Compassionate Use of Ceftriaxone in Patients With Amyotrophic Lateral Sclerosis (ALS)

This study has been withdrawn prior to enrollment.
(No further patient to be enrolled. This was a 1 patient compassionate use protocol.)
MDA/ALS Center of Hope
Information provided by:
Drexel University Identifier:
First received: July 14, 2008
Last updated: March 24, 2011
Last verified: March 2011
Amyotrophic lateral sclerosis is a uniformly progressive and fatal neurodegenerative disorder for which there is no known cure. In a novel attempt to widen the search for potential therapeutic agents, a NINDS- led cooperative group performed an in-vitro screening program of 1040 FDA approved drugs in over 28 assays relevant to various neurodegenerative disorders. Several cephalosporins showed hits in ALS relevant assays. Efficacy was noted in models suggesting increased expression of the astrocytic glutamate transporter, EAAT2, as well as models of superoxide dismutase mediated toxicity. Ceftriaxone is a third generation cephalosporin with good CNS penetration, a long half-life, and was effective in both types of ALS assays. Ceftriaxone has calcium binding activity, antioxidant properties, and rescues motor neurons in culture from chronic glutamate toxicity. Since completion of the original NINDS screen, Ceftriaxone has been shown to increase by three fold EAAT2 activity in rodent brains, due to ceftriaxone's ability to increase EAAT2 promotor activation This program is for the use of ceftriaxone in ALS for compassionate care. Currently ceftriaxone is approved by the U.S. Food and Drug Administration (FDA) for treating bacterial infections but not for treating ALS. However, there is an ongoing phase I study -by NEALS Consortium and the National Institute of Health- with three cohorts -a placebo group and two groups receiving either 2 or 4 grams of ceftriaxone daily-. Unfortunately there are only a limited number of patients being enrolled and the next phase of the project will not be undertaken until next year. At this point there are ALS patients unable to participate in this Phase I trial and unlikely to be alive when the next phase of study begins. Some of these patients want to receive the drug and are willing to pay for the drug and nursing care. We are therefore requesting a compassionate use protocol for these patients who request the medication and are willing to pay for the drug and nursing care to administer it. Dr. Terry Heiman-Patterson will supervise the administration and safety monitoring including labs for renal and hepatic function as well as IV site inspection.

Amyotrophic Lateral Sclerosis
Cerebrospinal Fluid
Neurodegenerative Disease
Motor Neuron Disease

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Cross-Sectional
Official Title: Compassionate Use of Ceftriaxone in Patients With ALS

Resource links provided by NLM:

Further study details as provided by Drexel University:

Estimated Enrollment: 5
Study Start Date: June 2007
Estimated Study Completion Date: September 2008
Estimated Primary Completion Date: April 2008 (Final data collection date for primary outcome measure)
Subjects having either definite or probable ALS by El Escorial Criteria.


Ages Eligible for Study:   20 Years to 85 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
ALS clinic patients at MDA/ALS Center of Hope.

Inclusion Criteria:

  • Diagnosis of definite or probable ALS by the El Escorial Criteria
  • Participants must be medically able to undergo the study procedures
  • Subjects must have a registered nurse who can administer the drug daily and also inspect the IV site
  • Not Pregnant
  • Willing to pay for the cost of drug, administration, and safety testing
  • Able to give informed consent

Exclusion Criteria:

  • Patients who are not diagnosed with ALS by a physician
  • Patients unable to give informed consent
  • Patients who have a history of sensitivity to cephalosporin or penicillin antibiotics (such as Ancef, Keflex, Ceclor, Ceftin, Lorabid, Suprax, or Fortaz).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

No Contacts or Locations Provided
  More Information

Responsible Party: Terry Heiman-Patterson, MD, MDA/ALS Center of Hope Identifier: NCT00718393     History of Changes
Other Study ID Numbers: Internal-16964 
Study First Received: July 14, 2008
Last Updated: March 24, 2011
Health Authority: United States: Institutional Review Board

Keywords provided by Drexel University:
Amyotrophic Lateral Sclerosis
Cerebrospinal Fluid
Neurodegenerative Disease
Motor Neuron Disease
Autonomic Nervous System
Neurodegenerative Diseases
Movement Disorders

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Neurodegenerative Diseases
Central Nervous System Diseases
Metabolic Diseases
Nervous System Diseases
Neuromuscular Diseases
Pathologic Processes
Proteostasis Deficiencies
Spinal Cord Diseases
TDP-43 Proteinopathies processed this record on May 24, 2016