Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults
- Registration Number
- NCT05615220
- Lead Sponsor
- Emalex Biosciences Inc.
- 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 double-blind, placebo-controlled, randomized withdrawal period.
- Detailed Description
Following a 28-day Screening period and Baseline visit, eligible subjects will be enrolled into the open-label Stabilization period comprised of a 4-week Titration phase to achieve a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) followed by an 8-week open-label Maintenance phase. Responders to ecopipam will be randomized to ecopipam 1.8 mg/kg/day (2 mg/kg/day ecopipam HCl) or placebo in a 1:1 fashion and enter the double-blind Randomized-Withdrawal (R/WD) period at Week 12. During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 216
- ≥ 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
- Previous exposure to ecopipam
- Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder)
- Unstable 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Placebo during R/WD Phase Ecopipam Hydrochloride Matching Placebo tablets during R/WD period taken orally in the evening. 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) Ecopipam Hydrochloride 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.
- Primary Outcome Measures
Name Time Method Yale Global Tic Severity Scale (YGTSS) Screening up to Week 24 Clinician-completed rating scale with score ranging from 0 to 50 (0=none to 50=severe)
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (95)
Borne og Ungeafdelingen
🇩🇰Herlev, Denmark
CHU Grenoble Alpes - Hopital Couple Enfant
🇫🇷Grenoble, France
Hôpital Fondation Rothschild
🇫🇷Paris, France
CHU Strasbourg-Hopital de Hautepierre
🇫🇷Strasbourg, France
Psychiatric Clinic of Ludwig Maximilians Universitaet Muenchen
🇩🇪Muenchen, Bayern, Germany
Department of Psychiatry, Socialpsychiatry and Psychotherapy, Hannover Medical School
🇩🇪Hannover, Niedersachsen, Germany
Zentralinstitut fuer Seelische Gesundheit, Mannheim
🇩🇪Mannheim, Germany
Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek
🇭🇺Szeged, Csongrád, Hungary
Vadaskert Gyermek- es Ifjusagpszichiatriai Korhaz es Szakambulancia
🇭🇺Budapest, Hungary
Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)
🇭🇺Budapest, Hungary
Istituto Di Ricovero E Cura A Carattere Scientifico IRCCS Eugenio Medea
🇮🇹Bosisio Parini, LC, Italy
Harmonex Neuroscience Research
🇺🇸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
Amnova Clinical Research
🇺🇸Irvine, California, United States
Cortica Site Network - San Rafael
🇺🇸San Rafael, California, United States
Syrentis Clinical Research
🇺🇸Santa Ana, California, United States
Yale School of Medicine
🇺🇸New Haven, Connecticut, United States
University of Florida
🇺🇸Gainesville, Florida, United States
NW FL Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, United States
Research in Miami Inc
🇺🇸Hialeah, Florida, United States
Emcrown Clinical Research
🇺🇸Jensen Beach, Florida, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Florida International Research Center
🇺🇸Miami, Florida, United States
Care 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
Jedidiah Clinical Research
🇺🇸Tampa, Florida, United States
Pediatric Neurology, PA
🇺🇸Winter Park, Florida, United States
Advanced Discovery Research, LLC
🇺🇸Atlanta, Georgia, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
Atlanta Behavioral Research, LLC.
🇺🇸Atlanta, Georgia, United States
Lurie Children Hospital of Chicago
🇺🇸Chicago, Illinois, United States
1725 W. Harrison St., Suite 755
🇺🇸Chicago, Illinois, United States
The University of Chicago Hospitals
🇺🇸Chicago, Illinois, United States
Josephson-Wallack-Munshower Neurology
🇺🇸Indianapolis, Indiana, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Umass Chan Medical School
🇺🇸Worcester, Massachusetts, United States
Michigan Clinical Research Institute PC
🇺🇸Ann Arbor, Michigan, United States
Neurobehavioral Medicine Group
🇺🇸Bloomfield Hills, Michigan, United States
The Childrens Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Midwest Research Group
🇺🇸Saint Charles, Missouri, United States
Washington University School of Medicine
🇺🇸St. Louis, Missouri, United States
Alivation Research
🇺🇸Lincoln, Nebraska, United States
NYU Child Study Center
🇺🇸New York, New York, United States
Mount Sinai School of Medicine
🇺🇸New York, New York, United States
University of Rochester
🇺🇸Rochester, New York, United States
Stony Brook University Medical Center
🇺🇸Stony Brook, New York, United States
Wake Forest Baptist Medical Center - PPDS
🇺🇸Winston Salem, North Carolina, United States
Cincinnati Childrens Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
North Star Medical Research, LLC
🇺🇸Middleburg Heights, Ohio, United States
Suburban Research Associates
🇺🇸Media, Pennsylvania, United States
Coastal Pediatric Research
🇺🇸Charleston, South Carolina, United States
Access Clinical Trials, Inc.
🇺🇸Nashville, Tennessee, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
Texas Children's Hospital (TCH)
🇺🇸Houston, Texas, United States
The University of Texas Health Science Center at Houston
🇺🇸Houston, Texas, United States
Perceptive Pharma Research (PPR)
🇺🇸Richmond, 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
ASMP-IP- d-r Kayryakova
🇧🇬Sofia, Sofia City, Bulgaria
Center Spectar-Plovdiv
🇧🇬Plovdiv, Bulgaria
Kalimat Medical Center_Sofia
🇧🇬Sofia, Bulgaria
DCC Mladost-M Varna
🇧🇬Varna, Bulgaria
University of Calgary
🇨🇦Calgary, Alberta, Canada
The Kids Clinic Inc
🇨🇦Ajax, Ontario, Canada
Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, RM, Italy
Azienda Ospedaliera Vittorio Emanuele Policlinico // Azienda Ospedaliera Policlinico San Marco
🇮🇹Catania, Italy
Ospedale Pediatrico Istituto Giannina Gaslini di Genova
🇮🇹Genova, Italy
IRCCS Istituto Neurologico Carlo Besta
🇮🇹Milano, Italy
Universita degli Studi di Napoli Federico II
🇮🇹Napoli, Italy
Gdanskie Centrum Zdrowia Sp z o.o.
🇵🇱Gdańsk, Pomorskie, Poland
Wielospecjalistyczna Poradnia Lekarska Synapsis
🇵🇱Katowice, Silesia, Poland
Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
🇵🇱Krakow, Woj. Malopolskie, Poland
Centrum Medyczne Plejady
🇵🇱Krakow, Poland
Clinical Research Center Sp. z o.o. MEDIC-R Sp.k.
🇵🇱Poznań, Poland
Spitalul Clinic de Psihiatrie Prof Dr Al. Obregia Bucureti
🇷🇴Dorobanti, Romania
Institute of Mental Health
🇷🇸Belgrade, Belgrad, Serbia
Clinic of Neurology and Psychiatry for Children and Adolescents
🇷🇸Belgrade, Serbia
Clinical Centre Nis Center of Mental Health
🇷🇸Nis, Serbia
Hospital Clínic de Barcelona
🇪🇸Barcelona, Spain
Hospital General Universitario Gregorio Maranon
🇪🇸Madrid, Spain
Clinical Center Vojvodina
🇷🇸Novi Sad, Serbia
Hospital Universitario Central de Asturias
🇪🇸Oviedo, Asturias, Spain
Hospital Universitario Fundacion Alcorcon HUFA
🇪🇸Alcorcón, Madrid, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Universitario Virgen Macarena Unidad de Investigacion Neurologia
🇪🇸Sevilla, Spain
Hospital Universitario Virgen del Rocío, C/Antonio Maura Montaner, s/n, edificio IBiS
🇪🇸Sevilla, Spain