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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

Compassionate Use of Ceftriaxone in Patients With ALS

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00718393
• Other study ID #: Internal-16964
• Status: Completed
• Phase: N/A
• First received: July 14, 2008
• Last updated: March 7, 2017
• Start date: June 2007
• Est. completion date: October 2015

Study information

• Verified date: March 2017
• Source: Drexel University
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: October 2015
• Est. primary completion date: October 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years to 85 Years

Eligibility

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).

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Cerebrospinal Fluid
• Motor Neuron Disease
• Neurodegenerative Disease
• Neurodegenerative Diseases
• Sclerosis

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Drexel University College of Medicine	MDA/ALS Center of Hope