Expanded Access Program for Treatment With Ecopipam for Tourette's Disorder

• Conditions: Tourettes Disorder; Tourette Disorder

• Registration Number: NCT07093541
• Lead Sponsor: Emalex Biosciences Inc.

Brief Summary

The goal of this expanded access study is to learn about the long-term effects of Ecopipam in participants with Tourette's Disorder (TD). The main question it aims to answer is:

* Is ecopipam safe and well-tolerated in participants equal and over the age of 6 to 50 with TD?

* Assess quality of life measures and to provide access to ecopipam while NDA is being prepared and submitted for review for marketing approval

Eligible participants with TD will take ecopipam with guidance from their treating physician. In addition to the standard of care from the treating physician, treatment with ecopipam will continue until ecopipam becomes commercially available in the United States or the program is terminated.

While in the EAP program, participants will be asked regarding how safe and tolerable ecopipam is to the participant. Several questionnaires will be collected regarding participants experiences while taking ecopipam during monthly visits for the first year and every three months thereafter until the conclusion of the study.

Detailed Description

The main purpose of this study is to further study the safety and tolerability of ecopipam in children, adolescents, and adults with Tourette's Disorder (TD).

The secondary objectives of this program are to assess patient-reported outcome measures including quality of life (QOL) and to provide access to ecopipam while a U.S. New Drug Application (NDA) is being prepared and submitted for review for marketing approval in the U.S.

This is an open label expanded access program for ecopipam (EBS-101) intended to collect safety information and physician-administered psychiatric scales and patient-reported outcomes in children, adolescents, and adults in the U.S. with TD who are unable to take currently approved medications for TD (aripiprazole, haloperidol, or pimozide) or any other D2 receptor antagonists (D2rA) due to treatment failure, intolerability, safety or lack of access.

At the Screening visit, participants will provide informed consent \[adult participants or children/adolescent Legally Acceptable Representative (LAR)\] and assent (children/adolescents) and be assessed against the inclusion/exclusion criteria for participation in the study. Demographic information, disease comorbidities, and concomitant medications will be documented, vital signs and a physical examination will be performed, blood and urine samples will be collected for safety laboratories, an electrocardiogram (ECG) will be performed, lifetime history for suicidality will be assessed using the Columbia Suicidality Severity Rating Scale (C-SSRS), and age group validated scales to assess anxiety and depression will be performed. The participant and in the case of pediatric participants their LAR will also be asked to complete scales to assess the severity of their disease and quality of life.

Based on satisfying Screening visit criteria, participants will receive a telephone call to confirm Enrollment (Day 1). Visit dates will be defined from date of first dose. Once Enrollment is confirmed, eligible participants will be registered with a specialty pharmacy who will dispense ecopipam. Participants will be gradually titrated over a period of 2-4 weeks depending on their weight to achieve a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl).

Following the Enrollment call, there will be a telemedicine assessment in approximately 2 weeks to assess adverse events (AEs) and participant tolerance to ecopipam. Participants will return to the clinic at monthly intervals for safety and efficacy assessments for the first year of treatment and every 3 months thereafter until study termination as outlined in the Schedule of Assessments (Table 5).

Participants will also be contacted 30 days after the last dose of study drug to determine any additional AEs or concomitant medications, to administer the C-SSRS and to confirm compliance with taper down medication and study drug return.

Study Timeline

• Study First Submitted: 2025-07-10
• Study First Submitted QC: 2025-07-21
• Study First Posted: 2025-07-30
• Status Verified: 2025-10
• Last Update Submitted: 2025-10-08
• Last Update Posted: 2025-10-09

Recruitment & Eligibility

• Status: TEMPORARILY_NOT_AVAILABLE
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified