Ecopipam Treatment of Tourette's Syndrome in Subjects 7-17 Years

Tourette's Syndrome is a neurological disease characterized by motor and vocal tics. It has been hypothesized that abnormal interactions of dopamine with its receptors may cause the tics. The purpose of this study is to test the hypothesis that a drug (ecopipam) that selectively blocks dopamine D1/D5 receptors can reduce the frequency and severity of the tics.

Study Overview

• Status: Completed
• Conditions: Tourette's Syndrome
• Intervention / Treatment: Drug: Ecopipam

Detailed Description

This is a double blind, randomized, placebo-controlled crossover study to determine whether ecopipam can reduce the symptoms of Tourette's in children age 7-17 years.

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90024
      • UCLA
  • Florida
    • St. Petersburg, Florida, United States, 33701
      • University of South Florida/Rothman Center for Neuropsychiatry
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University School of Medicine
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Medical School
  • Kansas
    • Kansas City, Kansas, United States, 64108
      • Children's Mercy Kansas City
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New Jersey
    • Summit, New Jersey, United States, 07902
      • Overlook Hospital
  • New York
    • Manhasset, New York, United States, 11030
      • North Shore-Long Island Jewish Hosptial
    • New York, New York, United States, 10065
      • Weill Cornell Medical School
  • Ohio
    • Cincinnati, Ohio, United States, 45228
      • Cincinnati Children's Hospital
  • Texas
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:• Subjects must have Tourette's Syndrome (TS) based on the clinician-administered Diagnostic Confidence Index (DCI) for TS.

• Subjects must exhibit both motor and vocal tics.
• Subjects must have a minimum score of 20 at both Screening and Baseline (just prior to the first treatment) on the Yale Global Tic Severity Scale.
• Subjects must be age (≥ 7 to < 18 years of age)
• Subjects must weigh ≥ 20 kg (45 lbs)
• Adolescent females of childbearing potential who are sexually active must be using effective contraception (i.e., oral contraceptives, intrauterine device, double barrier method of condom and spermicide) and agree to continue use of contraception for the duration of their participation in the study. They must also agree to use contraception for 30 days after their last dose of study drug.
• Sexually active male subjects must use a barrier method of contraception during the study and agree to continue the use of male contraception for at least 30 days after the last dose of study drug.
• Subject's parent or legal guardian must execute a written informed consent.
• Subject must execute a written informed assent.

Exclusion Criteria:

• Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests, or ECG at Screening.
• Subjects with a major depressive episode in the past 2 years
• Subjects with a history of attempted suicide
• Subjects with clinically significant suicidality (based on the Columbia Suicide Rating Scale (C-SSRS)
• Subjects with a first-degree relative with a major depressive episode that resulted in any psychiatric hospitalization, or attempted/ completed suicide with the exception of a hospitalization for post-partum depression.
• Subjects with a history of seizures (excluding febrile seizures that occurred >2 years in the past)
• Subjects with a myocardial infarction within 6 months.
• Girls who are currently pregnant or lactating.
• Subjects who have a need for medication (other than ecopipam) with possible effects on TS symptoms (i.e., lithium, psychostimulants)
• Subjects who have a need for medications which would have unfavorable interactions with ecopipam, e.g., dopamine antagonists or agonists [including bupropion], tetrabenazine, or monoamine oxidase inhibitors.
• Subjects with a lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any psychotic disorder determined by the Structured Clinical Interview for Diagnostic and Statistical Manual of Mental Disorders - 4th Edition (DSM-IV) Axis-I Disorders (SCID).
• Subjects with current or recent (past 3 months) DSM-IV substance abuse or dependence (with the exception of nicotine).
• Subjects with positive urine drug screen (cocaine, amphetamine, methamphetamine, tetrahydrocannabinol (THC), benzodiazepines, barbiturates, phencyclidine (PCP), opiates) at Screening. Subjects with urine positive only for benzodiazepines and/or marijuana (i.e., a user but not an abuser as based on DSM-IV criteria) may be eligible.
• Subjects who have had previous treatment with ecopipam.

• Subjects who have had treatment with:

  • investigational medication within 3 months of starting study
  • depot neuroleptics within 3 months of starting study
  • other psychotropics with possible effects on TS symptoms (i.e., lithium, tetrabenazine) within 2 weeks prior to Screening.
  • oral neuroleptics within 4 weeks
  • selective serotonin reuptake inhibitors unless the dosage has been stable for a minimum of 4 weeks prior to study start and not prescribed to relieve the neurological signs of TS

