The COMMIT study (NCT03254199) is a randomized, double-blind, trial that will be conducted at 20 centers the U.S. under Flex Pharma’s open investigational new drug (IND) application with the U.S. Food and Drug Administration.
Investigators hope to enroll 120 adult CMT patients who have severe muscle cramps. The study’s estimated completion date is August 2018. Although the trial is not yet recruiting, those who are interested can click on the clinical trial number above and scroll down to “Contacts and Locations.”
The trial will include a run-in period to establish a baseline in cramp frequency. Patients will be randomized to receive either 30 mg of FLX-787 three times a day for 28 days or a placebo. Determining cramp frequency over the 28-day period is the study’s primary goal.
“These new Phase 2 IND studies, as well as completion of the ongoing exploratory Phase 2 work in Australia, are expected to result in several important data readouts in 2018,” William McVicar, president and chief executive officer of Flex Pharma, said in a press release.
FLX-787 acts by activating two proteins, TRPA1 and TRPV1 ion channels. Previous studies have shown the drug candidate can successfully stop muscle cramps induced by electrical stimulation in humans. FLX-787 also reduced the frequency of cramps and the severity of pain in people who experience nighttime leg cramps.
The investigational therapy was granted fast track designation by the FDA for the treatment of severe muscle cramps associated with ALS (amyotrophic lateral sclerosis).
“Many of our patients with CMT neuropathy [nerve disease] suffer from frequent and severe muscle cramping, which can limit their activities of daily living and have a profound impact on their quality of life,” said Nicholas Johnson, the trial’s lead investigator and assistant professor of neurology at the University of Utah.
“FLX-787 promises to have significant clinical advantages because of its safety profile and the lack of systemic exposure,” he said.
The COMMIT trial in CMT disease was approved in July by the Inherited Neuropathies Consortium (INC). According to its website, INC is an “integrated group of academic medical centers, patient support organizations, and clinical research resources dedicated to conducting clinical research” in all types of CMT, as well as improving patient care.
INC is part of the Rare Diseases Clinical Research Network, and is funded through the National Institutes of Health.
Charcot-Marie-Tooth is a hereditary neurological disease that affects the body’s peripheral motor and sensory nerves. Flex Pharma is a clinical-stage biotech developing treatments for cramps and spasticity associated with severe neurological diseases and peripheral neuropathies like CMT, ALS, and multiple sclerosis (MS).