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

Purpose

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.

Condition

  • Tourette Disorder

Eligibility

Eligible Ages
Over 6 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Intervention Model Description
This is a multicenter study which includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)
Masking Description
This includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) 		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
    Selective dopamine D1 and D5 receptor antagonist
Placebo Comparator
Placebo during R/WD Phase 		Matching Placebo tablets during R/WD period taken orally in the evening.
  • Drug: Ecopipam Hydrochloride
    Selective dopamine D1 and D5 receptor antagonist

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114
Contact:
Daniel Geller, MD
617-724-5141
dan.geller@mgh.harvard.edu

More Details

Status
Recruiting
Sponsor
Emalex Biosciences Inc.

Study Contact

Meredith M Miller
773 343 0671
mmiller@emalexbiosciences.com

Detailed Description

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.