Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults

NCT05615220 | Completed Phase 3 Results DMC FDA Drug

• 3 other identifiers: EBS-101-TD-3012023-503494-38-002022-001961-11
• Study Type: interventional
• Target: 216
• Locations: 12 countries
• Sites: 95

Brief Summary

This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD). The study includes an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period.

Conditions

Tourette Disorder

Outcome Measures

Primary Outcomes (1)

• Time From Randomization to Relapse in Participants Greater Than and Equal to (>=) 6 and Less Than (<) 18 Years During the Double-Blind R/WD

  Time to relapse defined as a loss of \>=50 percent (%) of the improvement experienced on the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of Tourette's Disorder (TD), or requirement of hospitalization for worsening symptoms of TD in participants between the ages of \>= 6 and \<18 years for ecopipam compared to those receiving placebo during the double-blind, R/WD period. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in participants with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.

  From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)

Secondary Outcomes (1)

• Time From Randomization to Relapse in All Participants During the Double-Blind R/WD Period

  From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)

Study Arms (2)

1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl)

EXPERIMENTAL

Ecopipam 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 mg tablets (containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively); 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) target dose; oral administration daily in evenings.

Drug: Ecopipam Hydrochloride

Placebo during R/WD Phase

PLACEBO COMPARATOR

Matching Placebo tablets during R/WD period taken orally in the evening.

Drug: Ecopipam Hydrochloride

Interventions

Ecopipam Hydrochloride DRUG

Selective dopamine D1 and D5 receptor antagonist

1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl); Placebo during R/WD Phase

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 years of age
• ≥ 18 kg (\~ 40 lbs.)
• TD diagnosis and both motor and vocal tics that cause impairment with normal routines
• Minimum score of 20 on the YGTSS-R Total Tic Score
• May not be taking any medications used to treat motor or vocal tics for at least 14 days prior to Baseline.
• Effective contraception during the study and 30 days after last study dose for sexually active subjects

You may not qualify if:

• Previous exposure to ecopipam
• Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder)
• Unstable medical illness or clinically significant lab abnormalities
• Risk of suicide
• Pregnant or lactating women
• Moderate to severe renal insufficiency
• Hepatic insufficiency
• Positive urine drug screen
• Unstable doses for drugs to treat anxiety, depression, Attention Deficit Hyperactivity Disorder
• Certain medications that would lead to drug interactions
• Recent behavioral therapy

Sponsors & Collaborators

• Emalex Biosciences Inc.: lead

Study Sites (95)

Harmonex Neuroscience Research

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

Amnova Clinical Research

Irvine, California, 92604, United States

Location

Cortica Site Network - San Rafael

San Rafael, California, 94903, United States

Location

Syrentis Clinical Research

Santa Ana, California, 92705, United States

Location

Yale School of Medicine

New Haven, Connecticut, 06519, United States

Location

University of Florida

Gainesville, Florida, 32608, 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

Emcrown Clinical Research

Jensen Beach, Florida, 34957, 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

Care Research Center

Miami, Florida, 33175, 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, 33609-4181, United States

Location

Jedidiah Clinical Research

Tampa, Florida, 33617, United States

Location

Pediatric Neurology, PA

Winter Park, Florida, 32789, United States

Location

Advanced Discovery Research, LLC

Atlanta, Georgia, 30318, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30329, United States

Location

Atlanta Behavioral Research, LLC.

Atlanta, Georgia, 30338, United States

Location

Lurie Children Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

1725 W. Harrison St., Suite 755

Chicago, Illinois, 60612, United States

Location

The University of Chicago Hospitals

Chicago, Illinois, 60637-1447, United States

Location

Josephson-Wallack-Munshower Neurology

Indianapolis, Indiana, 46256, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Children's 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

Neurobehavioral Medicine Group

Bloomfield Hills, Michigan, 48302-1952, United States

Location

The Childrens Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Midwest Research Group

Saint Charles, Missouri, 63304, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Alivation Research

Lincoln, Nebraska, 68526, United States

Location

NYU Child Study Center

New York, New York, 10016-5815, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029-6504, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

Stony Brook University Medical Center

Stony Brook, New York, 11794, United States

Location

Wake Forest Baptist Medical Center - PPDS

Winston-Salem, North Carolina, 27157-0001, United States

Location

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229-3026, United States

Location

North Star Medical Research, LLC

Middleburg Heights, Ohio, 44130, United States

Location

Suburban Research Associates

Media, Pennsylvania, 19063, United States

Location

Coastal Pediatric Research

Charleston, South Carolina, 29414, United States

Location

Access Clinical Trials, Inc.

