A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder

A Multicenter, Open-Label, Study to Evaluate the Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to [>=] 6 and less than [<] 12 years of age), adolescents (>=12 and <18 years of age), and adults (>=18 years of age) with Tourette's Syndrome (TS).

Study Overview

• Status: Active, not recruiting
• Conditions: Tourette Syndrome
• Intervention / Treatment: Drug: Ecopipam

Detailed Description

This study is to evaluate the long-term safety and tolerability of ecopipam tablets in eligible participants. The eligible participants will be entered into a treatment period and start a 4-week titration phase to achieve a target steady-state dose of 1.8 milligram per kilogram per day (mg/kg/day) ecopipam (2 mg/kg/day dose of ecopipam HCl). During the 4-week titration phase ecopipam will be dispensed following weight bands before reaching their respective maintenance dose until end of the treatment. Safety assessment will be conducted at baseline visit and at all treatment visits (Months 1-12, 15, 18, 21 and 24). Safety follow up visits will be conducted 7 and 14 days and a follow up phone call will be conducted 30 days after the last dose of ecopipam.

• Study Type: Interventional
• Enrollment (Estimated): 150
• Phase: Phase 3

Contacts and Locations

Study Locations

• Bulgaria
  • Plovdiv, Bulgaria, 4004
    • Center Spectar-Plovdiv
  • Sofia, Bulgaria, 1680
    • Kalimat Medical Center_Sofia
  • Sofia City
    • Sofia, Sofia City, Bulgaria, 1360
      • ASMP-IP- d-r Kayryakova
• Canada
  • Ontario
    • Ajax, Ontario, Canada, L1Z 0M1
      • The Kids Clinic Inc
• Hungary
  • Budapest, Hungary, 1146
    • Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)
  • Szeged, Hungary, 6720
    • Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek-es Ifjusagpszicihatria
• Italy
  • Catania, Italy, 95123
    • Azienda Ospedaliero Universitaria " G. Rodolico - San Marco"
  • Milano, Italy, 20133
    • IRCCS Istituto Neurologico Carlo Besta
  • Napoli, Italy, 80131
    • Universita degli Studi di Napoli Federico II
  • GE
    • Genova, GE, Italy, 16148
      • Istituto Giannina Gaslini, Universita di Genova
  • RM
    • Roma, RM, Italy, 165
      • Ospedale Pediatrico Bambino Ges
• Poland
  • Katowice, Poland, 40-689
    • Neurologia Slaska Centrum Medyczne
  • Kraków, Poland, 30-363
    • Centrum Medyczne Plejady
  • Pomorskie
    • Gdańsk, Pomorskie, Poland, 80-542
      • Gdanskie Centrum Zdrowia Sp z o.o.
  • Wielkopolska
    • Poznań, Wielkopolska, Poland, 61-731
      • Clinical Research Center Sp. z o.o. MEDIC-R Sp.k.
  • Woj. Malopolskie
    • Krakow, Woj. Malopolskie, Poland, 31-503
      • Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
• Serbia
  • Nis, Serbia, 18000
    • Clinical Centre Nis Center of Mental Health
  • Belgrad
    • Belgrade, Belgrad, Serbia, 11120
      • Institute of Mental Health
  • Grad Beograd
    • Beograd, Grad Beograd, Serbia, 1100
      • Clinic of Neurology and Psychiatry for Children and Adolescents
• Spain
  • Madrid, Spain, 28041
    • Hospital Universitario 12 de Octubre
  • Madrid, Spain, 28007
    • Hospital General Universitario Gregorio Marañon
  • Sevilla, Spain, 41013
    • Hospital Universitario Virgen del Rocío
  • Asturias
    • Oviedo, Asturias, Spain, 33011
      • Hospital Universitario Central de Asturias
• United States
  • Alabama
    • Dothan, Alabama, United States, 36303
      • Harmonex, Inc.
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • Anaheim, California, United States, 92805
      • Advanced Research Center
    • Bellflower, California, United States, 90706
      • CenExel CIT-IE
    • Glendale, California, United States, 91203
      • Cortica Site Network
    • San Rafael, California, United States, 94903
      • Cortica Site Network - San Rafael
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale School of Medicine
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Childrens National Hospital
  • Florida
    • Gulf Breeze, Florida, United States, 32561
      • NW FL Clinical Research Group, LLC
    • Hialeah, Florida, United States, 33013-3834
      • Research in Miami Inc
    • Miami, Florida, United States, 33136
