Ecopipam Tablets to Study Tourette's Disorder in Children... | NCT05615220

ID	Type	Description	Link
2023-503494-38-00	EU Trial (CTIS) Number
2022-001961-11	EudraCT Number

Status Module

Description Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl)	Experimental	Ecopipam 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 mg tablets (containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively); 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) target dose; oral administration daily in evenings.	Drug: Ecopipam Hydrochloride
Placebo during R/WD Phase	Placebo Comparator	Matching Placebo tablets during R/WD period taken orally in the evening.	Drug: Ecopipam Hydrochloride

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Ecopipam Hydrochloride	Drug	Selective dopamine D1 and D5 receptor antagonist	1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) Placebo during R/WD Phase

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Time From Randomization to Relapse in Participants Greater Than and Equal to (>=) 6 and Less Than (<) 18 Years During the Double-Blind R/WD	Time to relapse defined as a loss of >=50 percent (%) of the improvement experienced on the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of Tourette's Disorder (TD), or requirement of hospitalization for worsening symptoms of TD in participants between the ages of >= 6 and <18 years for ecopipam compared to those receiving placebo during the double-blind, R/WD period. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in participants with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.	From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)

Secondary Outcomes

Measure	Description	Time Frame
Time From Randomization to Relapse in All Participants During the Double-Blind R/WD Period	Time From Randomization (Week 12) to relapse defined as a loss of >= 50% of the improvement experienced on the YGTSS-TTS from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of TD, or requirement of hospitalization for worsening symptoms of TD in all participants during the Double-Blind R/WD period for ecopipam compared to placebo. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in children and adults with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.

Eligibility Module

Facility	Status	City	State	ZIP	Country	Contacts
Harmonex Neuroscience Research		Dothan	Alabama	36303	United States
Arkansas Children's Hospital

PubMed Identifier	Type	Citation	Retractions
42189524	Derived	Gilbert DL, Atkinson SD, Kim DJB, Miller MM, Rice PM, Flatt JA, Karkanias GB, Munschauer FE, Bittman RM, Wanaski SP, Cunniff TM, Tomczak KK. Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial. JAMA Neurol. 2026 May 26:e261431. doi: 10.1001/jamaneurol.2026.1431. Online ahead of print.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Open-label Stabilization Period: Ecopipam 1.8 mg/kg/Day	All participants in the open-label stabilization period received a target steady-state dose of 1.8 milligram per kilogram per day (mg/kg/day) ecopipam (2 mg/kg/day ecopipam hydrochloride [HCl]) tablets based on body weight, administered orally, once daily in the evening during a 4-week titration phase and further followed by an 8-week maintenance phase.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Mar 5, 2024	Nov 12, 2025

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

Time From Randomization to Relapse in Participants Greater Than and Equal to (>=) 6 and Less Than (<) 18 Years During the Double-Blind R/WD

Time to relapse defined as a loss of >=50 percent (%) of the improvement experienced on the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of Tourette's Disorder (TD), or requirement of hospitalization for worsening symptoms of TD in participants between the ages of >= 6 and <18 years for ecopipam compared to those receiving placebo during the double-blind, R/WD period. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in participants with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.

The modified intention-to-treat (mITT) set included all randomized participants who received at least 1 dose of study drug post-randomization.

Posted

Median

95% Confidence Interval

weeks

From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)

ID	Title	Description
OG000	Double- Blind R/WD Period: Ecopipam 1.8 mg/kg/Day	Participants who met stabilization criteria were randomized to receive ecopipam 1.8 mg/kg/day (ecopipam HCl 2 mg/kg/day) orally once daily in the evening, up to 12 weeks in Double-Blind R/WD Phase.
OG001	Double- Blind R/WD Period: Placebo	Participants received matching placebo tablets, once daily, up to 12 weeks in Double-blind R/WD Phase. Participants randomized to placebo were tapered off ecopipam in a blinded fashion, in decrements of 22.4 milligrams per day (mg/day) (25 mg/day ecopipam HCl).

Units	Counts
Participants	OG00043 OG00147

Title

Denominators

Categories

Title	Measurements
	OG000NA(4.1 to NA)Here "NA" means median and upper limit of 95% confidence interval could not be estimated due to insufficient events within the treatment group. OG0014.0(2.4 to 6.1)

Secondary

Time From Randomization to Relapse in All Participants During the Double-Blind R/WD Period

Time From Randomization (Week 12) to relapse defined as a loss of >= 50% of the improvement experienced on the YGTSS-TTS from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of TD, or requirement of hospitalization for worsening symptoms of TD in all participants during the Double-Blind R/WD period for ecopipam compared to placebo. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in children and adults with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.

The mITT Set included all randomized participants who received at least 1 dose of study drug post-randomization.

