Clinical trial NCT04114539: Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension
Official title: A Multicenter, Open-Label, Extension Study Intended to Evaluate the Long-term Safety of Ecopipam Tablets in Children and Adolescent Subjects With Tourette's Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Tourette Syndrome in Children
Tourette Syndrome in Adolescence
Keywords: Tourette Syndrome
Lead sponsor: Emalex Biosciences Inc.
Sponsor class: INDUSTRY
First posted: 2019-10-03
Enrollment: 124
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open Label
Primary purpose: TREATMENT

Brief summary: This study was an international, multicenter, open-label, long term extension study evaluating the safety of ecopipam tablets for the treatment of children and adolescent subjects with Tourette Syndrome.

Detailed description: This was an international, multicenter, open-label, long term extension study to evaluate the safety of ecopipam tablets for the treatment of pediatric subjects (aged ≥6 to ≤18 years at Baseline) with Tourette Syndrome. Participants who completed the Phase 2b, randomized, double-blind, efficacy and safety study (EBS-101-CL-001) without major reportable protocol deviations, and who met all the inclusion/exclusion criteria for this study were eligible to participate in this study. All participants were titrated to a target dose of 2 mg/kg/day as participants rolled over from the Phase 2b double-blind efficacy and safety study were tapered off study drug to maintain the blind from that study. Participants were to complete study visits every month for 1 year. Follow-up visits were conducted 7 and 14 days after the last dose of the study drug and a follow-up phone call was conducted 30 days after the last dose of study drug

Eligibility criteria: Inclusion Criteria:

* Subjects must have completed the EBS-101-CL-001 study through the Day 14 Follow Up Visit within the last 30 days (or longer with permission of the medical monitor) without a major reportable protocol deviation and must be someone the Investigator feels would benefit from continued participation.

Exclusion Criteria:

* Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder).
* Unstable medical illness or clinically significant lab abnormalities.
* Risk of suicide.
* Pregnant or lactating women.
* Moderate to severe renal insufficiency.
* Positive urine drug screen.
* Certain medications that would lead to drug interactions.

