A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder

Emalex Biosciences

Status and phase: Enrolling; Phase 3

Conditions: Tourette Syndrome

Treatments: Drug: Ecopipam

Study type: Interventional

Funder types: Industry

Identifiers

NCT06021522

EBS-101-TD-391

2023-503545-67-00 (Other Identifier)

Details and patient eligibility

About

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to [>=] 6 and less than [<] 12 years of age), adolescents (>=12 and <18 years of age), and adults (>=18 years of age) with Tourette's Syndrome (TS).

Full description

This study is to evaluate the long-term safety and tolerability of ecopipam tablets in eligible participants. The eligible participants will be entered into a treatment period and start a 4-week titration phase to achieve a target steady-state dose of 1.8 milligram per kilogram per day (mg/kg/day) ecopipam (2 mg/kg/day dose of ecopipam HCl). During the 4-week titration phase ecopipam will be dispensed following weight bands before reaching their respective maintenance dose until end of the treatment. Safety assessment will be conducted at baseline visit and at all treatment visits (Months 1-12, 15, 18, 21 and 24). Safety follow up visits will be conducted 7 and 14 days and a follow up phone call will be conducted 30 days after the last dose of ecopipam.

Enrollment: 150 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• >=6 to >=18 years of age.
• Participants enrolling from the study EBS-101-TD-301; completed all visits through Week 24 and days 7 and 14 safety follow-up, met relapse criteria during the double-blind randomized (R/WD) period after completing the 301 end of trial (ET) visit, the 7 day and 14 day safety follow up visits, but not before 24 weeks following the 301 baseline visit or participants who met relapse criteria will be eligible after completing early termination visit, Day 7 and Day 14 follow up visits.
• Participants who completed the studies EBS-101-OL-001 or PSY302A.
• The enrolling participant must have had clinical benefit from ecopipam and would benefit from continued participation.
• Effective contraception during the study and 30 days after last study dose for sexually active participants
• <18 years of age participants parent/legal guardian must sign a written informed consent and participant must sign a written informed assent.
• Participant must have TD based on Diagnostic and Statistical Manual for Mental Disorders - 5th Edition (DSM-5-TR diagnostic criteria) for TD.
• TD diagnosis and both motor and vocal tics that cause impairment with normal routines

Exclusion criteria

• The participants who discontinued the studies PSY-302A, EBS-101-OL-001 or EBS-101-TD-301 due to reasons such as either lost to follow up, withdrawn consent, non-compliant or withdrawn by the discretion of either the site investigator or the sponsor.
• Participants with ongoing or past history of neurological condition (example [e.g.], Huntington's disease, Parkinson's disease, Wilson's disease, stroke, Restless Legs Syndrome).
• Any unstable mood disorder (DSM-5-TR criteria), mental illness or clinically significant lab abnormalities, moderate to severe renal or hepatic impairment, a PHQ-9 score >=10 at screening and history of neuroleptic malignant syndrome at the time of screening or baseline.
• Participants who completed the studies EBS-101-OL-001 or PSY-302A and who had previous exposure to ecopipam and oral neuroleptics within 4 weeks and depot neuroleptics within 3 months prior to screening, 6 months prior to Baseline.
• Participants receiving any other medication to treat motor or vocal tics and anti-depressant or anti-anxiety medications.
• Risk of suicide as per PI judgement
• Pregnant or lactating women
• Certain medications that would have unfavorable drug interactions with ecopipam, e.g., digoxin, fluoxetine, valproic acid, bupropion.
• Current or recent (past 3 months) DSM-5-TR substance use disorder (with the exception of nicotine).
• Recent behavioral therapy
• Positive urine drug screen for cocaine, amphetamine, benzodiazepines, barbiturates, phencyclidine (PCP) or opiates at Baseline, except those receiving stable, prescribed treatment for attention deficit/hyperactivity disorder (ADHD)
• Lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any other psychotic disorder.
• Unable to swallow tablets.
• Known hypersensitivity to any of ecopipam's excipients.
• History of seizures (excluding febrile seizures that occurred >2 years prior to Baseline).
• Myocardial infarction within 6 months from Screening.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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150 participants in 1 patient group

Ecopipam 1.8 mg/kg/day

Experimental group

Description:

Ecopipam tablets will be administered orally (PO) once daily in the evening without regard to meals at concentrations 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 milligrams (mg) containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively in 4-week titration phase to achieve a target dose of 1.8 milligram per kilogram per day (mg/kg/day) ecopipam (2 mg/kg/day ecopipam HCl). Participants will be evaluated for safety at each baseline visit and at all treatment visits up to 24 months and at follow up visits at 7 and 14 days after last dose of ecopipam.

Treatment:

Drug: Ecopipam