Clinical Trial Ceftriaxone in Subjects With ALS

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Merit E. Cudkowicz, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT00349622
First received: July 5, 2006
Last updated: November 13, 2012
Last verified: November 2012
  Purpose

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


Condition Intervention Phase
Amyotrophic Lateral Sclerosis
ALS
Drug: ceftriaxone
Other: placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Clinical Trial Ceftriaxone in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • Survival. [ Time Frame: duration of the study ] [ Designated as safety issue: No ]
  • ALSFRS-R. [ Time Frame: duration of study ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • vital capacity [ Time Frame: duration of the study ] [ Designated as safety issue: No ]
  • evaluation of multiple upper extremity muscles using hand held dynamometry [ Time Frame: duration of the study ] [ Designated as safety issue: No ]
  • quality of life [ Time Frame: duration of the study ] [ Designated as safety issue: No ]
  • long-term safety and tolerability of ceftriaxone. [ Time Frame: duration of the study ] [ Designated as safety issue: No ]

Enrollment: 513
Study Start Date: July 2006
Study Completion Date: November 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1
Ceftriaxone
Drug: ceftriaxone

Participants will be randomly assigned to receive treatment with ceftriaxone or placebo for at least 12 months. Two thirds of participants will receive ceftriaxone and one third will receive placebo. This is a blinded study, so neither participants nor study staff will know which treatment a participant is receiving.

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.

Placebo Comparator: 2 Other: placebo
an inactive substance

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 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00349622

  Show 58 Study Locations
Sponsors and Collaborators
Massachusetts General Hospital
Investigators
Principal Investigator: Merit Cudkowicz, MD, MSc. Associate Professor of Neurology, Harvard Medical School, Massachusetts General Hospital
  More Information

Additional Information:
No publications provided

Responsible Party: Merit E. Cudkowicz, MD, Professor of Neurology, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT00349622     History of Changes
Other Study ID Numbers: U01NS049640-02, NINDS, U01NS049640-02, NINDS CRC
Study First Received: July 5, 2006
Last Updated: November 13, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Massachusetts General Hospital:
amyotrophic lateral sclerosis
ALS
ceftriaxone
cephalosporin antibiotic
motor neurons

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes
Ceftriaxone
Cephalosporins
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013