One of the most common symptoms reported by people with ALS is severe muscle cramping and spasms. Several therapies have been explored and shown early signs of impact, such as mexiletine and cannabis, as potential treatments to date. The company Flex Pharma announced recently that they have opened for enrollment a new clinical trial on a version of their HotShot product (FLX-787) in people with ALS and other disorders. According to the company’s press releases, FLX-787 is a small molecule compound that aims to modify how specific proteins (ion channels) in the body act and influence how a person’s body reacts to its environment. Flex Pharma received fast-track designation from the FDA for FLX-787 for the treatment of cramps in people with ALS in July 2017.

The placebo controlled study will enroll across nearly 30 sites in the United States and aims to enroll 120 people. One of the things that makes this study unique is that investigators are enrolling not just those diagnosed with ALS, but those that have been diagnosed with other forms of motor neuron disease including, primary lateral sclerosis (PLS) or progressive muscular atrophy (PMA). Participants will be asked to take the medication (a pill) three times a day during the 28 day study and to make three visits to a study center over the three month long clinical trial of FLX-787 (likely one of those visits will be a baseline visit, another will be a visit to receive the medication and go over administration and a third and final visit will take a few measurements such as ALSFRS-R, and tests to determine cramping frequency).

 

To qualify for enrollment, individuals with ALS, PLS or PMA must have reported weekly episodes of sustained and often painful muscle cramping that lasts seconds to minutes. According to the published study design, those that suffer from major gastrointestinal disorders, have significant swallowing problems or are using a g-tube will be excluded from enrollment. While the inclusion/exclusion criteria for this t rial doesn’t specifically name months since onset or breathing measures potential enrollees must meet, it does list as an inclusion criteria “expected survival > 6 months”. This note in the trial design would suggest that individuals very late in disease progression may not qualify for this study. Other information about enrollment criteria, as well as contact information for principal investigator Bjorn Oskarsson, M.D. of the Mayo Clinic in Jacksonville, Florida can be found online here.

 

Helpful Links:

Clinical Trial Design and Contact Information

Flex Pharma Product Description

 

UPDATE

Flex Pharma announced in June 2018 they will halt this study due to tolerability concerns. Here is a link to their press release.