Designing Clinical Trials for Mitochondrial Disease Therapies

Time: 10:00 am
day: Day Two


  • Understanding how drug development for mitochondrial disease is fraught with challenges due to disease heterogeneity, lack of comprehensive natural history and lack of precedent of approved therapies to guide development
  • Examining how careful consideration of inclusion criteria to minimize heterogeneity and enrich study participants will be most likely to respond to a therapeutic intervention which is essential
  • Learning how trade-offs exist when choosing to include a limited number vs. a larger number of study sites
  • Exploring how ehe COVID-19 pandemic has led to an evolution in study design and conduct