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

NCT06021522 | Active Not Recruiting Phase 3 FDA Drug

• 2 other identifiers: EBS-101-TD-3912023-503545-67-00
• Study Type: interventional
• Target: 150
• Locations: 8 countries
• Sites: 67

Brief Summary

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).

Conditions

Tourette Syndrome

Keywords

Ecopipam; Neurodevelopmental Disorders; Mental Disorders; Central Nervous System Depressants; Neurodegenerative Diseases; Movement Disorders; Tic Disorders; Nervous System Diseases; Central Nervous System Diseases; Brain Diseases

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Ecopipam 1.8 mg/kg/day

EXPERIMENTAL

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

Interventions

Ecopipam DRUG

Selective dopamine D1 and D5 receptor antagonist

Also known as: Ecopipam Hydrochloride

Ecopipam 1.8 mg/kg/day

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \>=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

You may not qualify if:

• 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).

Sponsors & Collaborators

• Emalex Biosciences Inc.: lead

Study Sites (67)

Harmonex, Inc.

Dothan, Alabama, 36303, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Advanced Research Center

Anaheim, California, 92805, United States

Location

CenExel CIT-IE

Bellflower, California, 90706, United States

Location

Cortica Site Network

Glendale, California, 91203, United States

Location

Cortica Site Network - San Rafael

San Rafael, California, 94903, United States

Location

Yale School of Medicine

New Haven, Connecticut, 06519, United States

Location

Childrens National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

NW FL Clinical Research Group, LLC

Gulf Breeze, Florida, 32561, United States

Location

Research in Miami Inc

Hialeah, Florida, 33013-3834, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Florida International Research Center

Miami, Florida, 33173, United States

Location

Medical Research Group of Central Florida

Orange City, Florida, 32763, United States

Location

APG Research, LLC

Orlando, Florida, 32803, United States

Location

University of South Florida

St. Petersburg, Florida, 33701, United States

Location

Pediatric Epilepsy and Neurology Specialists

Tampa, Florida, 33609, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30329, United States

Location

Lurie Children Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Josephson-Wallack-Munshower Neurology

Indianapolis, Indiana, 46256, United States

Location

University of Louisville Research Foundation Inc.

Louisville, Kentucky, 40202, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Mass General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Childrens Hospital

Boston, Massachusetts, 02115, United States

Location

Umass Chan Medical School

Worcester, Massachusetts, 01655, United States

Location

Michigan Clinical research Institute PC

Ann Arbor, Michigan, 48105, United States

Location

Neurobahavioral Medicine Group

Bloomfield Hills, Michigan, 48302, United States

Location

St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Alivation Research

Lincoln, Nebraska, 68526, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

OnSite Clinical Solutions LLC

Charlotte, North Carolina, 28211, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

National Childrens Hospital - The Ohio State University

Columbus, Ohio, 43205, United States

Location

North Star Medical Research, LLC

Middleburg Heights, Ohio, 44130, United States

Location

Providence Brain and Spine Institute

Portland, Oregon, 97225, United States

Location

Coastal Pediatric Research

Charleston, South Carolina, 29414, United States

Location

Access Clinical Trials, Inc.

Nashville, Tennessee, 37203-6502, United States

Location

Access Clinical Trials, Inc.

Nashville, Tennessee, 37203, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-2551, United States

Location

UT Southwestern

Dallas, Texas, 75390, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Road Runner Research, Ltd

San Antonio, Texas, 78249, United States

Location

Cedar Clinical Research

Draper, Utah, 84020, United States

Location

Core Clinical Research

Everett, Washington, 98201, United States

Location

Center Spectar-Plovdiv

Plovdiv, Bulgaria, 4004, Bulgaria

Location

ASMP-IP- d-r Kayryakova

Sofia, Sofia-Grad, 1360, Bulgaria

Location

Kalimat Medical Center_Sofia

Sofia, 1680, Bulgaria

Location

The Kids Clinic Inc

Ajax, Ontario, L1Z 0M1, Canada

Location

Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek-es Ifjusagpszicihatria

Szeged, Hungary, 6720, Hungary

Location

Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)

Budapest, 1146, Hungary

Location

Istituto Giannina Gaslini, Universita di Genova

Genova, GE, 16148, Italy

Location

Universita degli Studi di Napoli Federico II

Napoli, Napoli, 80131, Italy

Location

Ospedale Pediatrico Bambino Ges

Roma, RM, 165, Italy

Location

Azienda Ospedaliero Universitaria " G. Rodolico - San Marco"

Catania, 95123, Italy

Location

IRCCS Istituto Neurologico Carlo Besta

Milan, 20133, Italy

Location

Neurologia Śląska Centrum Medyczne

Katowice, Poland, 40-689, Poland

Location

Gdanskie Centrum Zdrowia Sp z o.o.

Gdansk, Pomeranian Voivodeship, 80-542, Poland

Location

Clinical Research Center Sp. z o.o. MEDIC-R Sp.k.

Poznan, Wielkopolska, 61-731, Poland

Location

Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie

Krakow, Woj. Malopolskie, 31-503, Poland

Location

Centrum Medyczne Plejady

Krakow, 30-363, Poland

Location

Institute of Mental Health

Belgrade, Belgrad, 11120, Serbia

Location

Clinic of Neurology and Psychiatry for Children and Adolescents

Belgrade, Grad Beograd, 1100, Serbia

Location

Clinical Centre Nis Center of Mental Health

Niš, Serbia, 18000, Serbia

Location

Hospital General Universitario Gregorio Maranon

Madrid, Madrid, 28007, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, Madrid, 28041, Spain

Location

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33011, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, Sevilla, 41013, Spain

Location

MeSH Terms

Conditions

Tourette Syndrome; Neurodevelopmental Disorders; Mental Disorders; Neurodegenerative Diseases; Movement Disorders; Tic Disorders; Nervous System Diseases; Central Nervous System Diseases; Brain Diseases

Interventions

ecopipam

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Heredodegenerative Disorders, Nervous System; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 11, 2023
• First Posted: September 1, 2023
• Study Start: August 16, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): March 1, 2027
• Last Updated: August 13, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (44); BU (3); IT (5); CA (1); ES (4); HU (2); PL (5); SE (3)