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related)
timeFrame: From start of study drug administration until 30 days after last dose (Up to Month 13)
measure: Change From Baseline in Hematology Parameters: Basophils to Leukocytes Ratio Reported in Percentage of Cells
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Eosinophils to Leukocytes Ratio Reported in Percentage of Cells
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Erythrocytes
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Hematocrit
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Hemoglobin
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Leukocytes and Platelets
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Hemoglobin A1c (HbA1c)
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Vital Signs Parameter: Pulse Rate
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI]
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Vital Signs Parameter: Height
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Vital Signs Parameter: Weight
timeFrame: Baseline up to Month 12
measure: Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval
timeFrame: Baseline to Month 12
measure: Number of Participants With Clinically Significant Abnormal Physical Examination Findings
timeFrame: Baseline up to Month 12
Secondary outcomes:
measure: Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12
timeFrame: Baseline up to Months 1, 3, 6, 9 and 12
measure: Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12
timeFrame: Baseline up to Months 1, 3, 6, 9 and 12
measure: Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12
timeFrame: Baseline up to Months 1, 3, 6, 9 and 12
measure: Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12
timeFrame: Baseline up to Months 1, 3, 6, 9, 12
measure: Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12
timeFrame: Months 1, 3, 6, 9 and 12
measure: Change From Baseline in Gilles de la Tourette Syndrome-Quality of Life Scale for Children and Adolescents (C&A-GTS-QOL) Total Score at Months 1, 3, 6, 9 and 12
timeFrame: Baseline up to Months 1, 3, 6, 9 and 12
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Harmonex Neuroscience Research
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Anaheim
country: United States
facility: Advanced Research Center Inc.
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: San Diego
country: United States
facility: PCSD-Feighner Research
state: California
city: Santa Ana
country: United States
facility: Syrentis Clinical Research
state: California
city: New Haven
country: United States
facility: Yale School of Medicine
state: Connecticut
city: Gainesville
country: United States
facility: Sarkis Clinical Trials
state: Florida
city: Gulf Breeze
country: United States
facility: Northwest Florida Clinical Research Group, LLC
state: Florida
city: Hialeah
country: United States
facility: Research in Miami Inc.
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: North Miami
country: United States
facility: MedBio Trials
state: Florida
city: Orlando
country: United States
facility: APG Research LLC
state: Florida
city: Saint Petersburg
country: United States
facility: University of South Florida
state: Florida
city: Tampa
country: United States
facility: Pediatric Epilepsy and Neurology Specialists
state: Florida
city: Winter Park
country: United States
facility: Pediatric Neurology, PA
state: Florida
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Savannah
country: United States
facility: Meridian Clinical Research
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Chicago
country: United States
facility: The University of Chicago Hospitals
state: Illinois
city: Naperville
country: United States
facility: AMR - Baber Research Inc.
state: Illinois
city: Overland Park
country: United States
facility: Psychiatric Associates
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: Michigan Clinical Research Institute PC
state: Michigan
city: Bloomfield Hills
country: United States
facility: Neurobehavioral Medicine Group
state: Michigan
city: Wyoming
country: United States
facility: Helen DeVos Children's Hospital / Spectrum Health Medical Group
state: Michigan
city: Saint Charles
country: United States
facility: St. Charles Psychiatric Associates dba Midwest Research Group
state: Missouri
city: Saint Louis
country: United States
facility: Movement Disorders Center
state: Missouri
city: Lincoln
country: United States
facility: Alivation Research, LLC
state: Nebraska
city: Las Vegas
country: United States
facility: Center for Psychiatry and Behavioral Medicine Inc.
state: Nevada
city: Mount Arlington
country: United States
facility: The NeuroCognitive Institute
state: New Jersey
city: Voorhees
country: United States
facility: Clinical Research Center of NJ
state: New Jersey
city: New York
country: United States
facility: New York Neurology Associates P.C
state: New York
city: New York
country: United States
facility: Hapworth Research Inc.
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: New York
country: United States
facility: Mood Disorders Consulting Medicine PLLC
state: New York
city: Rochester
country: United States
facility: Finger Lakes Clinical Research
state: New York
city: Avon Lake
country: United States
facility: Quest Therapeutics of Avon Lake
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Childrens Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Middleburg Heights
country: United States
facility: North Star Medical Research LLC
state: Ohio
city: Media
country: United States
facility: Suburban Research Associates
state: Pennsylvania
city: Charleston
country: United States
facility: Coastal Pediatric Research
state: South Carolina
city: Nashville
country: United States
facility: Access Clinical Trials, Inc.
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Bellaire
country: United States
facility: Houston Clinical Trials LLC
state: Texas
city: Dallas
country: United States
facility: Relaro Medical Trials
state: Texas
city: Fort Worth
country: United States
facility: North Texas Clinical Trials
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: San Antonio
country: United States
facility: Road Runner Research Ltd.
state: Texas
city: Springville
country: United States
facility: Noetic Psychiatry
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Everett
country: United States
facility: Eastside Therapeutic Resource Inc dba Core Clinical Research
state: Washington
city: Ajax
country: Canada
facility: The Kids Clinic Inc
state: Ontario
city: Ottawa
country: Canada
facility: Center for Pediatric Excellence
state: Ontario
city: Montreal
country: Canada
facility: CHU Sainte-Justine
state: Quebec
city: Poitiers
country: France
facility: CHU Poitiers
city: Mittweida
country: Germany
facility: Pharmakologisches Studienzentrum Chemnitz GmbH
city: Gdansk
country: Poland
facility: Centrum Bada Klinicznych PI-House Sp. z o.o.
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Gdańsk
country: Poland
facility: Gdanskie Centrum Zdrowia Sp z o.o.
city: Katowice
country: Poland
facility: NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis
city: Krakow
country: Poland
facility: Centrum Medyczne Plejady
city: Krakow
country: Poland
facility: Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
city: Poznań
country: Poland
facility: Med-Polonia Sp. z o. o.
Study officials:
References: