Phase 2
N=40
Ecopipam Treatment of Tourette's Syndrome in Subjects 7-17 Years
Tourette's Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02102698 ↗Enrolled (actual)
40
Serious AEs
1.3%
Results posted
May 2026
Primary outcome: Primary: Yale Global Tic Severity Scale - Total Tic Score — -5.6; -3.4 Score on a scale — p=<0.05
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ecopipam (Drug)
- Age
- Pediatric · 7+ yrs
- Sex
- All
- Sponsor
- Emalex Biosciences Inc.
- Primary completion
- Dec 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Yale Global Tic Severity Scale - Total Tic Score |
-5.6; -3.4 | <0.05 sig |
| SECONDARY YGTSS Day 16 |
-6.1275; -1.9225 | <0.05 sig |
| SECONDARY DuPaul ADHD (Attention Deficit Hyperactivity Disorder) Rating Scale-IV |
-1.9; -2.7 | — |
| SECONDARY Child Yale-Brown Obsessive Compulsive Scale |
-0.8; -0.9 | — |
| SECONDARY Clinical Global Impression Scale - Severity |
-0.7637; -0.2293 | — |
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.
Eligibility Criteria
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 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
Data sourced from ClinicalTrials.gov (NCT02102698). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.