The University of Sydney, Sydney, New South Wales, Australia
Win Min Oo
Department of Rheumatology, Royal North Shore Hospital, Northern Clinical School, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia
Xia Wang
Department of Rheumatology, Royal North Shore Hospital, Northern Clinical School, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia
Felix Eckstein
Institute of Anatomy and Cell Biology and Ludwig Boltzmann Institute for Arthritis and Rehabilitation, Paracelsus Medical University Salzburg, Salzburg, Austria
Wolfgang Wirth
Institute of Anatomy and Cell Biology and Ludwig Boltzmann Institute for Arthritis and Rehabilitation, Paracelsus Medical University Salzburg, Salzburg, Austria
Anthony Harris
Centre for Health Economics, Monash University, Clayton, Victoria, Australia
Xiaoqian Liu
Institute of Bone and Joint Research, Kolling Institute of Medical Research, The University of Sydney, Sydney, New South Wales, Australia
Sarah Robbins
Department of Rheumatology, Royal North Shore Hospital, Northern Clinical School, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia
Sonika Virk
Department of Rheumatology, Royal North Shore Hospital, Northern Clinical School, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia
Benny Antony
Menzies Institute for Medical Research, University of Tasmania, Hobart, Tasmania, Australia
David Humphries
School of Medicine, College of Health and Medicine, University of Tasmania Faculty of Health, Hobart, Tasmania, Australia
Christopher Little
Raymond Purves Bone and Joint Research Labs, Kolling Institute of Medical Research, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia
James Linklater
Castlereagh Imaging, St Leonards, Sydney, New South Wales, Australia
Karen Schuck
Department of Rheumatology, Royal North Shore Hospital, Northern Clinical School, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia
Kirsty Carmichael
Menzies Institute for Medical Research, University of Tasmania, Hobart, Tasmania, Australia
Introduction Knee osteoarthritis (KOA) is a highly prevalent disabling joint disease. Intra-articular stem cell therapy is increasingly being used for treating KOA with little high-quality evidence to support its use. The aim of this study is to investigate the efficacy, safety and cost-effectiveness of allogeneic mesenchymal stem cells (Cymerus MSCs) for treating symptomatic tibiofemoral KOA and improving knee structure over 24 months.Methods and analysis The Stem Cell injections for symptomatic relief and strUctural improvement in people with Tibiofemoral knee OsteoaRthritis study is a phase III, multi-centre, parallel, superiority, randomised, double-blind, placebo-controlled trial, which will be conducted in Sydney and Hobart, Australia. 440 participants (220 per arm) aged over 40 years with painful KOA and mild to moderate structural change on X-ray (Kellgren and Lawrence grade 2 or 3) with medial minimum joint space width between 1 and 4 mm in the study knee will be recruited from the community and randomly allocated to receive either intra-articular MSCs or saline at baseline, week 3 and week 52. The coprimary outcomes will be the proportion of participants achieving patient-acceptable symptom state for knee pain at 24 months and quantitative central medial femorotibial compartment cartilage thickness change from baseline to 24 months. Main secondary outcomes include change in knee pain, Patient Global Assessment, physical function, quality of life and other structural changes. Additional data for cost-effectiveness analysis will also be recorded. Adverse events will be monitored throughout the study. The primary analysis will be conducted using modified intention-to-treat.Ethics and dissemination This protocol has been approved by The University of Sydney (USYD) Human Research Ethics Committee (HREC) #: 2020/119 and The University of Tasmania (UTAS) HREC #: H0021868. All participants will be required to provide informed consent. Dissemination will occur through conferences, social media, and scientific publications.Trial registration numbers Australian New Zealand Clinical Trials Registry (ACTRN12620000870954); U1111-1234-4897.
