Study design
ICH
MAY, APPENDIX, DEL
A randomized, double-blind, placebo-controlled trial was conducted at seven Italian ALS referral centers from 2017 through 2020. The trial was conducted in accordance with the Good Clinical Practice guidelines of the International Council for Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) and the ethical principles of the Declaration of Helsinki, as amended by the 64th WMA General Assembly, Fortaleza, Brazil, in October 2013. The study complies with ICMJE guidelines on reporting.Protocol (EUDRACT 2016-002399-28 registration: 31st May 2016) approval was provided for the coordinating center and all trial sites by Ethical Committee of the coordinating center (Comitato Etico Provinciale di Modena) on 23th May 2017 (file number 95/17) and by AIFA (Agenzia Italiana del Farmaco) on 14th July 2017. All the participants provided written informed consent before screening (first and last patient enrollment: 05/10/2017 to 02/01/2020). The trial was a non-profit trial, financed by ARISLA (Fondazione Italiana di Ricerca per la SLA) with the “2015 AriSLA Ice Bucket Call for Clinical Projects”. Pfizer Inc. provided the active drug. Participants received no compensation.The study promoter was Azienda Ospedaliero-Universitaria (AOU) di Modena. The trial design, data analysis, and manuscript development were shared by the Steering Committee of the Study represented by all the local PIs (Supplementary Appendix, Section An independent data and safety monitoring board was established at trial beginning and periodically reviewed unblinded safety data during the trial (Acknowledgements section). Statistical analyses were performed by the Unit of Statistical and Methodological Support to Clinical Research, AOU, Modena, Italy. All the authors guarantee for data completeness and accuracy, and for adherence to RAP-ALS study protocol (available at 