Posted

Median

95% Confidence Interval

weeks

From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)

ID	Title	Description
OG000	Double- Blind R/WD Period: Ecopipam 1.8 mg/kg/Day	Participants who met stabilization criteria were randomized to receive ecopipam 1.8 mg/kg/day (ecopipam HCl 2 mg/kg/day) orally OD in the evening, up to 12 weeks in Double-Blind R/WD Phase.
OG001	Double- Blind R/WD Period: Placebo

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Open-label Stabilization Period: Ecopipam 1.8 mg/kg/Day	All participants in the open-label stabilization period were received a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam hydrochloride [HCl]) tablets based on body weight, administered orally, once daily in the evening during a 4-week titration phase and further followed by an 8-week maintenance phase.	216	1	216	140	216
EG001	Double-blind R/WD Period: Ecopipam 1.8 mg/kg/Day	Participants who met stabilization criteria were randomized to receive ecopipam 1.8 mg/kg/day (ecopipam HCl 2 mg/kg/day) orally once daily in the evening, up to 12 weeks in Double-blind R/WD Phase.	51	1	51	20	51
EG002	Double- Blind R/WD Period: Placebo	Participants received matching placebo tablets, once daily, up to 12 weeks in Double-blind R/WD Phase. Participants randomized to placebo were tapered off ecopipam in a blinded fashion, in decrements of 22.4 mg/day (25 mg/day ecopipam HCl).	53	2	53	22	53

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Tourette's disorder	Congenital, familial and genetic disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0010 affected51 at risk EG0021 affected53 at risk
Blood creatine phosphokinase increased	Investigations	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0010 affected51 at risk EG0020 affected53 at risk
Type 1 diabetes mellitus	Metabolism and nutrition disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Obsessive-compulsive disorder	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0010 affected51 at risk EG0020 affected53 at risk
Suicidal ideation	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0010 affected51 at risk EG0021 affected53 at risk
Acute kidney injury	Renal and urinary disorders	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0010 affected51 at risk EG0020 affected53 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Atrioventricular block first degree	Cardiac disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Cryptorchism	Congenital, familial and genetic disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Nausea	Gastrointestinal disorders	MedDRA (27.0)	Systematic Assessment	EG0006 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA (27.0)	Systematic Assessment	EG0006 affected216 at risk EG0010 affected51 at risk EG0020 affected53 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA (27.0)	Systematic Assessment	EG0005 affected216 at risk EG0011 affected51 at risk EG0021 affected53 at risk
Vomiting	Gastrointestinal disorders	MedDRA (27.0)	Systematic Assessment	EG0006 affected216 at risk EG0011 affected51 at risk EG0021 affected53 at risk
Abdominal discomfort	Gastrointestinal disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Abdominal pain lower	Gastrointestinal disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Fatigue	General disorders	MedDRA (27.0)	Systematic Assessment	EG00014 affected216 at risk EG0010 affected51 at risk EG0020 affected53 at risk
Pyrexia	General disorders	MedDRA (27.0)	Systematic Assessment	EG0003 affected216 at risk EG0011 affected51 at risk EG0021 affected53 at risk
Influenza like illness	General disorders	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0012 affected51 at risk EG0020 affected53 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0008 affected216 at risk EG0013 affected51 at risk EG0020 affected53 at risk
Nasopharyngitis	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0005 affected216 at risk EG0010 affected51 at risk EG0022 affected53 at risk
Bronchitis	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0004 affected216 at risk EG0010 affected51 at risk EG0022 affected53 at risk
Gastroenteritis viral	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0003 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Influenza	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0002 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Viral infection	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0002 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Ear infection	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Gastroenteritis	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0011 affected51 at risk EG0022 affected53 at risk
Pharyngitis streptococcal	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0012 affected51 at risk EG0021 affected53 at risk
Acute sinusitis	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Urinary tract infection	Infections and infestations	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Ligament sprain	Injury, poisoning and procedural complications	MedDRA (27.0)	Systematic Assessment	EG0002 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Blood glucose increased	Investigations	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Blood potassium increased	Investigations	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA (27.0)	Systematic Assessment	EG0004 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Type 1 diabetes mellitus	Metabolism and nutrition disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Somnolence	Nervous system disorders	MedDRA (27.0)	Systematic Assessment	EG00024 affected216 at risk EG0010 affected51 at risk EG0020 affected53 at risk
Headache	Nervous system disorders	MedDRA (27.0)	Systematic Assessment	EG00019 affected216 at risk EG0012 affected51 at risk EG0023 affected53 at risk
Dizziness	Nervous system disorders	MedDRA (27.0)	Systematic Assessment	EG0002 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Migraine	Nervous system disorders	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Disturbance in attention	Nervous system disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Paraesthesia	Nervous system disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Anxiety	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG00020 affected216 at risk EG0011 affected51 at risk EG0021 affected53 at risk
Insomnia	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG00016 affected216 at risk EG0013 affected51 at risk EG0025 affected53 at risk
Tic	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG00015 affected216 at risk EG0012 affected51 at risk EG0021 affected53 at risk
Depressed mood	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0008 affected216 at risk EG0010 affected51 at risk EG0021 affected53 at risk
Irritability	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0006 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Restlessness	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0005 affected216 at risk EG0010 affected51 at risk EG0020 affected53 at risk
Suicidal ideation	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0005 affected216 at risk EG0010 affected51 at risk EG0021 affected53 at risk
Agitation	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0002 affected216 at risk EG0012 affected51 at risk EG0020 affected53 at risk
Initial insomnia	Psychiatric disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0023 affected53 at risk
Menstrual disorder	Reproductive system and breast disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA (27.0)	Systematic Assessment	EG0003 affected216 at risk EG0012 affected51 at risk EG0020 affected53 at risk
Asthma	Respiratory, thoracic and mediastinal disorders	MedDRA (27.0)	Systematic Assessment	EG0002 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Respiratory distress	Respiratory, thoracic and mediastinal disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Rhinitis allergic	Respiratory, thoracic and mediastinal disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA (27.0)	Systematic Assessment	EG0001 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Ecchymosis	Skin and subcutaneous tissue disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA (27.0)	Systematic Assessment	EG0000 affected216 at risk EG0011 affected51 at risk EG0020 affected53 at risk

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