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

Tourette Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06021522
• Other study ID #: EBS-101-TD-391
• Secondary ID: 2023-503545-67-0
• Status: Recruiting
• Phase: Phase 3
• Start date: August 16, 2023
• Est. completion date: March 2027

Study information

• Verified date: June 2024
• Source: Emalex Biosciences Inc.
• Contact: Meredith M Miller
• Phone: 773 343 0671
• Email: mmiller[@]emalexbiosciences.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: March 2027
• Est. primary completion date: November 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Tourette Syndrome

Intervention

Drug:
• Ecopipam
Selective dopamine D1 and D5 receptor antagonist

Locations

Country	Name	City	State
Bulgaria	ASMP-IP- d-r Kayryakova	Sofia	Lyulin 1
Canada	The Kids Clinic Inc	Ajax	Ontario
Hungary	Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)	Budapest
Italy	Universita degli Studi di Napoli Federico II	Napoli
Poland	Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie	Krakow	Woj. Malopolskie
Spain	Hospital General Universitario Gregorio Maranon	Madrid
Spain	Hospital Universitario Virgen del Rocío	Sevilla
United States	Advanced Research Center	Anaheim	California
United States	Michigan Clinical research Institute PC	Ann Arbor	Michigan
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	CenExel CIT-IE	Bellflower	California
United States	Neurobahavioral Medicine Group	Bloomfield Hills	Michigan
United States	Boston Childrens Hospital	Boston	Massachusetts
United States	Mass General Hospital	Boston	Massachusetts
United States	Coastal Pediatric Research	Charleston	South Carolina
United States	OnSite Clinical Solutions LLC	Charlotte	North Carolina
United States	Lurie Children Hospital of Chicago	Chicago	Illinois
United States	Rush University Medical Center	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	National Childrens Hospital - The Ohio State University	Columbus	Ohio
United States	UT Southwestern	Dallas	Texas
United States	Harmonex, Inc.	Dothan	Alabama
United States	Cedar Clinical Research	Draper	Utah
United States	Core Clinical Research	Everett	Washington
United States	Cortica Site Network	Glendale	California
United States	NW FL Clinical Research Group, LLC	Gulf Breeze	Florida
United States	Research in Miami Inc	Hialeah	Florida
United States	The University of Texas Health Science Center at Houston	Houston	Texas
United States	Josephson-Wallack-Munshower Neurology	Indianapolis	Indiana
United States	Alivation Research	Lincoln	Nebraska
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	University of Louisville Research Foundation Inc.	Louisville	Kentucky
United States	Florida International Research Center	Miami	Florida
United States	University of Miami Miller School of Medicine	Miami	Florida
United States	North Star Medical Research, LLC	Middleburg Heights	Ohio
United States	Access Clinical Trials, Inc.	Nashville	Tennessee
United States	Access Clinical Trials, Inc.	Nashville	Tennessee
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Yale School of Medicine	New Haven	Connecticut
United States	Medical Research Group of Central Florida	Orange City	Florida
United States	APG Research, LLC	Orlando	Florida
United States	Providence Brain and Spine Institute	Portland	Oregon
United States	University of Rochester	Rochester	New York
United States	University of South Florida	Saint Petersburg	Florida
United States	Road Runner Research, Ltd	San Antonio	Texas
United States	Cortica Site Network - San Rafael	San Rafael	California
United States	Pediatric Epilepsy and Neurology Specialists	Tampa	Florida
United States	Childrens National Hospital	Washington	District of Columbia
United States	Umass Chan Medical School	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Emalex Biosciences Inc.

Countries where clinical trial is conducted

United States,  Bulgaria,  Canada,  Hungary,  Italy,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	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.	Baseline up to Month 24