Tourette Syndrome in Adolescence, Tourette Syndrome in Children
Conditions
Keywords
Tourette Syndrome
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
Interventions
DRUGEcopipam
Ecopipam HCl 12.5-, 37.5-. 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings.
Sponsors
Emalex Biosciences Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open Label
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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 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. |
| 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 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. |
| 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. |
| 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: 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 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: 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 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: 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 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 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: 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 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 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 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 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 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. |
| 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. |
| 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 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. |
| 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. |
Countries
Canada, France, Germany, Poland, United States
Participant flow
Recruitment details
The study was conducted at 39 sites in United States, Canada and Poland from 4 October 2019 (first participant first visit) to 11 November 2022 (last participant last visit).
Pre-assignment details
A total of 124 participants who completed the open-labelled study EBS-101-CL-001 (NCT04007991) and who met the inclusion/exclusion criteria for this study were enrolled in the current study and received study treatment. Of the 124 enrolled participants, 121 participants were included in the modified ITT (mITT) Set and Safety Set.
Participants by arm
| Arm | Count |
|---|---|
| Ecopipam HCI 2 mg/kg/Day Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks. | 121 |
| Total | 121 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 14 |
| Overall Study | Investigator Decision | 2 |
| Overall Study | Lost to Follow-up | 2 |
| Overall Study | Non-compliance with Protocol | 2 |
| Overall Study | Non-compliance with Study Drug | 1 |
| Overall Study | Others not specified | 5 |
| Overall Study | Withdrawal by Parent/Caregiver | 8 |
| Overall Study | Withdrew consent | 10 |
Baseline characteristics
| Characteristic | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Age, Continuous | 12.8 years STANDARD_DEVIATION 2.82 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 14 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 107 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 3 Participants |
| Race (NIH/OMB) Black or African American | 7 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 110 Participants |
| Region of Enrollment Canada | 6 Participants |
| Region of Enrollment Poland | 24 Participants |
| Region of Enrollment United States | 91 Participants |
| Sex: Female, Male Female | 32 Participants |
| Sex: Female, Male Male | 89 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 121 |
| other Total, other adverse events | 84 / 121 |
| serious Total, serious adverse events | 2 / 121 |
Outcome results
Primary
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.
Time frame: Baseline to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval | Aggregate PR interval | -5.6 millisecond (msec) | Standard Deviation 18.81 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval | Aggregate QRS duration | -0.6 millisecond (msec) | Standard Deviation 9.76 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval | Aggregate QT interval | 5.5 millisecond (msec) | Standard Deviation 30.26 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval | Aggregate QTc interval | 0.2 millisecond (msec) | Standard Deviation 38.53 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Basophils to Leukocytes Ratio Reported in Percentage of Cells | -0.1 percentage of cells | Standard Deviation 0.91 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Eosinophils to Leukocytes Ratio Reported in Percentage of Cells | 0.1 percentage of cells | Standard Deviation 1.52 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here Overall number of participants analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Erythrocytes | 0.04 10^12 cells per liter | Standard Deviation 0.263 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here Overall number of participants analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Hematocrit | 0.01 liter of cells per liter of blood (L/L) | Standard Deviation 0.029 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here Overall number of participants analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Hemoglobin | 0.08 millimoles per liter (mmoL/L) | Standard Deviation 0.429 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Leukocytes and Platelets | Leukocytes | 0.15 10^9 cells per liter | Standard Deviation 1.349 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Leukocytes and Platelets | Platelets | 4.1 10^9 cells per liter | Standard Deviation 51.49 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells | 0.2 percentage of cells | Standard Deviation 10.2 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells | 0.2 percentage of cells | Standard Deviation 1.43 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells | -0.3 percentage of cells | Standard Deviation 10.45 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Hemoglobin A1c (HbA1c) | 0.03 percentage of HbA1c | Standard Deviation 0.313 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase | Alanine Aminotransferase | 2.3 units per liter (U/L) | Standard Deviation 9.41 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase | Alkaline Phosphatase | -4.7 units per liter (U/L) | Standard Deviation 80.71 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase | Aspartate Aminotransferase | -0.3 units per liter (U/L) | Standard Deviation 5.48 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase | Lactase Dehydrogenase | -6.2 units per liter (U/L) | Standard Deviation 33.96 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein | Albumin | 0.6 grams per liter (g/L) | Standard Deviation 2.87 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein | Globulin | 1.2 grams per liter (g/L) | Standard Deviation 2.92 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein | Protein | 1.9 grams per liter (g/L) | Standard Deviation 4.63 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Potassium | 0.02 millimoles per liter (mmol/L) | Standard Deviation 0.684 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Sodium | -0.1 millimoles per liter (mmol/L) | Standard Deviation 2.22 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Bicarbonate | -0.6 millimoles per liter (mmol/L) | Standard Deviation 3.57 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Calcium | 0.02 millimoles per liter (mmol/L) | Standard Deviation 0.153 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Chloride | -0.2 millimoles per liter (mmol/L) | Standard Deviation 2.54 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Cholesterol | 0.20 millimoles per liter (mmol/L) | Standard Deviation 0.704 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Glucose | -0.06 millimoles per liter (mmol/L) | Standard Deviation 0.781 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Phosphate | -0.02 millimoles per liter (mmol/L) | Standard Deviation 0.294 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Triglyceride | -0.09 millimoles per liter (mmol/L) | Standard Deviation 0.625 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen | Urea Nitrogen | -0.07 millimoles per liter (mmol/L) | Standard Deviation 1.176 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin | Bilirubin | -0.6 mcmol/L | Standard Deviation 4.78 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin | Creatinine | 6.2 mcmol/L | Standard Deviation 8.85 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin | Direct Bilirubin | -0.1 mcmol/L | Standard Deviation 0.77 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI] | 1.0 kilograms per square meter (kg/m^2) | Standard Deviation 4.58 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure | Diastolic Blood Pressure: Standing | 1.6 mmHg | Standard Deviation 10.65 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure | Systolic Blood Pressure | 0.3 mmHg | Standard Deviation 11.47 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure | Diastolic Blood Pressure | 0.9 mmHg | Standard Deviation 9.96 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure | Diastolic Blood Pressure: Supine | 0.6 mmHg | Standard Deviation 8.58 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure | Systolic Blood Pressure: Supine | 1.8 mmHg | Standard Deviation 11.35 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure | Systolic Blood Pressure: Standing | -1.1 mmHg | Standard Deviation 12.15 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Height | 4.0 centimeter (cm) | Standard Deviation 3.9 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Pulse Rate | Pulse Rate | 0.4 beats per minute (bpm) | Standard Deviation 13.76 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Pulse Rate | Pulse Rate Supine | 0.6 beats per minute (bpm) | Standard Deviation 14.2 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Pulse Rate | Pulse Rate Standing | 1.8 beats per minute (bpm) | Standard Deviation 18.09 |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Vital Signs Parameter: Weight | 5.4 kilogram (kg) | Standard Deviation 13.53 |
Primary
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.
Time frame: From start of study drug administration until 30 days after last dose (Up to Month 13)
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with any AEs | 84 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with SAEs | 2 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with unrelated AEs | 44 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with possibly related AEs | 27 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with probably related AEs | 13 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with unrelated SAEs | 1 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with possibly related SAEs | 1 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) | Participants with probably related SAEs | 0 Participants |
Primary
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.
Time frame: Baseline up to Month 12
Population: The Safety Set included all participants who received at least one dose of study drug from the open labelled study. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Clinically Significant Abnormal Physical Examination Findings | Other | 1 Participants |
| Ecopipam HCI 2 mg/kg/Day | Number of Participants With Clinically Significant Abnormal Physical Examination Findings | Neurological | 1 Participants |
Secondary
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.
