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

NCT ID: NCT06021522

Last Updated: 2025-08-13

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 150 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-16
• Study Completion Date: 2027-03-31

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).

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.

Conditions

Tourette Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Ecopipam

Intervention Type: DRUG

Selective dopamine D1 and D5 receptor antagonist

Interventions

Ecopipam

Selective dopamine D1 and D5 receptor antagonist

Intervention Type: DRUG

Other Intervention Names

Ecopipam Hydrochloride

Eligibility Criteria

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.

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Emalex Biosciences Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Harmonex, Inc.

Dothan, Alabama, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Advanced Research Center

Anaheim, California, United States

CenExel CIT-IE

Bellflower, California, United States

Cortica Site Network

Glendale, California, United States

Cortica Site Network - San Rafael

San Rafael, California, United States

Yale School of Medicine

New Haven, Connecticut, United States

Childrens National Hospital

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

NW FL Clinical Research Group, LLC

Gulf Breeze, Florida, United States

Research in Miami Inc

Hialeah, Florida, United States

University of Miami Miller School of Medicine

Miami, Florida, United States

Florida International Research Center

Miami, Florida, United States

Medical Research Group of Central Florida

Orange City, Florida, United States

APG Research, LLC

Orlando, Florida, United States

University of South Florida

St. Petersburg, Florida, United States

Pediatric Epilepsy and Neurology Specialists

Tampa, Florida, United States

Rare Disease Research, LLC

Atlanta, Georgia, United States

Lurie Children Hospital of Chicago

Chicago, Illinois, United States

Rush University Medical Center

Chicago, Illinois, United States

Josephson-Wallack-Munshower Neurology

Indianapolis, Indiana, United States

University of Louisville Research Foundation Inc.

Louisville, Kentucky, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Mass General Hospital

Boston, Massachusetts, United States

Boston Childrens Hospital

Boston, Massachusetts, United States

Umass Chan Medical School

Worcester, Massachusetts, United States

Michigan Clinical research Institute PC

Ann Arbor, Michigan, United States

Neurobahavioral Medicine Group

Bloomfield Hills, Michigan, United States

St. Louis Children's Hospital

St Louis, Missouri, United States

Alivation Research

Lincoln, Nebraska, United States

University of Rochester

Rochester, New York, United States

OnSite Clinical Solutions LLC

Charlotte, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

National Childrens Hospital - The Ohio State University

Columbus, Ohio, United States

North Star Medical Research, LLC

Middleburg Heights, Ohio, United States

Providence Brain and Spine Institute

Portland, Oregon, United States

Coastal Pediatric Research

Charleston, South Carolina, United States

Access Clinical Trials, Inc.

Nashville, Tennessee, United States

Access Clinical Trials, Inc.

Nashville, Tennessee, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

UT Southwestern

Dallas, Texas, United States

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Road Runner Research, Ltd

San Antonio, Texas, United States

Cedar Clinical Research

Draper, Utah, United States

Core Clinical Research

Everett, Washington, United States

Center Spectar-Plovdiv

Plovdiv, Bulgaria, Bulgaria

ASMP-IP- d-r Kayryakova

Sofia, Sofia-Grad, Bulgaria

Kalimat Medical Center_Sofia

Sofia, , Bulgaria

The Kids Clinic Inc

Ajax, Ontario, Canada

Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek-es Ifjusagpszicihatria

Szeged, Hungary, Hungary

Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)

Budapest, , Hungary

Istituto Giannina Gaslini, Universita di Genova

Genova, GE, Italy

Universita degli Studi di Napoli Federico II

Napoli, Napoli, Italy

Ospedale Pediatrico Bambino Ges

Roma, RM, Italy

Azienda Ospedaliero Universitaria " G. Rodolico - San Marco"

Catania, , Italy

IRCCS Istituto Neurologico Carlo Besta

Milan, , Italy

Neurologia Śląska Centrum Medyczne

Katowice, Poland, Poland

Gdanskie Centrum Zdrowia Sp z o.o.

Gdansk, Pomeranian Voivodeship, Poland

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

Poznan, Wielkopolska, Poland

Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie

Krakow, Woj. Malopolskie, Poland

Centrum Medyczne Plejady

Krakow, , Poland

Institute of Mental Health

Belgrade, Belgrad, Serbia

Clinic of Neurology and Psychiatry for Children and Adolescents

Belgrade, Grad Beograd, Serbia

Clinical Centre Nis Center of Mental Health

Niš, Serbia, Serbia

Hospital General Universitario Gregorio Maranon

Madrid, Madrid, Spain

Hospital Universitario 12 de Octubre

Madrid, Madrid, Spain

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, Spain

Hospital Universitario Virgen del Rocío

Seville, Sevilla, Spain

Countries

United States; Bulgaria; Canada; Hungary; Italy; Poland; Serbia; Spain

Other Identifiers

2023-503545-67-00

Identifier Type: OTHER

Identifier Source: secondary_id

EBS-101-TD-391

Identifier Type: -

Identifier Source: org_study_id