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ecopipam/Placebo; Given Ecopipam first and then crossed over to Placebo	Drug: Ecopipam; Ecopipam is a selective antagonist of the dopamine D1 receptor family.; Other Names: SCH 39166; PSYRX101
Experimental: Placebo/Ecopipam; Given placebo first and crossed over to Ecopipam	Drug: Ecopipam; Ecopipam is a selective antagonist of the dopamine D1 receptor family.; Other Names: SCH 39166; PSYRX101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Yale Global Tic Severity Scale - Total Tic Score	The Yale Global Tic Severity Scale (YGTSS) is the standard rating scale used to assess the effects of a new treatment on the symptoms of Tourette's Disorder (TD). The YGTSS is a clinician-rated, multi-dimensional instrument for assessing tic symptom severity in children and adults with TD. Both motor and vocal tics are assessed for symptom number, frequency, intensity, complexity, and interference on a 0-5 Likert scale. Scores from each dimension are totaled to reflect the severity of motor tics (range 0-25) (min of 0 to max 25), vocal tics (range 0-25) (min of 0 to max 25) and combined tics, or Total Tic Score (TTS) (range 0-50) (min of 0 to max of 50, sum of motor tics and vocal tic scores). For all scores, lower score represents better outcome. The primary outcome measure is the YGTSS-TTS (Yale Global Tic Severity Scale Total Tic Score which includes both the motor and phonic tic scores).	Baseline and 30 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
YGTSS Day 16	The Yale Global Tic Severity Scale (YGTSS) is the standard rating scale used to assess the effects of a new treatment on the symptoms of Tourette's Disorder (TD). The YGTSS is a clinician-rated, multi-dimensional instrument for assessing tic symptom severity in children and adults with TD. Both motor and vocal tics are assessed for symptom number, frequency, intensity, complexity, and interference on a 0-5 Likert scale. Scores from each dimension are totaled to reflect the severity of motor tics (range 0-25) (min of 0 to max 25), vocal tics (range 0-25) (min of 0 to max 25) and combined tics, or Total Tic Score (TTS) (range 0-50) (min of 0 to max of 50, sum of motor tics and vocal tic scores). For all scores, lower score represents better outcome. The outcome measure described is the YGTSS-TTS (Yale Global Tic Severity Scale Total Tic Score which includes both the motor and phonic tic scores) at Day 16 vs baseline.	Baseline and Day 16
DuPaul ADHD (Attention Deficit Hyperactivity Disorder) Rating Scale-IV	This is a validated rating scale for the symptoms of attention deficit disorder. Scale includes 18 core items, with higher score indicating greater severity of ADHD (Total score 0-54). These scores are compared from baseline and 30 days below; There are also subscales but not individually shown. The subscales include 9 items measured for Inattention; 9 for Hyperactivity/Impulsivity. A score of 2 or 3 on any item is considered clinically significant. Measure on 4-point Likert scale and also include optional performance items. Raw scores are summed for each subscale. Scores are compared to norm-referenced percentiles based on age and gender. Higher scores may indicate ADHD.	Baseline and 30 days
Child Yale-Brown Obsessive Compulsive Scale	The CY-BOCS is a clinician-rated, 10-item scale to both determine severity of OCD and to monitor improvement during treatment, with a higher score indicating greater severity. The scale is a clinician-rated, 10-item scale (5 items regarding severity and 5 items regarding obsessions) that includes questions about the amount of time spent on obsessions/compulsions, level of impairment or distress, and how much resistance and control subjects have over these thoughts. The 10 items are assessed on a 6-point scale with an overall score. Total score from 0 to 40 with higher score indicating greater severity. Total score change shown below.	Baseline and 30 days
Clinical Global Impression Scale - Severity	Clinical Global Impression Scales (improvement and severity) are validated rating scales that measure whether the treatment improves the symptoms of the disease (CGI-I) and whether the treatment reduces the severity of the disease (CGI-S). The CGI consists of two reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms. The severity scale (CGI-S) ranges from 1 (Normal, not ill) to 7 (extremely ill). The improvement scale (CGI-I) ranges from 1 (very much improved) to 7 (very much worse) with a score of 1 or 2 defining positive response. The improvement rating compares the subject's overall clinical condition to the Baseline visit and the rater should consider the following question: "Compared to tic severity at baseline, how much has the subject changed?" Data shows how much improvement at Day 30 with Ecopipam vs Placebo.	Baseline and 30 days

Collaborators and Investigators

• Sponsor: Emalex Biosciences Inc.
• Collaborators: Psyadon Pharma
• Investigators: Study Chair: Donald Gilbert, MD, Children's Hospital Medical Center, Cincinnati

Publications and helpful links

General Publications

• Gilbert DL, Budman CL, Singer HS, Kurlan R, Chipkin RE. A D1 receptor antagonist, ecopipam, for treatment of tics in Tourette syndrome. Clin Neuropharmacol. 2014 Jan-Feb;37(1):26-30. doi: 10.1097/WNF.0000000000000017.
• Gilbert DL, Murphy TK, Jankovic J, Budman CL, Black KJ, Kurlan RM, Coffman KA, McCracken JT, Juncos J, Grant JE, Chipkin RE. Ecopipam, a D1 receptor antagonist, for treatment of tourette syndrome in children: A randomized, placebo-controlled crossover study. Mov Disord. 2018 Aug;33(8):1272-1280. doi: 10.1002/mds.27457. Epub 2018 Sep 7.

Study record dates

Study Major Dates

• Study Start (Actual): June 19, 2014
• Primary Completion (Actual): December 1, 2019
• Study Completion (Actual): December 1, 2019

Study Registration Dates

• First Submitted: March 31, 2014
• First Submitted That Met QC Criteria: March 31, 2014
• First Posted (Estimated): April 3, 2014

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 19, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Tourette's Syndrome; Phase 2; Ecopipam; D1 Receptor
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Mental Disorders; Genetic Diseases, Inborn; Neurodegenerative Diseases; Neurodevelopmental Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Basal Ganglia Diseases; Tic Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Tourette Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Antipsychotic Agents; Tranquilizing Agents; Psychotropic Drugs; Dopamine Agents; Dopamine Antagonists; ecopipam
• Other Study ID Numbers: PSY302

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No