Clinical trial NCT04007991: Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents
Official title: Multicenter, Placebo-Controlled, Double-Blind, Randomized, Parallel-Group, Phase 2b Study to Evaluate the Efficacy and Safety of Ecopipam in Children and Adolescents With Tourette's Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Tourette Syndrome
Lead sponsor: Emalex Biosciences Inc.
Sponsor class: INDUSTRY
First posted: 2019-07-05
Enrollment: 153
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study evaluates the effect of ecopipam tablets in children and adolescents in the treatment of Tourette's Syndrome (TS). Half of the participants will receive ecopipam tablets, while the other half will receive matching placebo tablets

Detailed description: Multicenter, placebo-controlled, double-blind, randomized, parallel-group, Phase 2b study in pediatric subjects (aged greater than equal to 6 to less than 18 years of age) with TS. Following a 28-day Screening period and Baseline visit, eligible subjects will be randomized 1:1 to receive either ecopipam hydrochloride (HCl) or matching placebo for a 12 week treatment period. Doses will be titrated up and down from target dose of 2 mg/kg/day. Follow Up visit will be conducted after the last dose of study medication.

Eligibility criteria: Inclusion Criteria:

* ≥ 6 and \< 18 years of age
* ≥ 18 kg (\~ 40 lbs.)
* TS diagnosis and both motor and vocal tics that cause impairment with normal routines
* Minimum score of 20 on the YGTSS-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:

* 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

Primary outcomes:
measure: Change From Baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at Week 12
timeFrame: Baseline, Week 12
Secondary outcomes:
measure: Change From Baseline in Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) at Week 12
timeFrame: Baseline, Week 12
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Harmonex Neuroscience Research
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Anaheim
country: United States
facility: Advanced Research Center Inc.
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: San Diego
country: United States
facility: PCSD-Feighner Research
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: Sarkis Clinical Trials
state: Florida
city: Gulf Breeze
country: United States
facility: Northwest Florida 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
state: Florida
city: North Miami
country: United States
facility: MedBio Trials
state: Florida
city: Orlando
country: United States
facility: APG Research LLC
state: Florida
city: Saint 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: Winter Park
country: United States
facility: Pediatric Neurology, PA
state: Florida
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Savannah
country: United States
facility: Meridian Clinical Research
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Chicago
country: United States
facility: The University of Chicago Hospitals
state: Illinois
city: Naperville
country: United States
facility: AMR - Baber Research Inc.
state: Illinois
city: Overland Park
country: United States
facility: Psychiatric Associates
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
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: Wyoming
country: United States
facility: Helen DeVos Children's Hospital / Spectrum Health Medical Group
state: Michigan
city: Saint Charles
country: United States
facility: St. Charles Psychiatric Associates dba Midwest Research Group
state: Missouri
city: Saint Louis
country: United States
facility: Movement Disorders Center
state: Missouri
city: Lincoln
country: United States
facility: Alivation Research, LLC
state: Nebraska
city: Las Vegas
country: United States
facility: Center for Psychiatry and Behavioral Medicine Inc.
state: Nevada
city: Mount Arlington
country: United States
facility: The NeuroCognitive Institute
state: New Jersey
city: Voorhees
country: United States
facility: Clinical Research Center of NJ
state: New Jersey
city: New York
country: United States
facility: New York Neurology Associates P.C
state: New York
city: New York
country: United States
facility: Hapworth Research Inc.
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: New York
country: United States
facility: Mood Disorders Consulting Medicine PLLC
state: New York
city: Rochester
country: United States
facility: Finger Lakes Clinical Research
state: New York
city: Avon Lake
country: United States
facility: Quest Therapeutics of Avon Lake
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Childrens Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Cleveland 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: Bellaire
country: United States
facility: Houston Clinical Trials LLC
state: Texas
city: Dallas
country: United States
facility: Relaro Medical Trials
state: Texas
city: Fort Worth
country: United States
facility: North Texas Clinical Trials
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: San Antonio
country: United States
facility: Road Runner Research Ltd.
state: Texas
city: Springville
country: United States
facility: Noetic Psychiatry
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Everett
country: United States
facility: Eastside Therapeutic Resource Inc dba Core Clinical Research
state: Washington
city: Ajax
country: Canada
facility: The Kids Clinic Inc
state: Ontario
city: Ottawa
country: Canada
facility: Center for Pediatric Excellence
state: Ontario
city: Montreal
country: Canada
facility: CHU Sainte-Justine
state: Quebec
city: Paris
country: France
facility: Hopital Robert Debre
state: Ile De France
city: Poitiers
country: France
facility: CHU Poitiers
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: Freiburg
country: Germany
facility: Dept. Child Adolescent Psychiatry Uni. Freiburg
city: Mannheim
country: Germany
facility: Zentralinstitut fuer Seelische Gesundheit
city: Mittweida
country: Germany
facility: Pharmakologisches Studienzentrum Chemnitz GmbH
city: Gdansk
country: Poland
facility: Centrum Bada Klinicznych PI-House Sp. z o.o.
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Gdańsk
country: Poland
facility: Gdanskie Centrum Zdrowia Sp z o.o.
city: Katowice
country: Poland
facility: NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis
city: Krakow
country: Poland
facility: Centrum Medyczne Plejady
city: Krakow
country: Poland
facility: Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
city: Poznań
country: Poland
facility: Med-Polonia Sp. z o. o.
Study officials:
References: