Clinical Trial Ceftriaxone in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

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

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis , ALS

Study Type:


Type of Intervention:

Drug, N/A

Intervention Name:

ceftriaxone, placebo



Phase 3

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, MD, MSc., Associate Professor of Neurology, Harvard Medical School, Massachusetts General Hospital ID:


Neals Affiliated?


Coordinating Center Contact Information

Massachusetts General Hospital

149 13th Street
Charlestown, Massachusetts, 02129 United States

Full Study Summary:

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.

Study Sponsor:

Massachusetts General Hospital

Participant Duration:

52 weeks after last subject is enrolled.

Estimated Enrollment:


Estimated Study Start Date:

05 / 31 / 2009

Estimated Study Completion Date:

11 / 01 / 2012

Posting Last Modified Date:

04 / 21 / 2014

Date Study Added to

07 / 07 / 2006

Minimum Age:

18 Years

Maximum Age:


Can participants use Riluzole?


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).

Phoenix Neurological Associates

Phoenix, Arizona 85018
United States

University of California, Davis

Davis, California 95819
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University of California, San Francisco- Fresno

Fresno, California 93701
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Loma Linda University School of Medicine (CA)

Loma Linda, California 92354
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Cedars-Sinai Medical Center

Los Angeles, California 90048
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University of California, Los Angeles

Los Angeles, California 90095
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University of California, Irvine - MDA ALS Neuromuscular Center

Orange, California 92868
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California Pacific Medical Center

San Francisco, California 94115
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University of California, San Francisco

San Francisco, California 94117
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University of Colorado Health Sciences Center

Aurora, Colorado 80045
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Hospital for Special Care

New Britain, Connecticut 06053
United States

George Washington University

Washington, District of Columbia 20037
United States

Mayo Clinic Jacksonville

Jacksonville, Florida 32224
United States

University of Miami School of Medicine

Miami, Florida 33136
United States

ALS Center at Emory University

Atlanta, Georgia 30322
United States

Medical College of Georgia

Augusta, Georgia 30912
United States

Northwestern University Medical School

Chicago, Illinois 60611
United States

Indiana University (Regenstrief Health Center)

Indianapolis, Indiana 46202
United States

University of Kansas Medical Center

Kansas City, Kansas 66161
United States

University of Kentucky Medical Center

Lexington, Kentucky 40536
United States

Massachusetts General Hospital

Boston, Massachusetts 02114
United States

Lahey Clinic

Burlington, Massachusetts 01805
United States

Henry Ford Health System

Detroit, Michigan 48202
United States

Saint Mary's Healthcare

Grand Rapids, Michigan 49503
United States

Hennepin County Medical Center (Berman Center)

Minneapolis, Minnesota 55404
United States

St. Louis University

St. Louis, Missouri 63104
United States

Washington University

St. Louis, Missouri 63110
United States

Bryan LGH Medical Center (University of Nebraska)

Lincoln, Nebraska 68506
United States

UMDNJ- Robert Wood Johnson School of Medicine

New Brunswick, New Jersey 08901
United States

Albany Medical Center

Albany, New York 12208
United States

Beth Israel Medical Center (NY)

New York, New York 10003
United States

Cornell Medical Center

New York, New York 10021
United States

Columbia University

New York, New York 10032
United States

SUNY Upstate Medical University

Syracuse, New York 13210
United States

Carolinas Medical Center

Charlotte, North Carolina 28203
United States

Wake Forest University School of Medicine

Winston-Salem, North Carolina 27157
United States

The Cleveland Clinic Foundation

Cleveland, Ohio 44195
United States

Ohio State University

Columbus, Ohio 43210
United States

Oregon Clinic (Providence Clinic)

Portland, Oregon 97213
United States

Pennsylvania State University, Hershey Medical Center

Hershey, Pennsylvania 17033
United States

Drexel University College of Medicine (Hahnemann Campus)

Philadelphia, Pennsylvania 19107
United States

University of Pennsylvania

Philadelphia, Pennsylvania 19107
United States

Allegheny Hospital

Pittsburgh, Pennsylvania 15212
United States

University of Pittsburgh

Pittsburgh, Pennsylvania 15213
United States

Medical University of South Carolina

Charleston, South Carolina 29403
United States

Vanderbilt University

Nashville, Tennessee 37232
United States

Texas Neurology

Dallas, Texas 75214
United States

Methodist Neurological Institute

Houston, Texas 77030
United States

University of Utah Health Sciences Center

Salt Lake City, Utah 84132
United States

University of Virginia

Charlottesville, Virginia 22908
United States

University of Calgary

Calgary, Alberta T2N 4Z6

Univeristy of Alberta ALS Clinic

Edmonton, Alberta T6G 2B7

Dalhousie University

Halifax, Nova Scotia

London Health Sciences Center, University Campus

London, Ontario

University of Toronto

Toronto, Ontario

CHUM (Centre Hospitalier de l'Université de Montréal), Notre-Dame Hospital

Montreal, Quebec

Montreal Neurological Institute (McGill University)

Montreal, Quebec

Laval University

Quebec City, Quebec