Clinical trial NCT06021522: A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder
Official title: A Multicenter, Open-Label, Study to Evaluate the Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
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
Lead sponsor: Emalex Biosciences Inc.
Sponsor class: INDUSTRY
First posted: 2023-09-01
Enrollment: 150
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
timeFrame: Baseline up to Month 24
Secondary outcomes:
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Harmonex, Inc.
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: San Rafael
country: United States
facility: Cortica Site Network - San Rafael
state: California
city: New Haven
country: United States
facility: Yale School of Medicine
state: Connecticut
city: Washington
country: United States
facility: Childrens National Hospital
state: District of Columbia
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: 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: 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: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Chicago
country: United States
facility: Lurie Children Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Josephson-Wallack-Munshower Neurology
state: Indiana
city: Louisville
country: United States
facility: University of Louisville Research Foundation Inc.
state: Kentucky
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Mass General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Childrens 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: Neurobahavioral Medicine Group
state: Michigan
city: St. Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Lincoln
country: United States
facility: Alivation Research
state: Nebraska
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Charlotte
country: United States
facility: OnSite Clinical Solutions LLC
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: National Childrens Hospital - The Ohio State University
state: Ohio
city: Middleburg Heights
country: United States
facility: North Star Medical Research, LLC
state: Ohio
city: Portland
country: United States
facility: Providence Brain and Spine Institute
state: Oregon
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: Access Clinical Trials, Inc.
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: UT Southwestern
state: Texas
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
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: Ajax
country: Canada
facility: The Kids Clinic Inc
state: Ontario
city: Budapest
country: Hungary
facility: Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)
city: Szeged
country: Hungary
facility: Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek-es Ifjusagpszicihatria
city: Genova
country: Italy
facility: Istituto Giannina Gaslini, Universita di Genova
state: GE
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Ges
state: RM
city: Catania
country: Italy
facility: Azienda Ospedaliero Universitaria " G. Rodolico - San Marco"
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: Poznań
country: Poland
facility: Clinical Research Center Sp. z o.o. MEDIC-R Sp.k.
state: Wielkopolska
city: Krakow
country: Poland
facility: Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
state: Woj. Malopolskie
city: Katowice
country: Poland
facility: Neurologia Śląska Centrum Medyczne
city: Kraków
country: Poland
facility: Centrum Medyczne Plejady
city: Belgrade
country: Serbia
facility: Institute of Mental Health
state: Belgrad
city: Beograd
country: Serbia
facility: Clinic of Neurology and Psychiatry for Children and Adolescents
state: Grad Beograd
city: Nis
country: Serbia
facility: Clinical Centre Nis Center of Mental Health
city: Oviedo
country: Spain
facility: Hospital Universitario Central de Asturias
state: Asturias
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 del Rocío
Study officials:
References: