CEFTRIAXONE ALS CLINICAL RESEARCH TRIAL CEFTRIAXONE - NEALS CONSORTIUM

Recruitment Status:  Currently Recruiting

Sponsor and Collaborators:
Massachusetts General Hospital
National Institute of Neurological Disorders and Stroke (NINDS)

Purpose: The purpose of the study is to evaluate the safety and efficacy of ceftriaxone treatment in amyotrophic lateral sclerosis (ALS).

Primary Outcome Measures:
Survival.
ALSFRS-R.

Secondary Outcome Measures:
Vital capacity
Evaluation of multiple upper extremity muscles using hand held dynamometry
Quality of life
Long-term safety and tolerability of ceftriaxone
 
Estimated Enrollment: 600

Study Start Date: June 2006
Stage III Trial Start Date: May 2009

Detailed Description: 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 third stage, which will begin enrollment in Spring 2009, will determine whether the study drug prolong survival and slows decline in function due to ALS.

Eligibility:
Ages Eligible for Study:    18 Years and older
Genders Eligible for Study:    Both
Accepts Healthy Volunteers:    No

Criteria:
**This is a partial list.  Please check with the nearest site to review the full eligibility criteria.**

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).

Patients: Please Click HERE for a complete list of participating sites.
Patients may contact individual study coordinators at the participating sites directly.

Coordinating Center Contact Information: 

Amy Swartz, Project Manager
617-724-4246
alswartz@partners.org