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Tourette's SyndromePhase 2NCT02102698

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

A Phase 2 study testing Ecopipam in people with Tourette's Syndrome, run by Emalex Biosciences Inc. at 11 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 7 to 17 years
Conditions the study is for
Tourette's Syndrome
What is being tested?
Ecopipam
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
11 sitesUnited States
Study dates
19 Jun 2014to Dec 2019
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 7 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

None listed.

Who cannot join

  • 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)
Show all 20Show fewer
  • 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

Trial locations and contacts

11 sites in 1 country.

11 sites in 1 country

United States11 sites

  • Emory University School Of Medicine

    Atlanta, Georgia, 30322

  • University of Chicago Medical School

    Chicago, Illinois, 60637

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45228

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Children's Mercy Kansas City

    Kansas City, Kansas, 64108

  • UCLA

    Los Angeles, California, 90024

  • North Shore-Long Island Jewish Hosptial

    Manhasset, New York, 11030

  • Weill Cornell Medical School

    New York, New York, 10065

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • University of South Florida/Rothman Center for Neuropsychiatry

    St. Petersburg, Florida, 33701

  • Overlook Hospital

    Summit, New Jersey, 07902

Other trials in United States

About this study

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.

Read the 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.

Being tested
Ecopipam
Keywords
Tourette's Syndrome, Ecopipam, D1 Receptor, Phase 2
Medical subject headings
Tourette Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EcopipamDrug

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

Study groups in NCT02102698
Study groupRoleWhat they receive
Ecopipam/PlaceboExperimentalGiven Ecopipam first and then crossed over to Placebo
Receives: Drug: Ecopipam
Placebo/EcopipamExperimentalGiven placebo first and crossed over to Ecopipam
Receives: Drug: Ecopipam

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: Baseline and 30 days

Secondary outcomes (4)

  1. 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.

    Measured: Baseline and Day 16

  2. 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.

    Measured: Baseline and 30 days

  3. 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.

    Measured: Baseline and 30 days

  4. 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.

    Measured: Baseline and 30 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Apr 2014 (estimated)
  2. Study start19 Jun 2014
  3. Primary completionDec 2019
  4. Study completionDec 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
40 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02102698
Sponsor's protocol ID
PSY302
Status last verified
May 2026
First submitted
31 Mar 2014
Last updated
22 May 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

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.

    PubMed 24434529

  • 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.

    PubMed 30192018

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ecopipam in people with Tourette's Syndrome, run by Emalex Biosciences Inc. at 11 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 0 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 May 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Yale Global Tic Severity Scale - Total Tic Score (time frame: Baseline and 30 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02102698, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.

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