GCT has been selected to run a new project in patients diagnosed with PMM by its long-term Client. This Phase 2, multicenter, double-blind, placebo-controlled study will be managed by GCT team in Czech Republic and Hungary.
Primary Mitochondrial Myopathies are a group of genetic disorders affecting the skeletal muscle. The main symptoms of PMM are muscle fatigue, weakness, and exercise intolerance. The severity of these symptoms varies greatly from one person to another, and always affects the quality of life. It is caused by a mutation in genes of the mitochondria, and is one of the most common metabolic diseases. The new treatment aims to prove its safety and efficacy against the main symptoms of PMM.
GCT is responsible for a wide scope of services ranging from feasibility analysis and regulatory support to monitoring, site contracting and management. The trial is currently in startup in both countries with the sites already selected.