The School of Medicine at the University of California, Riverside (UC Riverside) will serve as lead investigational site for a Phase 2 clinical trial evaluating ecopipam, an investigational first-in-class drug under evaluation for the treatment of stuttering in adults and Tourette syndrome in pediatric patients. Sponsored by Emalex Biosciences, Inc., UC Riverside is seeking participants for the Speak Freely Study now through April 2021. The multi-center study is managed by contract research organization Premier Research Group.
Stuttering & Tourette Syndrome
Childhood-onset fluency disorder, also known as stuttering or stammering, is a common multifactorial speech disorder. It is normally seen with recurrent prolongations, reverberations, or blocks of sounds, syllables, phrases or words. It is estimated that more than three million people in America and over 70 million people worldwide stutter, with males affected more frequently than females. While there is no one cause of stuttering, possible causes may include family history and neurological differences in the way one’s brain works during speech. There are currently no U.S. Food and Drug Administration (FDA) approved medications for the treatment of stuttering.
Tourette syndrome is identified during childhood and involves uncontrollable repetitive movements or unwanted sounds (tics), such as repeatedly blinking the eyes, shrugging the shoulders or blurting out offensive words. Various treatments include medication or psychological therapies. For more on Tourette syndrome, see the National Institute of Neurological Disorders and Stroke Tourette syndrome fact sheet.
The Investigational Product
Ecopipam is an investigational first-in-class drug under evaluation or the treatment of childhood-onset fluency disorder (stuttering) in adults and Tourette syndrome (TS) in pediatric patients. The drug selectively blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two “families” based on their genetic structure: “D1” (including subtypes D1 and D5) and “D2” (including subtypes D2, D3 and D4). D1-receptor supersensitivity may be a mechanism for the repetitive and compulsive behaviors associated with TS.
Ecopipam is generally thus far shown to be well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with TS. Adverse events affecting primarily the central nervous system include sedation, insomnia and psychiatric changes while those associated with the gastrointestinal system include nausea and vomiting are thus far the most reported side effects.
The Speak Freely Study is for adults who were diagnosed with a stuttering disorder as a child and who still struggle with speaking clearly. The study will test the safety of an investigative medicine and whether it may help improve the ability to get words out without stammering. Adults interested in participating must be 18 or above in age, have a stuttering disorder since childhood and are willing to of course consent to take the investigational medication called Ecopipam for up to 13 weeks. During this study the participants are video-recorded during interviews to assess stuttering. Other study criteria will apply.
A Phase 2 multicenter, randomized, double-blind, placebo-controlled, parallel group exploratory trial, the Speak Freely Study (NCT04492956) investigates the safety and efficacy of investigational drug ecopipam in adult subjects with childhood onset fluency disorder. Sponsored by Emalex in collaboration with UC Riverside, global contract research organization (CRO) Premier Research Group will manage the clinical trial. Targeting a total of 68 participants, the study started October 2, 2020 and runs through till December 31, 2021.
The Principal Investigator Point of View
Dr. Gerald Maguire, professor and chair of psychiatry and neuroscience at UC Riverside also happens to be a world-class expert on stuttering who has spent the bulk of his professional career seeking an effective treatment for the childhood-onset fluency disorder. Dr. Maguire recently commented for the study press release, “Knowing first-hand how stuttering can be detrimental to a person’s overall well-being, I am excited to be part of this journey and the meaningful work being done by Emalex.” Dr. Maguire continued, “Many people are unaware that stuttering is a treatable neurological disorder condition, so there is a significant educational opportunity here that will serve those who stutter, their families, and even the public.”
The UC Riverside PI also emphasized, “If ecopipam is found at the end of this trial to be a potentially safe and effective treatment for stuttering, it would help millions of people who stutter, including myself.” His point: this is a significantly underserved population that will benefit tremendously from a safe and effective medication, should this one probe to be so.
Trial Site Locations
This study, although led by Emalex and UC Riverside, will be managed by a global CRO, Premier Research Group, with a number of sites in Florida, Georgia, Kansas, Michigan, New York and Tennessee.
Emalex Biosciences LLC is a biopharmaceutical company and the industry sponsor for this clinical trial. They focus on the development of new treatments for rare and orphan neurological conditions. Ecopipam is the first candidate, a novel compound that has been studied as a potential treatment for a variety of central nervous system (CNS) disorders, such as Tourette syndrome.
Launched in 2018 in the Chicago area, Emalex was founded by Jeff Aronin, a well-known life sciences entrepreneur and investor. He has been involved in the approval of nearly a dozen new medicines and technologies clearing FDA authorization.
Gerald Maguire, MD, professor and chair of psychiatry and neuroscience at UC Riverside
Call to Action: Do you or a loved one stutter? There may be a treatment in the future if this investigational candidate known as ecopipam can work. See the study website for additional information.