Autologous chondrocyte transplantation: a phase 1 study protocol to validate the safety and feasibility of a new advanced cell therapy product for articular cartilage repair in Brazil

ABSTRACT

Background:Membrane-assisted autologous chondrocyte transplantation is considered the gold standard surgical technique to treat greater than two millimetresdiameter cartilage lesions in the knee in patients after conservative treatment failure. However, this technique is only available in developed countries of North America, Europe and Japan. According to Brazilian law, it is considered an advanced cell therapy product. There is currently no product of this type enabled for clinical use in Brazil. Following the request of the Brazilian regulatory agency (ANVISA), this phase 1 study was developed. The objective is to access feasibility and safety of a new membrane-assisted autologous chondrocyte product.Methods:Three participants with a larger than two millimetresarticular cartilage lesion in the distal femur or the patella, which did not improvetheir symptoms with conservative treatment, will be submitted to an arthroscopically assisted cartilage biopsy. After isolation and expansion in a good manufacturing practicesfacility, chondrocyte seeded collagen membranes will be surgically inserted in the lesion and fixed with fibrin glue. The follow-up period will last 1 year. Primary outcome will be incidence and severity of complications according to NCI-CTCAE version4.0. Secondary outcomes will be Western-Ontario McMaster Universities Osteoarthritis Indexscale, International Knee Documentation Committeesubjective scale and magnetic resonance observation of cartilage repair tissuemagnetic resonancescale. Conclusions: This study, together with previous preclinical results and international experience, will allow patients in Latin America to have access to this advanced cell therapy.Trial RegistrationBrazilian registry of clinical trials RBR-6fgy76 (http//www.ensaiosclinicos.gov.br/rg/RBR-6fgy76/). Ethical approval CAAE 73911617.2.0000.0071.