Tourette Syndrome in ChildrenPhase 2NCT04114539
Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension
A Phase 2 study testing Ecopipam in people with Tourette Syndrome in Children and Tourette Syndrome in Adolescence, run by Emalex Biosciences Inc. at 65 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Tourette Syndrome in Children, Tourette Syndrome in Adolescence
- What is being tested?
- Ecopipam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 124 participants
- Where
- 65 sitesin 5 countries
- Study dates
- 4 Oct 2019to 11 Nov 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
65 sites in 5 countries.
65 sites in 5 countries
United States53 sites
Advanced Research Center Inc.
Anaheim, California, 92805
Michigan Clinical Research Institute PC
Ann Arbor, Michigan, 48105
Rare Disease Research, LLC
Atlanta, Georgia, 30318
Quest Therapeutics of Avon Lake
Avon Lake, Ohio, 44012-1004
Houston Clinical Trials LLC
Bellaire, Texas, 77401
Neurobehavioral Medicine Group
Bloomfield Hills, Michigan, 48302-1952
Massachusetts General Hospital
Boston, Massachusetts, 02114
Coastal Pediatric Research
Charleston, South Carolina, 29414
University of Virginia
Charlottesville, Virginia, 22908-0829
Rush University Medical Center
Chicago, Illinois, 60612-3841
The University of Chicago Hospitals
Chicago, Illinois, 60637-1447
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229-3026
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Relaro Medical Trials
Dallas, Texas, 75243
Harmonex Neuroscience Research
Dothan, Alabama, 36303
Eastside Therapeutic Resource Inc dba Core Clinical Research
Everett, Washington, 98201-4077
North Texas Clinical Trials
Fort Worth, Texas, 76104
Sarkis Clinical Trials
Gainesville, Florida, 32607
Northwest Florida Clinical Research Group, LLC
Gulf Breeze, Florida, 32561
Research in Miami Inc.
Hialeah, Florida, 33013
Baylor College of Medicine
Houston, Texas, 77030
Center for Psychiatry and Behavioral Medicine Inc.
Las Vegas, Nevada, 89128
Alivation Research, LLC
Lincoln, Nebraska, 68526
UCLA
Los Angeles, California, 90095
Suburban Research Associates
Media, Pennsylvania, 19063
University of Miami
Miami, Florida, 33136
North Star Medical Research LLC
Middleburg Heights, Ohio, 44130
The NeuroCognitive Institute
Mount Arlington, New Jersey, 07856
AMR - Baber Research Inc.
Naperville, Illinois, 60563-6510
Access Clinical Trials, Inc.
Nashville, Tennessee, 37203-6502
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-0028
Yale School of Medicine
New Haven, Connecticut, 06519
New York Neurology Associates P.C
New York, New York, 10003
Hapworth Research Inc.
New York, New York, 10019
Mount Sinai School of Medicine
New York, New York, 10029-6504
Mood Disorders Consulting Medicine PLLC
New York, New York, 10036
MedBio Trials
North Miami, Florida, 33180
APG Research LLC
Orlando, Florida, 32803
Psychiatric Associates
Overland Park, Kansas, 66211
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Finger Lakes Clinical Research
Rochester, New York, 14618
St. Charles Psychiatric Associates dba Midwest Research Group
Saint Charles, Missouri, 63304
Road Runner Research Ltd.
San Antonio, Texas, 78249-3539
PCSD-Feighner Research
San Diego, California, 92108
Syrentis Clinical Research
Santa Ana, California, 92705
Meridian Clinical Research
Savannah, Georgia, 31406
Noetic Psychiatry
Springville, Utah, 84663
Movement Disorders Center
St Louis, Missouri, 63110-1093
University of South Florida
St. Petersburg, Florida, 33701-4825
Pediatric Epilepsy and Neurology Specialists
Tampa, Florida, 33609-4181
Clinical Research Center of NJ
Voorhees Township, New Jersey, 08043-1910
Pediatric Neurology, PA
Winter Park, Florida, 32789
Helen DeVos Children's Hospital / Spectrum Health Medical Group
Wyoming, Michigan, 49418
Poland7 sites
Gdanskie Centrum Zdrowia Sp z o.o.
Gdansk, 80-542
Centrum Bada Klinicznych PI-House Sp. z o.o.
Gdansk, 80-546
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis
Katowice, 40-123
Centrum Medyczne Plejady
Krakow, 30-363
Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
Krakow, 31-503
Med-Polonia Sp. z o. o.
Poznan, 60-693
Canada3 sites
The Kids Clinic Inc
Ajax, Ontario, L1Z 0M1
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
Center for Pediatric Excellence
Ottawa, Ontario, K2G 1W2
About this study
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
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.
Read the 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
- Being tested
- Ecopipam
- Keywords
- Tourette Syndrome
- Medical subject headings
- Tourette Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EcopipamDrug
Ecopipam HCl 12.5-, 37.5-. 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings.
| Study group | Role | What they receive |
|---|---|---|
| Ecopipam | Experimental | Not described Receives: Drug: Ecopipam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related)
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.
Measured: From start of study drug administration until 30 days after last dose (Up to Month 13)
Change From Baseline in Hematology Parameters: Basophils to Leukocytes Ratio Reported in Percentage of Cells
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Eosinophils to Leukocytes Ratio Reported in Percentage of Cells
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Erythrocytes
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Hematocrit
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Hemoglobin
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Leukocytes and Platelets
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells
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.
Measured: Baseline up to Month 12
Change From Baseline in Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells
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.
Measured: Baseline up to Month 12
Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase
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.
Measured: Baseline up to Month 12
Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein
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.
Measured: Baseline up to Month 12
Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen
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.
Measured: Baseline up to Month 12
Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin
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.
Measured: Baseline up to Month 12
Change From Baseline in Hemoglobin A1c (HbA1c)
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.
Measured: Baseline up to Month 12
Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure
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.
Measured: Baseline up to Month 12
Change From Baseline in Vital Signs Parameter: Pulse Rate
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.
Measured: Baseline up to Month 12
Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI]
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.
Measured: Baseline up to Month 12
Change From Baseline in Vital Signs Parameter: Height
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.
Measured: Baseline up to Month 12
Change From Baseline in Vital Signs Parameter: Weight
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.
Measured: Baseline up to Month 12
Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval
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.
Measured: Baseline to Month 12
Number of Participants With Clinically Significant Abnormal Physical Examination Findings
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.
Measured: Baseline up to Month 12
Secondary outcomes (6)
Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at 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.
Measured: Baseline up to Months 1, 3, 6, 9 and 12
Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at 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.
Measured: Baseline up to Months 1, 3, 6, 9 and 12
Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at 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.
Measured: Baseline up to Months 1, 3, 6, 9 and 12
Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at 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.
Measured: Baseline up to Months 1, 3, 6, 9, 12
Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at 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.
Measured: Months 1, 3, 6, 9 and 12
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
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.
Measured: Baseline up to Months 1, 3, 6, 9 and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2019
- Study start4 Oct 2019
- Primary completion11 Nov 2022
- Study completion11 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Tourette Syndrome in ChildrenStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open Label
- Masking
- None (open label)
- Enrolment
- 124 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emalex Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04114539
- Sponsor's protocol ID
- EBS-101-OL-001
- Status last verified
- Nov 2023
- First submitted
- 30 Sep 2019
- Last updated
- 1 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04114539
- Acronym / sponsor ID
- EBS-101-OL-001
- Keywords
- Tourette Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 124 participants (actual)
- Lead sponsor
- Emalex Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Oct 2019 (actual)
- Primary completion
- 11 Nov 2022 (actual)
- Study completion
- 11 Nov 2022 (actual)
- First posted
- 3 Oct 2019 (actual)
- Results
- Posted 1 Dec 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open Label
| Arm | Type | Receives |
|---|---|---|
| Ecopipam | Experimental | Drug: Ecopipam |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EcopipamEcopipam HCl 12.5-, 37.5-. 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings. | Drug | Ecopipam |
Primary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related)
Time frame: 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: Basophils to Leukocytes Ratio Reported in Percentage of Cells
Time frame: 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
Time frame: 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
Time frame: 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 Hematology Parameters: Hematocrit
Time frame: 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: Hemoglobin
Time frame: 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 Hematology Parameters: Leukocytes and Platelets
Time frame: 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: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells
Time frame: 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 Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells
Time frame: 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 Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells
Time frame: 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: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase
Time frame: 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 Serum Chemistry Parameters: Albumin, Globulin and Protein
Time frame: 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 Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen
Time frame: 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 Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin
Time frame: 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 Hemoglobin A1c (HbA1c)
Time frame: 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: Diastolic Blood Pressure and Systolic Blood Pressure
Time frame: 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 Vital Signs Parameter: Pulse Rate
Time frame: 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.
Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI]
Time frame: 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 Vital Signs Parameter: Height
Time frame: 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
Time frame: 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.
Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval
Time frame: 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.
Number of Participants With Clinically Significant Abnormal Physical Examination Findings
Time frame: 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.
6 secondary outcomes
Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12
Time frame: Baseline up to Months 1, 3, 6, 9, 12
Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12
Time frame: Months 1, 3, 6, 9 and 12
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
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
65 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ecopipam in people with Tourette Syndrome in Children and Tourette Syndrome in Adolescence, run by Emalex Biosciences Inc. at 65 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 65 sites in United States, Poland, Canada, France, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Dec 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) (time frame: From start of study drug administration until 30 days after last dose (Up to Month 13)). The study lists 22 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Feb 2024.