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| Name | Class |
|---|---|
| Psyadon Pharma | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ecopipam/Placebo | Experimental | Given Ecopipam first and then crossed over to Placebo |
|
| Placebo/Ecopipam | Experimental | Given placebo first and crossed over to Ecopipam |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ecopipam | Drug | Ecopipam is a selective antagonist of the dopamine D1 receptor family. |
|
| 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 |
| 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. |
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Inclusion Criteria:• Subjects must have Tourette's Syndrome (TS) based on the clinician-administered Diagnostic Confidence Index (DCI) for TS.
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:
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| Name | Affiliation | Role |
|---|---|---|
| Donald Gilbert, MD | Children's Hospital Medical Center, Cincinnati | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA | Los Angeles | California | 90024 | United States | ||
| University of South Florida/Rothman Center for Neuropsychiatry |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24434529 | Background | 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. | |
| 30192018 | Derived | 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. |
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19 in Ecopipam/Placebo arm and 21 in Placebo/Ecopipam arm
First subject enrolled 19Jun2014 and last subject enrolled 17Nov2016
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| ID | Title | Description |
|---|---|---|
| FG000 | Ecopipam First, Then Placebo | Ecopipam first, then Placebo Treatment Phase 1: 30 days (ecopipam) Washout Phase 2: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Treatment Phase 3: 30 days (placebo) Follow-up Phase: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Ecopipam HCl tablets (12.5 mg/tablet and 50 mg/tablet) were administered orally each day at bed time. The dose was based on body weight at baseline (range 12.5 mg/day to 100 mg/day) following a protocol-specified regimen. Placebo tablets were administered orally each day at bed time. |
| FG001 | Placebo First Then Ecopipam | Placebo first then Ecopipam Treatment Phase 1: 30 days (placebo) Washout Phase 2: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Treatment Phase 3: 30 days (placebo) Follow-up Phase: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Placebo tablets were administered orally each day at bed time. Ecopipam HCl tablets (12.5 mg/tablet and 50 mg/tablet) were administered orally each day at bed time. The dose was based on body weight at baseline (range 12.5 mg/day to 100 mg/day) following a protocol-specified regimen. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Ecopipam First Then Placebo | Ecopipam first, then Placebo Treatment Phase 1: 30 days (ecopipam) Washout Phase 2: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Treatment Phase 3: 30 days (placebo) Follow-up Phase: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Ecopipam HCl tablets (12.5 mg/tablet and 50 mg/tablet) were administered orally each day at bed time. The dose was based on body weight at baseline (range 12.5 mg/day to 100 mg/day) following a protocol-specified regimen. Placebo tablets were administered orally each day at bed time. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | Crossover design. Both groups received Ecopipam and Placebo. Total numbers include 40 for each group. | Posted | Mean | Standard Deviation | Score on a scale | Baseline and 30 days |
|
The time period included from enrollment (first day of study drug) until follow up (14 days after the subject's last visit) or until resolution of the AE whichever came first. Time period from enrollment until follow up visit can be up to 88 days.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ecopipam | Treatment Phase 1: 30 days (ecopipam) Washout Phase 2: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Treatment Phase 3: 30 days (placebo) Follow-up Phase: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Ecopipam HCl tablets (12.5 mg/tablet and 50 mg/tablet) were administered orally each day at bed time. The dose was based on body weight at baseline (range 12.5 mg/day to 100 mg/day) following a protocol-specified regimen. Placebo tablets were administered orally each day at bed time. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Psychotic Disorder/Psychosis | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| David Kim | Emalex Bosciences | 312.847.1342 | dkim@emalexbiosciences.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 20, 2025 | Jun 6, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 21, 2016 | Jun 6, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D005879 | Tourette Syndrome |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C058081 | ecopipam |
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| 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 |
| St. Petersburg |
| Florida |
| 33701 |
| United States |
| Emory University School Of Medicine | Atlanta | Georgia | 30322 | United States |
| University of Chicago Medical School | Chicago | Illinois | 60637 | United States |
| Children's Mercy Kansas City | Kansas City | Kansas | 64108 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Overlook Hospital | Summit | New Jersey | 07902 | United States |
| North Shore-Long Island Jewish Hosptial | Manhasset | New York | 11030 | United States |
| Weill Cornell Medical School | New York | New York | 10065 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45228 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| BG001 | Placebo First Then Ecopipam | Placebo first then Ecopipam Treatment Phase 1: 30 days (ecopipam) Washout Phase 2: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Treatment Phase 3: 30 days (placebo) Follow-up Phase: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Ecopipam HCl tablets (12.5 mg/tablet and 50 mg/tablet) were administered orally each day at bed time. The dose was based on body weight at baseline (range 12.5 mg/day to 100 mg/day) following a protocol-specified regimen. Placebo tablets were administered orally each day at bed time. |
| BG002 | Total | Total of all reporting groups |
| Participants |
| No |
|
| Sex: Female, Male | Count of Participants | Participants | No |
|
| Race (NIH/OMB) | Count of Participants | Participants | No |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants | No |
|
| Region of Enrollment | Number | Participants |
|
| Description |
|---|
| OG000 | Ecopipam | Dosed with Ecopipam first and crossed over to Placebo |
| OG001 | Placebo | Dosed with Placebo first and crossed over to Ecopipam |
|
|
|
| Secondary | 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. | Crossover design. YGTSS at Day 16 | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline and Day 16 |
|
|
|
|
| Secondary | 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. | Change from Baseline at Day 30 | Posted | Mean | Standard Deviation | score on a scale | Baseline and 30 days |
|
|
|
| Secondary | 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. | Change from baseline at Day 30 | Posted | Mean | Standard Deviation | score on a scale | Baseline and 30 days |
|
|
|
| Secondary | 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. | CGI Severity | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | Baseline and 30 days |
|
|
|
| 0 |
| 40 |
| 0 |
| 40 |
| 32 |
| 40 |
| EG001 | Placebo | Treatment Phase 1: 30 days (ecopipam) Washout Phase 2: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Treatment Phase 3: 30 days (placebo) Follow-up Phase: 14 days (down-titration Days 1-4 and then washout/no study drug for 10 days) Ecopipam HCl tablets (12.5 mg/tablet and 50 mg/tablet) were administered orally each day at bed time. The dose was based on body weight at baseline (range 12.5 mg/day to 100 mg/day) following a protocol-specified regimen. Placebo tablets were administered orally each day at bed time. | 0 | 40 | 1 | 40 | 26 | 40 |
| Nausea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
|
| Decreased appetite | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 18.1 | Systematic Assessment |
|
| Nasopharyngitis | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
|
| Somnolence | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
|
| sedation | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
|
| Restlessness | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
|
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| D013981 | Tic Disorders |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |