Flex Pharma is launching the Phase 2 COMMEND trial, which will investigate the effects of FLX-787 in patients with muscle cramps associated with amyotrophic lateral sclerosis (ALS). The preliminary results of the trial are expected to be released in mid-2018.
The COMMEND trial (NCT03196375) will start with an assessment of cramp frequency in ALS patients at baseline. Patients then will receive FLX-787 (30 mg, three times daily), or a placebo, for 28 days. The study’s primary goal is a change in cramp frequency in those treated with FLX-787, but researchers also will analyze other parameters.
“Muscle cramps can have a severe impact on functional performance and quality of life for patients with motor neuron diseases like ALS,” Björn Oskarsson, MD, the trial’s investigator at the Mayo Clinic in Jacksonville, said in a press release. “Neurologists are often confronted with a situation where the patient experiences symptoms, and current treatments are either ineffective or have potentially limiting side effects. Flex Pharma’s FLX-787 may provide a better approach to this challenging problem.”
FLX-787 acts by activating two proteins, the TRPA1 and TRPV1 ion channels. Previous studies have demonstrated its capacity to stop muscle cramps induced by electrical stimulation in humans. The drug also reduced cramp frequency and pain in individuals with nocturnal leg cramps.
Last month, FLX-787 was granted fast track status by the U.S. Food and Drug Administration (FDA) as a treatment for severe muscle cramps in ALS patients.
“The Fast Track designation for FLX-787 is a validation by FDA that cramps are a severe, unmet medical need in ALS,” said William McVicar, Flex Pharma’s president and CEO. “While physicians treating ALS patients have come to understand how great an impact severe cramping – and the pain that attends it – can have on the lives of ALS patients, the lack of safe and effective treatments has provided little impetus to focus on the problem. We are working hard to change this with FLX-787,” he said.
FLX-787 also is being evaluated in ALS patients in Australia, as well as a potential treatment for cramps in patients with Charcot-Marie-Tooth disease, a hereditary neurological disease that affects the peripheral motor and sensory nerves motor of the body.