Clinical trial NCT05615220: Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults
Official title: A Multicenter, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Maintenance of Efficacy of Ecopipam in Children, Adolescents and Adults With Tourette's Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Tourette Disorder
Lead sponsor: Emalex Biosciences Inc.
Sponsor class: INDUSTRY
First posted: 2022-11-14
Enrollment: 216
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: This is a multicenter study which includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.
Primary purpose: TREATMENT

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 During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.

Eligibility criteria: Inclusion Criteria:

* ≥ 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Yale Global Tic Severity Scale (YGTSS)
timeFrame: Screening up to Week 24
Secondary outcomes:
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Harmonex Neuroscience Research
state: Alabama
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Anaheim
country: United States
facility: Advanced Research Center
state: California
city: Bellflower
country: United States
facility: CenExel CIT-IE
state: California
city: Glendale
country: United States
facility: Cortica Site Network
state: California
city: Irvine
country: United States
facility: Amnova Clinical Research
state: California
city: San Rafael
country: United States
facility: Cortica Site Network - San Rafael
state: California
city: Santa Ana
country: United States
facility: Syrentis Clinical Research
state: California
city: New Haven
country: United States
facility: Yale School of Medicine
state: Connecticut
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Gulf Breeze
country: United States
facility: NW FL Clinical Research Group, LLC
state: Florida
city: Hialeah
country: United States
facility: Research in Miami Inc
state: Florida
city: Jensen Beach
country: United States
facility: Emcrown Clinical Research
state: Florida
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Miami
country: United States
facility: Florida International Research Center
state: Florida
city: Miami
country: United States
facility: Care Research Center
state: Florida
city: Orange City
country: United States
facility: Medical Research Group of Central Florida
state: Florida
city: Orlando
country: United States
facility: APG Research LLC
state: Florida
city: St. Petersburg
country: United States
facility: University of South Florida
state: Florida
city: Tampa
country: United States
facility: Pediatric Epilepsy and Neurology Specialists
state: Florida
city: Tampa
country: United States
facility: Jedidiah Clinical Research
state: Florida
city: Winter Park
country: United States
facility: Pediatric Neurology, PA
state: Florida
city: Atlanta
country: United States
facility: Advanced Discovery Research, LLC
state: Georgia
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Atlanta
country: United States
facility: Atlanta Behavioral Research, LLC.
state: Georgia
city: Chicago
country: United States
facility: Lurie Children Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: 1725 W. Harrison St., Suite 755
state: Illinois
city: Chicago
country: United States
facility: The University of Chicago Hospitals
state: Illinois
city: Indianapolis
country: United States
facility: Josephson-Wallack-Munshower Neurology
state: Indiana
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Worcester
country: United States
facility: Umass Chan Medical School
state: Massachusetts
city: Ann Arbor
country: United States
facility: Michigan Clinical Research Institute PC
state: Michigan
city: Bloomfield Hills
country: United States
facility: Neurobehavioral Medicine Group
state: Michigan
city: Kansas City
country: United States
facility: The Childrens Mercy Hospital
state: Missouri
city: Saint Charles
country: United States
facility: Midwest Research Group
state: Missouri
city: St. Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Lincoln
country: United States
facility: Alivation Research
state: Nebraska
city: New York
country: United States
facility: NYU Child Study Center
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Stony Brook
country: United States
facility: Stony Brook University Medical Center
state: New York
city: Winston Salem
country: United States
facility: Wake Forest Baptist Medical Center - PPDS
