Ceftriaxone

Background
It is known that nerve cells called motor neurons die in the brains and spinal cords of people with ALS. However, the cause of the cell death is unknown. Researchers think that increased levels of a chemical called glutamate may be related to the cell death. For this reason researchers want to study drugs that decrease glutamate levels near nerves. Ceftriaxone is an antibiotic (in a class known as "cephalosporins") that is approved to treat certain types of infections. The investigators involved in this study are interested in studying ceftriaxone because the drug may also increase the level of a protein that decreases glutamate levels near nerves. Studies of ceftriaxone in the laboratory suggest that it may protect motor neurons form injury.

The purpose of the study is to evaluate the safety and the effecicacy of prolonged intravenous ceftriaxone treatment in people with ALS.

Study Details
This is a double-blind, placebo controlled multi-center clinical trial of ceftriaxone in 600 subjects with ALS. The study uses a sequential, non-stop drug development design. This design was chosen as a method to expedite drug development, while safely testing a new therapeutic agent in ALS. We will determine optimal dosage, safety and efficacy of ceftriaxone in ALS. Our goals are to determine the steady state levels and population kinetics of ceftriaxone in the CSF (Aim 1, STAGE 1), define the safety and tolerability of ceftriaxone after a minimum of 20 weeks of daily treatment (Aim 2, STAGE 2) and to ascertain whether chronic treatment with ceftriaxone prolongs survival in ALS (Aim 3, STAGE 3.)

Contact
Kate Bednarz, RD, (212) 305-2027 or kbednarz@neuro.columbia.edu