CEFTRIAXONE

Description
Ceftriaxone- Identifier Number NCT00349622*
This clinical trial is in Phase III testing. It is a double-blind, randomized, placebo-controlled, multi-center study trial, which means the participant may or may not receive the actual medication. Safety has already been established in Phase I and Phase II. Results of efficacy in this Phase III are based upon the ALSFRS-R score. The medication requires a Hickman catheter through which intravenous infusions are given twice daily by the patient or caregiver. This trial will continue until the last patient is enrolled, plus 12 months.

Why this trial is notable
Dr. Merit Cudkowicz, Director of the ALS Clinic at Massachusetts General Hospital, Boston, is the Principal Investigator for this trial and one of the top echelon ALS clinician researchers. Ceftriaxone is based upon sound scientific principles and is now in the last stages of the Phase III enrollment, which is a significant milestone. The trial is robust with more than 500 subjects at 57 US and Canada sites, with several openings still available. Patients should live near the center because frequent visits are necessary. READ MORE

Issues of concern
1. Patient must have someone to assist with the infusions and be willing to tolerate the catheter.
2. Long duration: 12 months post-last patient enrolled

ALSWW Commentary  
The Ceftriaxone Phase III Clinical Trial is actively pursuing final participants at centers in the US and Canada through the Northeast ALS Consortium (NEALS) organization at Center locations. Ceftriaxone is an intravenous (IV) administered antibiotic ordinarily used for lung, brain and urinary tract infection, but Ceftriaxone has shown strong neuroprotective properties in addition to its anti-bacterial effects. Extensive laboratory studies of Ceftriaxone show demonstrable motor neuron protection from different types of injury. For example, Ceftriaxone increases EATT2 protein levels for those with ALS, most of whom have excess glutamate in both brain and spinal cord and a diminished level of EATT2 protein. EATT2 is important because it reduces excessive and damaging glutamate.

Clinical Trial Phases I and II determined that Ceftriaxone enters the brain and spinal cord at sufficiently high levels to protect  motor neurons. The optimal dosage level of 2 grams twice daily has been established. Further, potential side effects are clearly manageable. The home IV treatment through a central venous catheter is safe and easily maintained.

Anecdotal information is encouraging from several current participants we have met, who appear to be some of the most stable persons with ALS among those with whom we have ongoing contact.

Wikipedia Information about Ceftriaxone
Hoffman-LaRoche Developer of Rocephin, trade name of generic Ceftriaxone