Massachusetts General Hospital

Statement on the Clinical Trial of Ceftriaxone

08/Aug/2012

In July 2012, the DSMB for the NINDS-sponsored clinical trial of ceftriaxone in ALS recommended that based on existing data the trial be stopped because the study was unlikely to reach the pre-determined efficacy criteria. The NINDS leadership concurred.  Pre-clinical research identified ceftriaxone as a promising treatment for ALS therefore it was important for people with ALS to find out if the drug could be beneficial in ameliorating the disease.  The study used a novel seamless adaptive design. Final analysis and presentation of the results will occur after completion of site monitoring and database lock. The important contributions of patients, their families and the hard work of the investigators and their teams made it possible to implement the trial. While all had hoped for a more positive result, the trial has moved ALS research forward.