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This study evaluates the effect of ecopipam tablets in children and adolescents in the treatment of Tourette's Syndrome (TS). Half of the participants will receive ecopipam tablets, while the other half will receive matching placebo tablets
Multicenter, placebo-controlled, double-blind, randomized, parallel-group, Phase 2b study in pediatric subjects (aged greater than equal to 6 to less than 18 years of age) with TS. Following a 28-day Screening period and Baseline visit, eligible subjects will be randomized 1:1 to receive either ecopipam hydrochloride (HCl) or matching placebo for a 12 week treatment period. Doses will be titrated up and down from target dose of 2 mg/kg/day. Follow Up visit will be conducted after the last dose of study medication.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/day | Experimental | Ecopipam HCl 12.5-, 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings |
|
| Placebo | Placebo Comparator | Matching Placebo tablets taken orally in the evening |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ecopipam | Drug | Ecopipam HCI tablets administered PO to establish 2 mg/kg/day |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at Week 12 | The YGTSS was a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0=none to 5=severe for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe) with higher score represent more severe symptoms. A negative change from baseline indicates improvement. | Baseline, Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) at Week 12 | Clinical Global Impression (CGI) scale was used to assess overall severity on a 7-point Likert scale consisted of 2 reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms. The CGI severity scale ranges from 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill subject. 1 = "normal, not ill at all" to 7 = "extremely ill." A negative change indicates improvement in the condition. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Harmonex Neuroscience Research | Dothan | Alabama | 36303 | United States | ||
| Phoenix Children's Hospital |
A total of 215 participants were screened, of which 61 participants were screen failures and 153 participants were randomized in 1:1 ratio to receive ecopipam HCl and placebo.
The study was conducted at 45 sites in 5 countries (United States, Canada, Germany, France, and Poland) from 28 June 2019 (first participant first visit) and 23 September 2021 (last participant last visit).
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| ID | Title | Description |
|---|---|---|
| FG000 | Ecopipam HCI 2 mg/kg/Day | Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks. |
| FG001 | Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Apr 13, 2020 | Aug 16, 2023 |
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| Placebo |
| Drug |
Matching Placebo |
|
| Baseline, Week 12 |
| Phoenix |
| Arizona |
| 85016 |
| United States |
| Advanced Research Center Inc. | Anaheim | California | 92805 | United States |
| UCLA | Los Angeles | California | 90095 | United States |
| PCSD-Feighner Research | San Diego | California | 92108 | United States |
| Syrentis Clinical Research | Santa Ana | California | 92705 | United States |
| Yale School of Medicine | New Haven | Connecticut | 06519 | United States |
| Sarkis Clinical Trials | Gainesville | Florida | 32607 | United States |
| Northwest Florida Clinical Research Group, LLC | Gulf Breeze | Florida | 32561 | United States |
| Research in Miami Inc. | Hialeah | Florida | 33013 | United States |
| University of Miami | Miami | Florida | 33136 | United States |
| MedBio Trials | North Miami | Florida | 33180 | United States |
| APG Research LLC | Orlando | Florida | 32803 | United States |
| University of South Florida | St. Petersburg | Florida | 33701-4825 | United States |
| Pediatric Epilepsy and Neurology Specialists | Tampa | Florida | 33609-4181 | United States |
| Pediatric Neurology, PA | Winter Park | Florida | 32789 | United States |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | United States |
| Meridian Clinical Research | Savannah | Georgia | 31406 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612-3841 | United States |
| The University of Chicago Hospitals | Chicago | Illinois | 60637-1447 | United States |
| AMR - Baber Research Inc. | Naperville | Illinois | 60563-6510 | United States |
| Psychiatric Associates | Overland Park | Kansas | 66211 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Michigan Clinical Research Institute PC | Ann Arbor | Michigan | 48105 | United States |
| Neurobehavioral Medicine Group | Bloomfield Hills | Michigan | 48302-1952 | United States |
| Helen DeVos Children's Hospital / Spectrum Health Medical Group | Wyoming | Michigan | 49418 | United States |
| St. Charles Psychiatric Associates dba Midwest Research Group | Saint Charles | Missouri | 63304 | United States |
| Movement Disorders Center | St Louis | Missouri | 63110-1093 | United States |
| Alivation Research, LLC | Lincoln | Nebraska | 68526 | United States |
| Center for Psychiatry and Behavioral Medicine Inc. | Las Vegas | Nevada | 89128 | United States |
| The NeuroCognitive Institute | Mount Arlington | New Jersey | 07856 | United States |
| Clinical Research Center of NJ | Voorhees Township | New Jersey | 08043-1910 | United States |
| New York Neurology Associates P.C | New York | New York | 10003 | United States |
| Hapworth Research Inc. | New York | New York | 10019 | United States |
| Mount Sinai School of Medicine | New York | New York | 10029-6504 | United States |
| Mood Disorders Consulting Medicine PLLC | New York | New York | 10036 | United States |
| Finger Lakes Clinical Research | Rochester | New York | 14618 | United States |
| Quest Therapeutics of Avon Lake | Avon Lake | Ohio | 44012-1004 | United States |
| Cincinnati Childrens Hospital Medical Center | Cincinnati | Ohio | 45229-3026 | United States |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| North Star Medical Research LLC | Middleburg Heights | Ohio | 44130 | United States |
| Suburban Research Associates | Media | Pennsylvania | 19063 | United States |