      • University of Miami Miller School of Medicine
    • Miami, Florida, United States, 33173
      • Florida International Research Center
    • Orange City, Florida, United States, 32763
      • Medical Research Group of Central Florida
    • Orlando, Florida, United States, 32803
      • APG Research, LLC
    • St. Petersburg, Florida, United States, 33701
      • University of South Florida
    • Tampa, Florida, United States, 33609
      • Pediatric Epilepsy and Neurology Specialists
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Rare Disease Research, LLC
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
    • Chicago, Illinois, United States, 60611
      • Lurie Children Hospital of Chicago
  • Indiana
    • Indianapolis, Indiana, United States, 46256
      • Josephson-Wallack-Munshower Neurology
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • University of Louisville Research Foundation Inc.
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Kennedy krieger Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Childrens Hospital
    • Boston, Massachusetts, United States, 02114
      • Mass General Hospital
    • Worcester, Massachusetts, United States, 01655
      • UMass Chan Medical School
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Michigan Clinical Research Institute PC
    • Bloomfield Hills, Michigan, United States, 48302
      • Neurobahavioral Medicine Group
  • Missouri
    • St. Louis, Missouri, United States, 63110
      • St. Louis Children's Hospital
  • Nebraska
    • Lincoln, Nebraska, United States, 68526
      • Alivation Research
  • New York
    • Rochester, New York, United States, 14642
      • University of Rochester
  • North Carolina
    • Charlotte, North Carolina, United States, 28211
      • OnSite Clinical Solutions LLC
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
    • Columbus, Ohio, United States, 43205
      • National Childrens Hospital - The Ohio State University
    • Middleburg Heights, Ohio, United States, 44130
      • North Star Medical Research, LLC
  • Oregon
    • Portland, Oregon, United States, 97225
      • Providence Brain and Spine Institute
  • South Carolina
    • Charleston, South Carolina, United States, 29414
      • Coastal Pediatric Research
  • Tennessee
    • Nashville, Tennessee, United States, 37232-2551
      • Vanderbilt University Medical Center
    • Nashville, Tennessee, United States, 37203-6502
      • Access Clinical Trials, Inc.
    • Nashville, Tennessee, United States, 37203
      • Access Clinical Trials, Inc.
  • Texas
    • Dallas, Texas, United States, 75390
      • UT Southwestern
    • Houston, Texas, United States, 77030
      • The University of Texas Health Science Center at Houston
    • San Antonio, Texas, United States, 78249
      • Road Runner Research, Ltd
  • Utah
    • Draper, Utah, United States, 84020
      • Cedar Clinical Research
  • Washington
    • Everett, Washington, United States, 98201
      • Core Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• >=6 to >=18 years of age.
• Participants enrolling from the study EBS-101-TD-301; completed all visits through Week 24 and days 7 and 14 safety follow-up, met relapse criteria during the double-blind randomized (R/WD) period after completing the 301 end of trial (ET) visit, the 7 day and 14 day safety follow up visits, but not before 24 weeks following the 301 baseline visit or participants who met relapse criteria will be eligible after completing early termination visit, Day 7 and Day 14 follow up visits.
• Participants who completed the studies EBS-101-OL-001 or PSY302A.
• The enrolling participant must have had clinical benefit from ecopipam and would benefit from continued participation.
• Effective contraception during the study and 30 days after last study dose for sexually active participants
• <18 years of age participants parent/legal guardian must sign a written informed consent and participant must sign a written informed assent.
• Participant must have TD based on Diagnostic and Statistical Manual for Mental Disorders - 5th Edition (DSM-5-TR diagnostic criteria) for TD.
• TD diagnosis and both motor and vocal tics that cause impairment with normal routines

