Presented at the 27th International Symposium on Amyotrophic Lateral Sclerosis and Motor Neurone Disease, Dublin Ireland, December 2016.

Objectives: We asked, for what types of ALS human clinical trials can concurrent controls, historical controls, and virtual controls be most efficient.


  • Historically Controlled Trials (HCTs) are an efficient design when large treatment effects are expected, e.g., >50% improvement
  • The threshold at which HCTs are more efficient than Concurrently Controlled Trials (CCTs) will change as improved prediction of outcomes reduces unexplained trial-to-trial variation
  • Use of HCTs will also be aided by accumulation of additional historical control data as secular trends in ALS care may bias older trials
  • Historically controlled trials are an appropriate design when studying intensive treatments that would require a large treatment effect to be practical

Authors: David A Schoenfeld, Robert Küffner, Eric A Macklin, David L Ennist, Dan H Moore, Neta Zach, Nazem Atassi



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