A Phase 2 interventional study of Ecopipam in Tourette Syndrome in Children and Tourette Syndrome in Adolescence, sponsored by Emalex Biosciences Inc.. Completed at 65 sites in 5 countries. Open to participants aged 6 Years to 18 Years. Per ClinicalTrials.gov, last updated 2024-02-01.
Sponsored by Emalex Biosciences Inc. · Phase 2, Interventional, and Treatment
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.
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
220 studies on the registry are indexed under Tourette Syndrome; 37 are open to participants now.
This study's enrollment of 124 is above the median of 34 across 166 interventional studies indexed under Tourette Syndrome.
Browse Tourette Syndrome studies →Emalex Biosciences Inc. is the lead sponsor of 17 studies on the registry; 1 is open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 5 (42%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Drug: Ecopipam
Ecopipam HCl 12.5-, 37.5-. 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings.
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)
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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).
| Milestone | Ecopipam HCl 2 mg/kg/Day |
|---|---|
| Started | 124 |
| Safety set (treated) | 121 |
| Modified intention-to-treat set | 121 |
| Completed | 80 |
| Not completed | 44 |
| Withdrew: Adverse event | 14 |
| Withdrew: Lost to follow-up | 2 |
| Withdrew: Non-compliance with protocol | 2 |
| Withdrew: Non-compliance with study drug | 1 |
| Withdrew: Withdrawal by parent/caregiver | 8 |
| Withdrew: Withdrew consent | 10 |
| Withdrew: Others not specified | 5 |
| Withdrew: Investigator decision | 2 |
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.
| Participants | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Participants with any AEs | 84 |
| Participants with SAEs | 2 |
| Participants with unrelated AEs | 44 |
| Participants with possibly related AEs | 27 |
| Participants with probably related AEs | 13 |
| Participants with unrelated SAEs | 1 |
| Participants with possibly related SAEs | 1 |
| Participants with probably related SAEs | 0 |
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.
| percentage of cells | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Basophils to Leukocytes Ratio Reported in Percentage of Cells | -0.1 ± 0.91 |
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.
| percentage of cells | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Eosinophils to Leukocytes Ratio Reported in Percentage of Cells | 0.1 ± 1.52 |
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.
| 10^12 cells per liter | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Erythrocytes | 0.04 ± 0.263 |
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.
| liter of cells per liter of blood (L/L) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Hematocrit | 0.01 ± 0.029 |
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.
| millimoles per liter (mmoL/L) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Hemoglobin | 0.08 ± 0.429 |
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.
| 10^9 cells per liter | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Leukocytes | 0.15 ± 1.349 |
| Platelets | 4.1 ± 51.49 |
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.
| percentage of cells | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells | 0.2 ± 10.20 |
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.
| percentage of cells | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells | 0.2 ± 1.43 |
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.
| percentage of cells | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells | -0.3 ± 10.45 |
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.
| units per liter (U/L) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Alanine Aminotransferase | 2.3 ± 9.41 |
| Alkaline Phosphatase | -4.7 ± 80.71 |
| Aspartate Aminotransferase | -0.3 ± 5.48 |
| Lactase Dehydrogenase | -6.2 ± 33.96 |
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.
| grams per liter (g/L) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Albumin | 0.6 ± 2.87 |
| Globulin | 1.2 ± 2.92 |
| Protein | 1.9 ± 4.63 |
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.
| millimoles per liter (mmol/L) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Bicarbonate | -0.6 ± 3.57 |
| Calcium | 0.02 ± 0.153 |
| Chloride | -0.2 ± 2.54 |
| Cholesterol | 0.20 ± 0.704 |
| Glucose | -0.06 ± 0.781 |
| Phosphate | -0.02 ± 0.294 |
| Potassium | 0.02 ± 0.684 |
| Sodium | -0.1 ± 2.22 |
| Triglyceride | -0.09 ± 0.625 |
| Urea Nitrogen | -0.07 ± 1.176 |
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.
| mcmol/L | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Bilirubin | -0.6 ± 4.78 |
| Creatinine | 6.2 ± 8.85 |
| Direct Bilirubin | -0.1 ± 0.77 |
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.
| percentage of HbA1c | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Hemoglobin A1c (HbA1c) | 0.03 ± 0.313 |
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.
| mmHg | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Diastolic Blood Pressure | 0.9 ± 9.96 |
| Diastolic Blood Pressure: Supine | 0.6 ± 8.58 |
| Diastolic Blood Pressure: Standing | 1.6 ± 10.65 |
| Systolic Blood Pressure | 0.3 ± 11.47 |
| Systolic Blood Pressure: Supine | 1.8 ± 11.35 |
| Systolic Blood Pressure: Standing | -1.1 ± 12.15 |
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.