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Childrens Hospital Medical Center
state: Ohio
city: Middleburg Heights
country: United States
facility: North Star Medical Research, LLC
state: Ohio
city: Media
country: United States
facility: Suburban Research Associates
state: Pennsylvania
city: Charleston
country: United States
facility: Coastal Pediatric Research
state: South Carolina
city: Nashville
country: United States
facility: Access Clinical Trials, Inc.
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital (TCH)
state: Texas
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
city: Richmond
country: United States
facility: Perceptive Pharma Research (PPR)
state: Texas
city: San Antonio
country: United States
facility: Road Runner Research Ltd.
state: Texas
city: Draper
country: United States
facility: Cedar Clinical Research
state: Utah
city: Everett
country: United States
facility: Core Clinical Research
state: Washington
city: Sofia
country: Bulgaria
facility: ASMP-IP- d-r Kayryakova
state: Sofia City
city: Plovdiv
country: Bulgaria
facility: Center Spectar-Plovdiv
city: Sofia
country: Bulgaria
facility: Kalimat Medical Center_Sofia
city: Varna
country: Bulgaria
facility: DCC Mladost-M Varna
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Ajax
country: Canada
facility: The Kids Clinic Inc
state: Ontario
city: Herlev
country: Denmark
facility: Borne og Ungeafdelingen
city: Grenoble
country: France
facility: CHU Grenoble Alpes - Hopital Couple Enfant
city: Paris
country: France
facility: Hôpital Fondation Rothschild
city: Strasbourg
country: France
facility: CHU Strasbourg-Hopital de Hautepierre
city: Muenchen
country: Germany
facility: Psychiatric Clinic of Ludwig Maximilians Universitaet Muenchen
state: Bayern
city: Hannover
country: Germany
facility: Department of Psychiatry, Socialpsychiatry and Psychotherapy, Hannover Medical School
state: Niedersachsen
city: Mannheim
country: Germany
facility: Zentralinstitut fuer Seelische Gesundheit, Mannheim
city: Szeged
country: Hungary
facility: Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek
state: Csongrád
city: Budapest
country: Hungary
facility: Vadaskert Gyermek- es Ifjusagpszichiatriai Korhaz es Szakambulancia
city: Budapest
country: Hungary
facility: Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)
city: Bosisio Parini
country: Italy
facility: Istituto Di Ricovero E Cura A Carattere Scientifico IRCCS Eugenio Medea
state: LC
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
state: RM
city: Catania
country: Italy
facility: Azienda Ospedaliera Vittorio Emanuele Policlinico // Azienda Ospedaliera Policlinico San Marco
city: Genova
country: Italy
facility: Ospedale Pediatrico Istituto Giannina Gaslini di Genova
city: Milano
country: Italy
facility: IRCCS Istituto Neurologico Carlo Besta
city: Napoli
country: Italy
facility: Universita degli Studi di Napoli Federico II
city: Gdańsk
country: Poland
facility: Gdanskie Centrum Zdrowia Sp z o.o.
state: Pomorskie
city: Katowice
country: Poland
facility: Wielospecjalistyczna Poradnia Lekarska Synapsis
state: Silesia
city: Krakow
country: Poland
facility: Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
state: Woj. Malopolskie
city: Krakow
country: Poland
facility: Centrum Medyczne Plejady
city: Poznań
country: Poland
facility: Clinical Research Center Sp. z o.o. MEDIC-R Sp.k.
city: Dorobanti
country: Romania
facility: Spitalul Clinic de Psihiatrie Prof Dr Al. Obregia Bucureti
city: Belgrade
country: Serbia
facility: Institute of Mental Health
state: Belgrad
city: Belgrade
country: Serbia
facility: Clinic of Neurology and Psychiatry for Children and Adolescents
city: Nis
country: Serbia
facility: Clinical Centre Nis Center of Mental Health
city: Novi Sad
country: Serbia
facility: Clinical Center Vojvodina
city: Oviedo
country: Spain
facility: Hospital Universitario Central de Asturias
state: Asturias
city: Alcorcón
country: Spain
facility: Hospital Universitario Fundacion Alcorcon HUFA
state: Madrid
city: Barcelona
country: Spain
facility: Hospital Clínic de Barcelona
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Maranon
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen Macarena Unidad de Investigacion Neurologia
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío, C/Antonio Maura Montaner, s/n, edificio IBiS
Study officials:
References: