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.