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

Emalex Biosciences Inc.216 enrolled

Overview

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 a double-blind, placebo-controlled, randomized withdrawal period.

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 During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

216

Conditions

Tourette Disorder

Interventions

Ecopipam HydrochlorideDRUG

Selective dopamine D1 and D5 receptor antagonist

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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 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

Locations (95)

Outcomes

Primary Outcomes

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.

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

Secondary Outcomes

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.

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

Data from ClinicalTrials.gov

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