Time frame: Baseline up to Months 1, 3, 6, 9, 12
Population: The mITT set included all participants who received at least one dose of study drug and had at least one post Baseline scoring of YGTSS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12 | Change at Month 1 | -0.8 score on a scale | Standard Deviation 1.02 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12 | Change at Month 3 | -0.8 score on a scale | Standard Deviation 1.1 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12 | Change at Month 6 | -1.0 score on a scale | Standard Deviation 1.1 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12 | Change at Month 9 | -1.2 score on a scale | Standard Deviation 1.13 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12 | Change at Month 12 | -1.2 score on a scale | Standard Deviation 1.21 |
Secondary
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.
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Population: The mITT set included all participants who received at least one dose of study drug and had at least one post Baseline scoring of YGTSS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | 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 | Change at Month 1 | -4.0 score on a scale | Standard Deviation 11.3 |
| Ecopipam HCI 2 mg/kg/Day | 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 | Change at Month 3 | -4.5 score on a scale | Standard Deviation 12.3 |
| Ecopipam HCI 2 mg/kg/Day | 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 | Change at Month 6 | -7.7 score on a scale | Standard Deviation 13.97 |
| Ecopipam HCI 2 mg/kg/Day | 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 | Change at Month 9 | -6.7 score on a scale | Standard Deviation 16.91 |
| Ecopipam HCI 2 mg/kg/Day | 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 | Change at Month 12 | -8.0 score on a scale | Standard Deviation 16.17 |
Secondary
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.
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Population: The mITT set included all participants who received at least one dose of study drug and had at least one post Baseline scoring of YGTSS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12 | Change at Month 1 | -13.8 score on a scale | Standard Deviation 15.07 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12 | Change at Month 3 | -15.9 score on a scale | Standard Deviation 16.91 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12 | Change at Month 6 | -21.0 score on a scale | Standard Deviation 17.64 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12 | Change at Month 9 | -24.0 score on a scale | Standard Deviation 17.31 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12 | Change at Month 12 | -23.8 score on a scale | Standard Deviation 19.11 |
Secondary
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.
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Population: The mITT set included all participants who received at least one dose of study drug and had at least one post Baseline scoring of YGTSS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12 | Change at Month 1 | -7.2 score on a scale | Standard Deviation 9.74 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12 | Change at Month 3 | -8.3 score on a scale | Standard Deviation 10.88 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12 | Change at Month 6 | -10.4 score on a scale | Standard Deviation 10.49 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12 | Change at Month 9 | -12.5 score on a scale | Standard Deviation 11.11 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12 | Change at Month 12 | -12.1 score on a scale | Standard Deviation 11.59 |
Secondary
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.
Time frame: Baseline up to Months 1, 3, 6, 9 and 12
Population: The mITT set included all participants who received at least one dose of study drug and had at least one post Baseline scoring of YGTSS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12 | Change at Month 1 | -6.5 score on a scale | Standard Deviation 7.1 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12 | Change at Month 3 | -7.5 score on a scale | Standard Deviation 8.04 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12 | Change at Month 6 | -10.6 score on a scale | Standard Deviation 8.88 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12 | Change at Month 9 | -11.5 score on a scale | Standard Deviation 8.34 |
| Ecopipam HCI 2 mg/kg/Day | Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12 | Change at Month 12 | -11.7 score on a scale | Standard Deviation 9.48 |
Secondary
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.
Time frame: Months 1, 3, 6, 9 and 12
Population: The mITT set included all participants who received at least one dose of study drug and had at least one post Baseline scoring of YGTSS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure and Number Analyzed signifies participants who were evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12 | Month 9 | 2.0 score on a scale | Standard Deviation 1.02 |
| Ecopipam HCI 2 mg/kg/Day | Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12 | Month 1 | 2.7 score on a scale | Standard Deviation 1.05 |
| Ecopipam HCI 2 mg/kg/Day | Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12 | Month 3 | 2.5 score on a scale | Standard Deviation 1.24 |
| Ecopipam HCI 2 mg/kg/Day | Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12 | Month 6 | 2.2 score on a scale | Standard Deviation 1.04 |
| Ecopipam HCI 2 mg/kg/Day | Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12 | Month 12 | 2.0 score on a scale | Standard Deviation 1.09 |