Long-Term Safety Evaluation of Ecopipam Hydrochloride Tablets in Pediatric, Adolescent, and Adult Patients with Tourette's Disorder

The primary objective of this study is to evaluate the long-term **safety** and tolerability of ecopipam tablets in children (≥ 6 and < 12 years of age), adolescents (≥ 12 and < 18 years of age), and adults (≥ 18 years of age) with **Tourette's Disorder** who are eligible following their participation in studies EBS-101-TD-301, EBS-101-OL-001, or PSY-302A. This is clinically relevant as it aims to ensure that the treatment is safe for long-term use across different age groups, which is crucial for managing a chronic condition like Tourette's Disorder.

The secondary objective of this study is to assess the durability of the treatment effect in the same patient population described above. This evaluation is important to determine the sustained efficacy of ecopipam over an extended period, which can inform treatment plans and improve patient outcomes.

The clinical trial involves a total of **103 participants** diagnosed with **Tourette's Disorder**. The study population includes children aged 6 to 11, adolescents aged 12 to 17, and adults aged 18 and above. Both male and female subjects are included, and the trial specifically considers vulnerable populations. Participants were selected based on their prior involvement in studies EBS-101-TD-301, EBS-101-OL-001, or PSY-302A, and their eligibility was determined by their completion of these studies and the clinical benefit observed from ecopipam treatment. The trial does not specify particular lifestyle considerations such as diet or physical activity. The selection criteria ensure that participants exhibit both motor and vocal tics that interfere with normal routines, as per the Diagnostic and Statistical Manual for Mental Disorders, 5th Edition (DSM-5-TR) criteria for Tourette's Disorder.

The clinical trial is designed to evaluate the long-term safety and tolerability of **ecopipam hydrochloride** tablets in individuals with **Tourette's Disorder**. This study is an open-label trial, meaning that both the researchers and participants are aware of the treatment being administered. The trial will involve children, adolescents, and adults who have previously participated in specific studies and have shown clinical benefit from the medication. The trial is expected to commence recruitment on March 4, 2024, and conclude by April 13, 2027, with a maximum treatment period of 105 days for each participant.

Participants will undergo a series of study visits, beginning with an inclusion (screening) visit to confirm eligibility based on criteria such as previous study participation and clinical benefit from **ecopipam hydrochloride**. Following the screening, participants will receive the medication in tablet form, administered orally. The study does not have specific primary or secondary endpoints due to its open-label nature, focusing instead on the safety and tolerability of the treatment over the long term.

Throughout the trial, participants will attend regular follow-up visits to monitor their response to the medication and any potential side effects. These visits are crucial for ensuring participant safety and collecting data on the medication's long-term effects. The trial will conclude with an end-of-study visit, where final assessments will be conducted to evaluate the overall impact of the treatment.

Participant involvement is expected to last up to 105 days, with conditions for early termination including withdrawal of consent, adverse reactions, or any other reason deemed necessary by the investigator. The trial aims to provide valuable insights into the long-term use of **ecopipam hydrochloride** in managing **Tourette's Disorder**, contributing to the understanding of its safety profile in a diverse patient population.

The clinical trial involves the administration of **Ecopipam hydrochloride** in various dosages to evaluate its long-term safety and tolerability in individuals with Tourette's Disorder. The experimental medication is provided in the form of tablets, with each tablet containing a specific dosage of Ecopipam hydrochloride. The available dosages for the trial include 11.2 mg, 22.4 mg, 33.6 mg, 44.8 mg, 67.2 mg, and 89.6 mg. The tablets are administered orally, and the maximum daily dose varies depending on the specific dosage form, ranging from 33.6 mg to 179.2 mg. The treatment period for each participant is up to 105 days.

Each dosage form of Ecopipam hydrochloride is manufactured by Emalex Biosciences, Inc. and is classified as a chemical substance. The tablets are not formulated specifically for pediatric use, and the trial does not involve any orphan drug designation. The administration of the medication is consistent across all dosage forms, with oral use being the sole route of administration. The trial does not include any non-experimental treatments such as standard-of-care therapy, placebo, or comparator treatment. Participant compliance with the dosing schedule is monitored throughout the study to ensure adherence to the prescribed regimen.

The clinical trial aims to evaluate the long-term safety and tolerability of **ecopipam hydrochloride** tablets in children, adolescents, and adults with Tourette's Disorder. As this is an open-label study, there are no specific efficacy endpoints defined. The primary focus is on safety assessments rather than efficacy measurements. Participants eligible for this study are those who have previously participated in studies EBS-101-TD-301, EBS-101-OL-001, or PSY-302A and have shown clinical benefit from ecopipam. The trial will monitor participants over a maximum treatment period of 105 days, with the primary objective being the assessment of safety and tolerability of the medication in the specified population.