D1AMOND: Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults

Sponsor

Emalex Biosciences Inc. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05615220

Collaborator

(none)

196

Enrollment

Locations

Arms

Anticipated Duration (Months)

65.3

Patients Per Site

3.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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 or Disease	Intervention/Treatment	Phase
Tourette Disorder	Drug: Ecopipam Hydrochloride	Phase 3

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 During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

196 participants

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.This is a multicenter study which includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.

Masking:

Triple (Participant, Care Provider, Investigator)

Masking Description:

This includes an open-label period followed by double-blind, placebo-controlled, randomized withdrawal period.

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Feb 1, 2023

Anticipated Primary Completion Date :

Nov 1, 2024

Anticipated Study Completion Date :

Nov 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
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

Arm

Intervention/Treatment

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

Outcome Measures

Primary Outcome Measures

Yale Global Tic Severity Scale (YGTSS) [Screening up to Week 24]
Clinician-completed rating scale with score ranging from 0 to 50 (0=none to 50=severe)

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	APG Research LLC	Orlando	Florida	United States	32803
2	Access Clinical Trials, Inc.	Nashville	Tennessee	United States	37203-6502
3	Baylor College of Medicine	Houston	Texas	United States	77030

Sponsors and Collaborators

Emalex Biosciences Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Emalex Biosciences Inc.

ClinicalTrials.gov Identifier:

NCT05615220

Other Study ID Numbers:

EBS-101-TD-301

First Posted:

Nov 14, 2022

Last Update Posted:

Jan 18, 2023

Last Verified:

Nov 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Tourette Syndrome

Ecopipam

Study Results

No Results Posted as of Jan 18, 2023