Clinical Trial Ceftriaxone in Subjects With ALS
Clinical Trial Ceftriaxone in Subjects With ALS
The purpose of the study is to evaluate the safety and efficacy of ceftriaxone treatment in
 amyotrophic lateral sclerosis (ALS).
It is known that nerve cells called motor neurons die in the brains and spinal cords of
 people with amyotrophic lateral sclerosis (ALS). However, the cause of this 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-a semi-synthetic, third generation cephalosporin
 antibiotic-may 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 from
 injury.

 Ceftriaxone is approved by the U.S. Food and Drug Administration (FDA) for treating bacterial
 infections but not for treating ALS. Also, ceftriaxone has not been given to people over a
 long period of time, such as months or years. The goals of this study are to evaluate the
 safety and effectiveness of ceftriaxone as a treatment for ALS, and to determine the safety
 and effectiveness of long-term use of the drug in people with ALS.

 A total of 600 eligible people with ALS will be enrolled in this multi-center research study.
 Participants will be randomly assigned to receive treatment with ceftriaxone (2/3 of
 participants) or placebo (1/3 of participants) for at least 12 months.

 The study consists of three stages. The first stage, which has completed enrollment, will
 look at whether ceftriaxone enters the cerebrospinal fluid (the fluid that surrounds the
 spinal cord, also called CSF) in amounts that are high enough to be of possible benefit. The
 second stage, which has also completed enrollment, will look at the safety and side effects
 of the study drug when taken daily for at least 20 weeks. The study is currently enrolling
 subjects for the third stage, which began in Spring 2009, and will determine whether the
 study drug prolongs survival and slows decline in function due to ALS.
Inclusion Criteria:

 -  Participants will be people with ALS, at least 18 years of age.

 -  Participants must be medically able to undergo the study procedures and have a
 caregiver or other individual who will be available to help with daily study
 medication administration.

 -  Participants should live within a reasonable distance of the study site, due to
 frequent study visits.

 Exclusion Criteria:

 -  Participants cannot be taking any other experimental medications for ALS, or have a
 history of sensitivity to cephalosporin antibiotics (such as Ancef, Keflex, Ceclor,
 Ceftin, Lorabid, Suprax, or Fortaz).