A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder
- Registration Number
- NCT06021522
- Lead Sponsor
- Emalex Biosciences Inc.
- Brief Summary
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).
- Detailed 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
- >=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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Ecopipam 1.8 mg/kg/day Ecopipam 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.
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Baseline up to Month 24 An adverse event (AE) is defined as any untoward medical occurrence in a subject administered a study drug and which does not necessarily have a causal relationship with this treatment. A TEAE is the development of an undesirable medical condition or the deterioration of a pre-existing medical condition following or during exposure to a pharmaceutical product, whether or not considered casually related to the product.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (66)
Harmonex, Inc.
🇺🇸Dothan, Alabama, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Advanced Research Center
🇺🇸Anaheim, California, United States
CenExel CIT-IE
🇺🇸Bellflower, California, United States
Cortica Site Network
🇺🇸Glendale, California, United States
Cortica Site Network - San Rafael
🇺🇸San Rafael, California, United States
Yale School of Medicine
🇺🇸New Haven, Connecticut, United States
Childrens National Hospital
🇺🇸Washington, District of Columbia, United States
NW FL Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, United States
Research in Miami Inc
🇺🇸Hialeah, Florida, United States
Scroll for more (56 remaining)Harmonex, Inc.🇺🇸Dothan, Alabama, United StatesNelson Handal-Thome, MDContact334-836-2000nhandal@harmonex.us