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

NCT02102698 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: PSY302
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 11

Brief Summary

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.

Conditions

Tourette's Syndrome

Keywords

Tourette's Syndrome; Ecopipam; D1 Receptor; Phase 2

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• YGTSS Day 16

  Baseline and Day 16

• DuPaul ADHD (Attention Deficit Hyperactivity Disorder) Rating Scale-IV

  Baseline and 30 days

• Child Yale-Brown Obsessive Compulsive Scale

  Baseline and 30 days

• Clinical Global Impression Scale - Severity

  Baseline and 30 days

Study Arms (2)

Ecopipam/Placebo

EXPERIMENTAL

Given Ecopipam first and then crossed over to Placebo

Drug: Ecopipam

Placebo/Ecopipam

EXPERIMENTAL

Given placebo first and crossed over to Ecopipam

Drug: Ecopipam

Interventions

Ecopipam DRUG

Ecopipam is a selective antagonist of the dopamine D1 receptor family.

Ecopipam/Placebo; Placebo/Ecopipam

Eligibility Criteria

• Age: 7 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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:

Sponsors & Collaborators

• Psyadon Pharma: collaborator
• Emalex Biosciences Inc.: lead

Study Sites (11)

UCLA

Los Angeles, California, 90024, United States

Location

University of South Florida/Rothman Center for Neuropsychiatry

St. Petersburg, Florida, 33701, United States

Location

Emory University School Of Medicine

Atlanta, Georgia, 30322, United States

Location

University of Chicago Medical School

Chicago, Illinois, 60637, United States

Location

Children's Mercy Kansas City

Kansas City, Kansas, 64108, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Overlook Hospital

Summit, New Jersey, 07902, United States

Location

North Shore-Long Island Jewish Hosptial

Manhasset, New York, 11030, United States

Location

Weill Cornell Medical School

New York, New York, 10065, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45228, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Publications (2)

• 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.

  PMID: 24434529 BACKGROUND

• 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.

  PMID: 30192018 DERIVED

MeSH Terms

Conditions

Tourette Syndrome

Interventions

ecopipam

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: David Kim
• Organization: Emalex Bosciences

dkim@emalexbiosciences.com

312.847.1342

Study Officials

• Donald Gilbert, MD

  Children's Hospital Medical Center, Cincinnati

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2014
• First Posted: April 3, 2014
• Study Start: June 19, 2014
• Primary Completion: December 1, 2019
• Study Completion: December 1, 2019
• Last Updated: May 22, 2026
• Results First Posted: May 22, 2026

Record last verified: 2026-05

Locations

US (11)