A group of collaborators announced their intent to launch a clinical trial in 12 academic sites on the anti-epileptic drug, Retigabine (a/k/a Ezogabine), in people diagnosed with ALS. The sites will be chosen by the Northeast ALS Consortium, and include sites in California (3), Massachusetts (2), Michigan, North Carolina, Maryland, Georgia, New York, Florida and Arizona. Screening for enrollment should be expected to begin sometime before the end of 2015. The primary goal of this research project is to study the effect of retigabine on upper and lower motor neuron physiology in ALS patients, however researchers have outlined a number of secondary outcome measures as well such as safety etc.

At the end of 2014, during a scientific meeting in Brussels, Kevin Eggan, Ph.D., who led the team doing the preclinical research behind the selection of this drug, reported that the trial will enroll up to 120 people between the 12 sites and that approximately 1/3 of PALS will be given placebo in the trial. According to Brian Wainger, M.D., PALS enrolled and selected for the active compound arm will be split into two cohorts, one receiving 600 mg and the other 900 mg of Retigabine.

In a webinar held shortly after the announcement, Wainger told PALS to expect to be in the study for at least 10 weeks. To be considered for enrollment, PALS must meet certain enrollment criteria such as having a possible, probable or definite ALS diagnosis and symptom onset occurring no more than then 36 months ago.  There are several specific exclusion items, most important perhaps to PALS with bulbar onset is that to enroll in the study a PALS must be able to swallow the retigabine pills throughout the study. Other examples of exclusion include the presence of a feeding tube or treatment for a serious cardiac issue at time of screening. Full study information is available in the global ALS clinical trial database.

As mentioned in that report from December, researchers at Eggan’s lab intend to collect skin samples from enrolled PALS from which they can create patient derived stem cells, sometimes referred to as induced pluripotent stem cells or more simply, iPSc.  Researchers plan to then test those iPS lines to determine if they could be used in future trials to determine whether or not a specific PALS would potentially benefit from this drug. The group working on this clinical trial include Harvard Stem Cell Institute, where Dr. Eggan is based, Massachusetts General Hospital Neurological Clinical Research Institute, GlaxoSmithKline, and the ALS Association.

This trial, along with all others, regardless of who funds them or where they are located, are listed and tracked by staff at the ALS Therapy Development Institute. You can view that global database and subscribe to receive email updates on trial launches, etc, by clicking here.

Bottom Line:

The Retigabine trial is NOT currently enrolling PALS. The ALS Therapy Development Institute has spoken about this drug and the trial multiple times, and we encourage people to review comments from our science team members, Drs. Lincecum and Perrin and others in recent webinars on the Brussels meeting and Clinical Trials in general. In addition, there is an active discussion thread on this proposed clinical trial on the ALS Forum, in which our CEO, Dr. Steve Perrin provides his individual comments on the drug and trial.