Exclusion Criteria:

• The participants who discontinued the studies PSY-302A, EBS-101-OL-001 or EBS-101-TD-301 due to reasons such as either lost to follow up, withdrawn consent, non-compliant or withdrawn by the discretion of either the site investigator or the sponsor.
• Participants with ongoing or past history of neurological condition (example [e.g.], Huntington's disease, Parkinson's disease, Wilson's disease, stroke, Restless Legs Syndrome).
• Any unstable mood disorder (DSM-5-TR criteria), mental illness or clinically significant lab abnormalities, moderate to severe renal or hepatic impairment, a PHQ-9 score >=10 at screening and history of neuroleptic malignant syndrome at the time of screening or baseline.
• Participants who completed the studies EBS-101-OL-001 or PSY-302A and who had previous exposure to ecopipam and oral neuroleptics within 4 weeks and depot neuroleptics within 3 months prior to screening, 6 months prior to Baseline.
• Participants receiving any other medication to treat motor or vocal tics and anti-depressant or anti-anxiety medications.
• Risk of suicide as per PI judgement
• Pregnant or lactating women
• Certain medications that would have unfavorable drug interactions with ecopipam, e.g., digoxin, fluoxetine, valproic acid, bupropion.
• Current or recent (past 3 months) DSM-5-TR substance use disorder (with the exception of nicotine).
• Recent behavioral therapy
• Positive urine drug screen for cocaine, amphetamine, benzodiazepines, barbiturates, phencyclidine (PCP) or opiates at Baseline, except those receiving stable, prescribed treatment for attention deficit/hyperactivity disorder (ADHD)
• Lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any other psychotic disorder.
• Unable to swallow tablets.
• Known hypersensitivity to any of ecopipam's excipients.
• History of seizures (excluding febrile seizures that occurred >2 years prior to Baseline).
• Myocardial infarction within 6 months from Screening.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Ecopipam 1.8 mg/kg/day; Ecopipam tablets will be administered orally (PO) once daily in the evening without regard to meals at concentrations 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 milligrams (mg) containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively in 4-week titration phase to achieve a target dose of 1.8 milligram per kilogram per day (mg/kg/day) ecopipam (2 mg/kg/day ecopipam HCl). Participants will be evaluated for safety at each baseline visit and at all treatment visits up to 24 months and at follow up visits at 7 and 14 days after last dose of ecopipam.

Drug: Ecopipam; Selective dopamine D1 and D5 receptor antagonist; Other Names: Ecopipam Hydrochloride

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	An adverse event (AE) is defined as any untoward medical occurrence in a subject administered a study drug and which does not necessarily have a causal relationship with this treatment. A TEAE is the development of an undesirable medical condition or the deterioration of a pre-existing medical condition following or during exposure to a pharmaceutical product, whether or not considered casually related to the product.	Baseline up to Month 24

Collaborators and Investigators

• Sponsor: Emalex Biosciences Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 16, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): March 1, 2027

Study Registration Dates

• First Submitted: July 11, 2023
• First Submitted That Met QC Criteria: August 28, 2023
• First Posted (Actual): September 1, 2023

Study Record Updates

• Last Update Posted (Actual): August 13, 2025
• Last Update Submitted That Met QC Criteria: August 8, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: Nervous System Diseases; Movement Disorders; Central Nervous System Diseases; Neurodegenerative Diseases; Mental Disorders; Tic Disorders; Brain Diseases; Neurodevelopmental Disorders; Central Nervous System Depressants; Ecopipam
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Mental Disorders; Genetic Diseases, Inborn; Neurodegenerative Diseases; Neurodevelopmental Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Basal Ganglia Diseases; Tic Disorders; Tourette Syndrome; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Neurotransmitter Agents; Tranquilizing Agents; Psychotropic Drugs; Dopamine Agents; Antipsychotic Agents; Dopamine Antagonists; Ecopipam
• Other Study ID Numbers: EBS-101-TD-391; 2023-503545-67-00 (Other Identifier: EUCT)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No