MIROCALS: Modifying Immune Response and OutComes in ALS

The secondary objectives of MIROCALS are: To validate a new phase-II study design to improve the efficiency of drug development in ALS with early determination of drug response using established biomarkers (BMs). The aims of this new trial design are: (i) To shorten future trials duration in ALS using an early drug responding surrogate marker of disease activity; (ii) To establish the proof of mechanism (PoM) of the tested drugs; (iii) To identify drug responder status. Additional exploratory objectives are: (i) Deep immune \& inflammatory phenotyping (ii) Brain biomarkers (iii) Genomics and Transcriptomics