Nashville, Tennessee, 37203-6502, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-7610, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Texas Children's Hospital (TCH)

Houston, Texas, 77030, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Perceptive Pharma Research (PPR)

Richmond, Texas, 77407, United States

Location

Road Runner Research Ltd.

San Antonio, Texas, 78249-3539, 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

DCC Mladost-M Varna

Varna, 9020, Bulgaria

Location

University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

Location

The Kids Clinic Inc

Ajax, Ontario, L1Z 0M1, Canada

Location

Borne og Ungeafdelingen

Herlev, 02730, Denmark

Location

CHU Grenoble Alpes - Hopital Couple Enfant

Grenoble, 38000, France

Location

Hôpital Fondation Rothschild

Paris, 75019, France

Location

CHU Strasbourg-Hopital de Hautepierre

Strasbourg, 67098, France

Location

Psychiatric Clinic of Ludwig Maximilians Universitaet Muenchen

Munich, Bavaria, 80336, Germany

Location

Department of Psychiatry, Socialpsychiatry and Psychotherapy, Hannover Medical School

Hanover, Lower Saxony, 30625, Germany

Location

Zentralinstitut fuer Seelische Gesundheit, Mannheim

Mannheim, 68159, Germany

Location

Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek

Szeged, Csongrád megye, 6725, Hungary

Location

Vadaskert Gyermek- es Ifjusagpszichiatriai Korhaz es Szakambulancia

Budapest, 1021, Hungary

Location

Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)

Budapest, 1146, Hungary

Location

Istituto Di Ricovero E Cura A Carattere Scientifico IRCCS Eugenio Medea

Bosisio Parini, LC, 23842, Italy

Location

IRCCS Istituto Neurologico Carlo Besta

Milan, Milano, 20133, Italy

Location

Universita degli Studi di Napoli Federico II

Napoli, Napoli, 80131, Italy

Location

Ospedale Pediatrico Bambino Gesù

Roma, RM, 00165, Italy

Location

Azienda Ospedaliera Vittorio Emanuele Policlinico // Azienda Ospedaliera Policlinico San Marco

Catania, 95123, Italy

Location

Ospedale Pediatrico Istituto Giannina Gaslini di Genova

Genova, 16148, Italy

Location

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

Poznan, Poland, 61-731, Poland

Location

Gdanskie Centrum Zdrowia Sp z o.o.

Gdansk, Pomeranian Voivodeship, 80-542, Poland

Location

Wielospecjalistyczna Poradnia Lekarska Synapsis

Katowice, Silesian Voivodeship, 42-123, 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

Spitalul Clinic de Psihiatrie Prof Dr Al. Obregia Bucureti

Dorobanți, 41914, Romania

Location

Institute of Mental Health

Belgrade, Belgrad, 11120, Serbia

Location

Clinical Centre Nis Center of Mental Health

Niš, Serbia, 18000, Serbia

Location

Clinical Center Vojvodina

Novi Sad, Serbia, 21000, Serbia

Location

Clinic of Neurology and Psychiatry for Children and Adolescents

Belgrade, 11000, Serbia

Location

Hospital Universitario Fundacion Alcorcon HUFA

Alcorcón, Madrid, 28922, Spain

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, C/Antonio Maura Montaner, s/n, edificio IBiS

Seville, Sevilla, 41013, Spain

Location

Hospital Clínic de Barcelona

Barcelona, 8036, Spain

Location

Hospital Universitario Virgen Macarena Unidad de Investigacion Neurologia

Seville, 41009, Spain

Location

MeSH Terms

Conditions

Tourette Syndrome

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Drug Development and Medical Affairs
• Organization: Emalex Biosciences Inc.

mmiller@emalexbiosciences.com

773 343 0671

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: This includes an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Sequential Assignment This is a multicenter study which includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.

Study Record Dates

• First Submitted: November 7, 2022
• First Posted: November 14, 2022
• Study Start: January 31, 2023
• Primary Completion: January 13, 2025
• Study Completion: February 4, 2025
• Last Updated: November 25, 2025
• Results First Posted: November 25, 2025

Record last verified: 2025-04

Locations

DK (1); IT (6); RO (1); SE (4); HU (3); US (56); DE (3); BU (4); CA (2); PL (5); ES (7); FR (3)