Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults

Study Overview
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Brief Summary
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This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD). The study includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.

Detailed Description
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Following a 28-day Screening period and Baseline visit, eligible subjects will be enrolled into the open-label Stabilization period comprised of a 4-week Titration phase to achieve a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) followed by an 8-week open-label Maintenance phase. Responders to ecopipam will be randomized to ecopipam 1.8 mg/kg/day (2 mg/kg/day ecopipam HCl) or placebo in a 1:1 fashion and enter the double-blind Randomized-Withdrawal (R/WD) period at Week 12. During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.

Official Title
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A Multicenter, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Maintenance of Efficacy of Ecopipam in Children, Adolescents and Adults With Tourette's Disorder

Conditions
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Tourette Disorder

Intervention / Treatment
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* Drug: Ecopipam Hydrochloride


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: ≥ 6 years of age ≥ 18 kg (  40 lbs.) TD diagnosis and both motor and vocal tics that cause impairment with normal routines Minimum score of 20 on the YGTSS-R Total Tic Score May not be taking any medications used to treat motor or vocal tics for at least 14 days prior to Baseline. Effective contraception during the study and 30 days after last study dose for sexually active subjects Exclusion Criteria: Previous exposure to ecopipam Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder) Unstable unstable medical illness or clinically significant lab abnormalities Risk of suicide Pregnant or lactating women Moderate to severe renal insufficiency Hepatic insufficiency Positive urine drug screen Unstable doses for drugs to treat anxiety, depression, Attention Deficit Hyperactivity Disorder Certain medications that would lead to drug interactions Recent behavioral therapy

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Interventional Model Description: This is a multicenter study which includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl)<br>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<br>* Selective dopamine D1 and D5 receptor antagonist<br>|
| Placebo Comparator: Placebo during R/WD Phase<br>Matching Placebo tablets during R/WD period taken orally in the evening. | Drug: Ecopipam Hydrochloride<br>* Selective dopamine D1 and D5 receptor antagonist<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Yale Global Tic Severity Scale (YGTSS) | Clinician-completed rating scale with score ranging from 0 to 50 (0=none to 50=severe) | Screening up to Week 24 |