| beats per minute (bpm) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Pulse Rate | 0.4 ± 13.76 |
| Pulse Rate Supine | 0.6 ± 14.20 |
| Pulse Rate Standing | 1.8 ± 18.09 |
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.
| kilograms per square meter (kg/m^2) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI] | 1.0 ± 4.58 |
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.
| centimeter (cm) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Vital Signs Parameter: Height | 4.0 ± 3.90 |
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.
| kilogram (kg) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change From Baseline in Vital Signs Parameter: Weight | 5.4 ± 13.53 |
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.
| millisecond (msec) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Aggregate PR interval | -5.6 ± 18.81 |
| Aggregate QRS duration | -0.6 ± 9.76 |
| Aggregate QT interval | 5.5 ± 30.26 |
| Aggregate QTc interval | 0.2 ± 38.53 |
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.
| Participants | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Neurological | 1 |
| Other | 1 |
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.
| score on a scale | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change at Month 1 | -6.5 ± 7.10 |
| Change at Month 3 | -7.5 ± 8.04 |
| Change at Month 6 | -10.6 ± 8.88 |
| Change at Month 9 | -11.5 ± 8.34 |
| Change at Month 12 | -11.7 ± 9.48 |
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.
| score on a scale | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change at Month 1 | -7.2 ± 9.74 |
| Change at Month 3 | -8.3 ± 10.88 |
| Change at Month 6 | -10.4 ± 10.49 |
| Change at Month 9 | -12.5 ± 11.11 |
| Change at Month 12 | -12.1 ± 11.59 |
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.
| score on a scale | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change at Month 1 | -13.8 ± 15.07 |
| Change at Month 3 | -15.9 ± 16.91 |
| Change at Month 6 | -21.0 ± 17.64 |
| Change at Month 9 | -24.0 ± 17.31 |
| Change at Month 12 | -23.8 ± 19.11 |
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.
| score on a scale | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change at Month 1 | -0.8 ± 1.02 |
| Change at Month 3 | -0.8 ± 1.10 |
| Change at Month 6 | -1.0 ± 1.10 |
| Change at Month 9 | -1.2 ± 1.13 |
| Change at Month 12 | -1.2 ± 1.21 |
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.
| score on a scale | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Month 1 | 2.7 ± 1.05 |
| Month 3 | 2.5 ± 1.24 |
| Month 6 | 2.2 ± 1.04 |
| Month 9 | 2.0 ± 1.02 |
| Month 12 | 2.0 ± 1.09 |
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.
| score on a scale | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Change at Month 1 | -4.0 ± 11.30 |
| Change at Month 3 | -4.5 ± 12.30 |
| Change at Month 6 | -7.7 ± 13.97 |
| Change at Month 9 | -6.7 ± 16.91 |
| Change at Month 12 | -8.0 ± 16.17 |
Collected over From Baseline up to 30 days after last dose of the study drug (Up to Month 13). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ecopipam HCI 2 mg/kg/Day | 0/121 (0%) | 2/121 (1.7%) | 84/121 (69.4%) |
| Event | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Abdominal pain upperGastrointestinal disorders | 1/121 |
| NauseaGastrointestinal disorders | 1/121 |
| Non-cardiac chest painGeneral disorders | 1/121 |
| Rib fractureInjury, poisoning and procedural complications | 1/121 |
| Skin lacerationInjury, poisoning and procedural complications | 1/121 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/121 |
| Loss of consciousnessNervous system disorders | 1/121 |
| Obsessive thoughtsPsychiatric disorders | 1/121 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 1/121 |
| PneumothoraxRespiratory, thoracic and mediastinal disorders | 1/121 |
| Event | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| NasopharyngitisInfections and infestations | 17/121 |
| AnxietyPsychiatric disorders | 11/121 |
| DiarrhoeaGastrointestinal disorders | 9/121 |
| HeadacheNervous system disorders | 9/121 |
| InsomniaPsychiatric disorders | 9/121 |
| SomnolenceNervous system disorders | 8/121 |
| PyrexiaGeneral disorders | 7/121 |
| Upper respiratory tract infectionInfections and infestations | 7/121 |
| NauseaGastrointestinal disorders | 6/121 |
| COVID-19Infections and infestations | 6/121 |
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.
| Age, Continuous(years) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Mean | 12.8 ± 2.82 |
| Sex: Female, Male(Participants) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Female | 32 |
| Male | 89 |
| Ethnicity (NIH/OMB)(Participants) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Hispanic or Latino | 14 |
| Not Hispanic or Latino | 107 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 3 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 7 |
| White | 110 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
| Region of Enrollment(Participants) | Ecopipam HCI 2 mg/kg/Day |
|---|---|
| Canada | 6 |
| United States | 91 |
| Poland | 24 |
Documents are hosted by the registry — open the source record to download them.
This study is completed, as verified in Nov 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Emalex Biosciences Inc.