| Coastal Pediatric Research | Charleston | South Carolina | 29414 | United States |
| Access Clinical Trials, Inc. | Nashville | Tennessee | 37203-6502 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232-0028 | United States |
| Houston Clinical Trials LLC | Bellaire | Texas | 77401 | United States |
| Relaro Medical Trials | Dallas | Texas | 75243 | United States |
| North Texas Clinical Trials | Fort Worth | Texas | 76104 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Road Runner Research Ltd. | San Antonio | Texas | 78249-3539 | United States |
| Noetic Psychiatry | Springville | Utah | 84663 | United States |
| University of Virginia | Charlottesville | Virginia | 22908-0829 | United States |
| Eastside Therapeutic Resource Inc dba Core Clinical Research | Everett | Washington | 98201-4077 | United States |
| The Kids Clinic Inc | Ajax | Ontario | L1Z 0M1 | Canada |
| Center for Pediatric Excellence | Ottawa | Ontario | K2G 1W2 | Canada |
| CHU Sainte-Justine | Montreal | Quebec | H3T 1C5 | Canada |
| CHU Poitiers | Poitiers | 86021 | France |
| Hopital Robert Debre | Paris | Île-de-France Region | 75019 | France |
| Psychiatric Clinic of Ludwig Maximilians Universitaet Muenchen | Munich | Bavaria | 80336 | Germany |
| Department of Psychiatry, Socialpsychiatry and Psychotherapy, Hannover Medical School | Hanover | Lower Saxony | 30625 | Germany |
| Dept. Child Adolescent Psychiatry Uni. Freiburg | Freiburg im Breisgau | 79104 | Germany |
| Zentralinstitut fuer Seelische Gesundheit | Mannheim | 68159 | Germany |
| Pharmakologisches Studienzentrum Chemnitz GmbH | Mittweida | 09648 | Germany |
| Gdanskie Centrum Zdrowia Sp z o.o. | Gdansk | 80-542 | Poland |
| Centrum Bada Klinicznych PI-House Sp. z o.o. | Gdansk | 80-546 | Poland |
| Uniwersyteckie Centrum Kliniczne | Gdansk | 80-952 | Poland |
| NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis | Katowice | 40-123 | Poland |
| Centrum Medyczne Plejady | Krakow | 30-363 | Poland |
| Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie | Krakow | 31-503 | Poland |
| Med-Polonia Sp. z o. o. | Poznan | 60-693 | Poland |
Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks. |
| Safety Set | The Safety Set (SS) included all participants who received at least one dose of study drug. |
|
| The Modified Intention-to-Treat Set | The Modified Intention-to-Treat (mITT) set included all randomized participants who received at least one dose of study drug and had at least 1 post-baseline scoring of the YGTSS (Yale Global Tic Severity Scale). |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
The Safety Set included all participants who received at least one dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Ecopipam HCI 2 mg/kg/Day | Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks. |
| BG001 | Placebo | Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | 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 | Change From Baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at Week 12 | The YGTSS was a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0=none to 5=severe for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe) with higher score represent more severe symptoms. A negative change from baseline indicates improvement. | The mITT set included all randomized participants who received at least 1 dose of study drug and had at least 1 post-baseline scoring of the YGTSS. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 12 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) at Week 12 | Clinical Global Impression (CGI) scale was used to assess overall severity on a 7-point Likert scale consisted of 2 reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms. The CGI severity scale ranges from 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill subject. 1 = "normal, not ill at all" to 7 = "extremely ill." A negative change indicates improvement in the condition. | The mITT set included all randomized subjects who received at least 1 dose of study drug and had at least 1 post-baseline scoring of the YGTSS. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 12 |
|
Screening up to 30 days after the last dose ( up to Week 16)
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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 HCI 2 mg/kg/Day | Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks. | 0 | 76 | 2 | 76 | 47 | 76 |
| EG001 | Placebo | Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks. | 0 | 77 | 1 | 77 | 38 | 77 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Coronavirus infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Contusion | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Depressed mood | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Middle insomnia | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Tic | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Coronavirus infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (23.1) | Systematic Assessment |
|
PI must consult their clinical trial agreement. In summary, PI shall have the right to publish, present or otherwise use the results for their research publication objectives, provided that such Publication does not disclose Confidential Information. PI shall submit in writing to Sponsor any material at least 90 days for review and comment. Sponsor shall advise PI of any information which is Confidential Information or which may impair Sponsor's ability to obtain patent protection.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical contact | Emalex Biosciences, Inc. | 7150562 | 1847 | dkim@emalexbiosciences.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 23, 2021 | Aug 16, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D005879 | Tourette Syndrome |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| 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 |
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| ID | Term |
|---|---|
| C058081 | ecopipam |
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| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black or African American |
|
| Asian |
|
| American Indian or Alaska Native |
|
| Other |
|
| Units | Counts |
|---|---|
| Participants |
|
|