Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension
- Conditions
- Tourette Syndrome in ChildrenTourette Syndrome in Adolescence
- Interventions
- Registration Number
- NCT04114539
- Lead Sponsor
- Emalex Biosciences Inc.
- 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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 124
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Ecopipam Ecopipam -
- Primary Outcome Measures
Name Time Method Change From Baseline in Hematology Parameters: Hemoglobin Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameter hemoglobin was reported.
Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (bicarbonate, calcium, chloride, cholesterol, glucose, phosphate, potassium, sodium, triglyceride, urea nitrogen) expressed in millimoles per liter (mmol/L) were reported.
Change From Baseline in Hematology Parameters: Basophils to Leukocytes Ratio Reported in Percentage of Cells Baseline up to Month 12 Basophils/Leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in basophils to leukocytes ratio is reported in terms of percentage of cells.
Change From Baseline in Hematology Parameters: Eosinophils to Leukocytes Ratio Reported in Percentage of Cells Baseline up to Month 12 Eosinophils/Leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in eosinophils to leukocytes ratio is reported in terms of percentage of cells.
Change From Baseline in Hematology Parameters: Erythrocytes Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameter erythrocytes was reported.
Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (albumin, globulin and protein) were reported.
Change From Baseline in Vital Signs Parameter: Pulse Rate Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital sign parameter pulse rate and according to assessment position (supine and standing) was reported.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) From start of study drug administration until 30 days after last dose (Up to Month 13) An AE was any untoward medical condition that occurs in a participant while participating in this clinical study. It can be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study drug, whether or not related to the study. An SAE was any untoward medical occurrence that at any dose met one, more of the following criteria: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event. The relationship of the study drug in causing or contributing to the AE whether unrelated, possibly related, or probably related was decided by investigator medical judgment.
Change From Baseline in Hematology Parameters: Hematocrit Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameter hematocrit was reported.
Change From Baseline in Hematology Parameters: Leukocytes and Platelets Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in hematology parameters (Leukocytes and Platelets) expressed in 10\^9 cells per liter were reported.
Change From Baseline in Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells Baseline up to Month 12 Monocytes/leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in monocytes to leukocytes ratio is reported in terms of percentage of cells.
Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase, and lactate dehydrogenase) were reported.
Change From Baseline in Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells Baseline up to Month 12 Neutrophils/leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in neutrophils to leukocytes ratio is reported in terms of percentage of cells.
Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in Serum chemistry parameters (bilirubin, creatinine and direct bilirubin) expressed in micromole per liter (mcmol/L) were reported.
Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs parameters diastolic blood pressure and systolic blood pressure and according to the assessment position (supine and standing) expressed in millimeter(s) of mercury (mmHg) was reported.
Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval Baseline to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change From Baseline in ECG parameters aggregate PR interval, aggregate QRS duration, aggregate QT interval, and aggregate QTc interval expressed in millisecond (msec) was reported.
Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells Baseline up to Month 12 Lymphocytes/leukocytes was measured in percentages (%). Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in lymphocytes to leukocytes ratio is reported in terms of percentage of cells.
Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI] Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs parameter BMI was reported.
Change From Baseline in Hemoglobin A1c (HbA1c) Baseline up to Month 12 HbA1c is the glycosylated fraction of hemoglobin A. It is measured to identify average blood glucose concentration over prolonged periods of time. Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in HbA1c was reported.
Change From Baseline in Vital Signs Parameter: Height Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs height was reported.
Change From Baseline in Vital Signs Parameter: Weight Baseline up to Month 12 Baseline was defined as the last measurement taken before the first dose of open-label treatment on Day 1 of current study. Change from baseline in vital signs parameter weight was reported.
Number of Participants With Clinically Significant Abnormal Physical Examination Findings Baseline up to Month 12 Physical examination included examination of the following body areas and systems: Head, Eyes, Ears, Nose, Mouth, Throat, Neck (including Thyroid), Thorax, Abdomen, Urogenital, Extremities, Neurological, Skin and Mucosae and Others. Any clinically significant abnormalities in physical examination were judged by the investigator. Only non-zero values are reported.
- Secondary Outcome Measures
Name Time Method Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12 Baseline up to Months 1, 3, 6, 9, 12 The CGI consists of 2 reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms. The CGI severity scale (CGI-TS-S) ranges from 1 = "not ill at all" to 7 = "among the most extremely ill." Higher scores represent more severe symptoms. A negative change from baseline indicates improvement.
Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12 Baseline up to Months 1, 3, 6, 9 and 12 The YGTSS was a clinician-completed rating scale used to quantify overall tic severity as well as specific subdomains of tic number, frequency, duration, intensity, and complexity. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a tic severity score ranging from 0 to 50. The YGTSS also provides for an overall impairment rating (0 = ''none'' to 50 = ''severe''). YGTSS-GS is the total of YGTSS-TTS and YGTSS-I. The maximum YGTSS Global score is 100, while the maximum motor score is 25, the maximum vocal score is 25, and the maximum impairment score is 50. Higher scores indicate more severe tics. A negative change from baseline indicates improvement.
Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12 Months 1, 3, 6, 9 and 12 The CGI consists of 2 reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms. The scale ranges from 1 = "very much improved" to 7 = very much worse" for the CGI-TS-I. Higher score represent more severe symptoms.
Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12 Baseline up to Months 1, 3, 6, 9 and 12 The YGTSS was a clinician-completed rating scale used to quantify overall tic severity as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represent more severe symptoms. A negative change from baseline indicates improvement.
Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12 Baseline up to Months 1, 3, 6, 9 and 12 The YGTSS was a clinician-completed rating scale used to quantify overall tic severity as well as specific subdomains of tic number, frequency, duration, intensity, and complexity. Each of these subdomains was scored, on 0 to 5 scale, separately for an overall impairment rating from (0 = ''none'' to 50 = ''severe''). Higher score represent more severe symptoms. A negative change from baseline indicates improvement.
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 Baseline up to Months 1, 3, 6, 9 and 12 The C\&A-GTS-QOL was a 27-item questionnaire specific to TS patients that asks the patient to assess the extent to which their quality of life is impacted by their symptoms. The C\&A-GTS-QOL consists of 6 subscales (cognitive \[score range 0-32\], coprophenomena \[range 0-12\], psychological \[range 0-24\], physical \[range 0-12\], obsessive-compulsive \[range 0-16\], and activities of daily living (ADL) \[range 0-12\] and uses a 5 point Likert scale ranging from no problem to extreme problem. Scores for six subscales were generated by summing items and, for ease of interpretation, transformation to a range of 0 to 100 (100\* \[(observed score-min possible score)/(max possible score-min possible score)\]). Total score, resulted from sum of the subscale scores, was also normalized to a 0 to 100 range. Higher score indicated worst quality of life. A negative change from baseline indicates better quality of life.
Trial Locations
- Locations (65)
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
The University of Chicago Hospitals
🇺🇸Chicago, Illinois, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Center for Psychiatry and Behavioral Medicine Inc.
🇺🇸Las Vegas, Nevada, United States
Cincinnati Childrens Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Road Runner Research Ltd.
🇺🇸San Antonio, Texas, United States
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdansk, Poland
NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis
🇵🇱Katowice, Poland
Yale School of Medicine
🇺🇸New Haven, Connecticut, United States
CHU Sainte-Justine
🇨🇦Montreal, Quebec, Canada
Gdanskie Centrum Zdrowia Sp z o.o.
🇵🇱Gdańsk, Poland
Alivation Research, LLC
🇺🇸Lincoln, Nebraska, United States
Centrum Medyczne Plejady
🇵🇱Krakow, Poland
The NeuroCognitive Institute
🇺🇸Mount Arlington, New Jersey, United States
University of Miami
🇺🇸Miami, Florida, United States
Centrum Bada Klinicznych PI-House Sp. z o.o.
🇵🇱Gdansk, Poland
Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
🇵🇱Krakow, Poland
University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
Med-Polonia Sp. z o. o.
🇵🇱Poznań, Poland
Coastal Pediatric Research
🇺🇸Charleston, South Carolina, United States
APG Research LLC
🇺🇸Orlando, Florida, United States
PCSD-Feighner Research
🇺🇸San Diego, California, United States
University of South Florida
🇺🇸Saint Petersburg, Florida, United States
Syrentis Clinical Research
🇺🇸Santa Ana, California, United States
Michigan Clinical Research Institute PC
🇺🇸Ann Arbor, Michigan, United States
Helen DeVos Children's Hospital / Spectrum Health Medical Group
🇺🇸Wyoming, Michigan, United States
Quest Therapeutics of Avon Lake
🇺🇸Avon Lake, Ohio, United States
North Texas Clinical Trials
🇺🇸Fort Worth, Texas, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of Virginia
🇺🇸Charlottesville, Virginia, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Access Clinical Trials, Inc.
🇺🇸Nashville, Tennessee, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Sarkis Clinical Trials
🇺🇸Gainesville, Florida, United States
MedBio Trials
🇺🇸North Miami, Florida, United States
Research in Miami Inc.
🇺🇸Hialeah, Florida, United States
Northwest Florida Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, United States
Pediatric Epilepsy and Neurology Specialists
🇺🇸Tampa, Florida, United States
Pediatric Neurology, PA
🇺🇸Winter Park, Florida, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
Meridian Clinical Research
🇺🇸Savannah, Georgia, United States
AMR - Baber Research Inc.
🇺🇸Naperville, Illinois, United States
Psychiatric Associates
🇺🇸Overland Park, Kansas, United States
Neurobehavioral Medicine Group
🇺🇸Bloomfield Hills, Michigan, United States
New York Neurology Associates P.C
🇺🇸New York, New York, United States
Movement Disorders Center
🇺🇸Saint Louis, Missouri, United States
St. Charles Psychiatric Associates dba Midwest Research Group
🇺🇸Saint Charles, Missouri, United States
Hapworth Research Inc.
🇺🇸New York, New York, United States
Mount Sinai School of Medicine
🇺🇸New York, New York, United States
Suburban Research Associates
🇺🇸Media, Pennsylvania, United States
North Star Medical Research LLC
🇺🇸Middleburg Heights, Ohio, United States
Houston Clinical Trials LLC
🇺🇸Bellaire, Texas, United States
Relaro Medical Trials
🇺🇸Dallas, Texas, United States
Center for Pediatric Excellence
🇨🇦Ottawa, Ontario, Canada
The Kids Clinic Inc
🇨🇦Ajax, Ontario, Canada
CHU Poitiers
🇫🇷Poitiers, France
Pharmakologisches Studienzentrum Chemnitz GmbH
🇩🇪Mittweida, Germany
Harmonex Neuroscience Research
🇺🇸Dothan, Alabama, United States
Advanced Research Center Inc.
🇺🇸Anaheim, California, United States
UCLA
🇺🇸Los Angeles, California, United States
Mood Disorders Consulting Medicine PLLC
🇺🇸New York, New York, United States
Finger Lakes Clinical Research
🇺🇸Rochester, New York, United States
Clinical Research Center of NJ
🇺🇸Voorhees, New Jersey, United States
Noetic Psychiatry
🇺🇸Springville, Utah, United States
Eastside Therapeutic Resource Inc dba Core Clinical Research
🇺🇸Everett, Washington, United States