Trial Outcomes & Findings for Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension (NCT NCT04114539)

Last Updated: 2024-02-01

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

124 participants

Primary outcome timeframe

From start of study drug administration until 30 days after last dose (Up to Month 13)

Results posted on

2024-02-01

Participant Flow

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).

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.

Participant milestones

Participant milestones
Measure	Ecopipam HCl 2 mg/kg/Day Participants received ecopipam hydrochloride (HCl) tablets at a targeted dose of 2 milligram per kilogram per day (mg/kg/day), orally, once daily for up to 52 weeks.
Overall Study STARTED	124
Overall Study Safety Set (Treated)	121
Overall Study Modified Intention-to-Treat Set	121
Overall Study COMPLETED	80
Overall Study NOT COMPLETED	44

Reasons for withdrawal

Reasons for withdrawal
Measure	Ecopipam HCl 2 mg/kg/Day Participants received ecopipam hydrochloride (HCl) tablets at a targeted dose of 2 milligram per kilogram per day (mg/kg/day), orally, once daily for up to 52 weeks.
Overall Study Adverse Event	14
Overall Study Lost to Follow-up	2
Overall Study Non-compliance with Protocol	2
Overall Study Non-compliance with Study Drug	1
Overall Study Withdrawal by Parent/Caregiver	8
Overall Study Withdrew consent	10
Overall Study Others not specified	5
Overall Study Investigator Decision	2

Baseline Characteristics

Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ecopipam HCI 2 mg/kg/Day n=121 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Age, Continuous	12.8 years STANDARD_DEVIATION 2.82 • n=39 Participants
Sex: Female, Male Female	32 Participants n=39 Participants
Sex: Female, Male Male	89 Participants n=39 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	14 Participants n=39 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	107 Participants n=39 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants
Race (NIH/OMB) Asian	3 Participants n=39 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants
Race (NIH/OMB) Black or African American	7 Participants n=39 Participants
Race (NIH/OMB) White	110 Participants n=39 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=39 Participants
Region of Enrollment Canada	6 Participants n=39 Participants
Region of Enrollment United States	91 Participants n=39 Participants
Region of Enrollment Poland	24 Participants n=39 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=121 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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
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
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
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
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
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
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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 outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Hematology Parameters: Erythrocytes	0.04 10^12 cells per liter Standard Deviation 0.263

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Hematology Parameters: Hematocrit	0.01 liter of cells per liter of blood (L/L) Standard Deviation 0.029

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=38 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Hematology Parameters: Hemoglobin	0.08 millimoles per liter (mmoL/L) Standard Deviation 0.429

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Hematology Parameters: Leukocytes and Platelets Leukocytes	0.15 10^9 cells per liter Standard Deviation 1.349
Change From Baseline in Hematology Parameters: Leukocytes and Platelets Platelets	4.1 10^9 cells per liter Standard Deviation 51.49

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells	0.2 percentage of cells Standard Deviation 10.20

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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 outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=37 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=29 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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
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
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 outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=29 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein Albumin	0.6 grams per liter (g/L) Standard Deviation 2.87
Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein Globulin	1.2 grams per liter (g/L) Standard Deviation 2.92
Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein Protein	1.9 grams per liter (g/L) Standard Deviation 4.63

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=29 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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
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
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
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
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
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
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
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
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=29 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin Bilirubin	-0.6 mcmol/L Standard Deviation 4.78
Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin Creatinine	6.2 mcmol/L Standard Deviation 8.85
Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin Direct Bilirubin	-0.1 mcmol/L Standard Deviation 0.77

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=30 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Hemoglobin A1c (HbA1c)	0.03 percentage of HbA1c Standard Deviation 0.313

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=75 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure Diastolic Blood Pressure	0.9 mmHg Standard Deviation 9.96
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
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
Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure Systolic Blood Pressure	0.3 mmHg Standard Deviation 11.47
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
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=75 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Vital Signs Parameter: Pulse Rate Pulse Rate	0.4 beats per minute (bpm) Standard Deviation 13.76
Change From Baseline in Vital Signs Parameter: Pulse Rate Pulse Rate Supine	0.6 beats per minute (bpm) Standard Deviation 14.20
Change From Baseline in Vital Signs Parameter: Pulse Rate Pulse Rate Standing	1.8 beats per minute (bpm) Standard Deviation 18.09

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=75 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=75 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Vital Signs Parameter: Height	4.0 centimeter (cm) Standard Deviation 3.90

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=75 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Change From Baseline in Vital Signs Parameter: Weight	5.4 kilogram (kg) Standard Deviation 13.53

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=73 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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
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
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=74 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Number of Participants With Clinically Significant Abnormal Physical Examination Findings Neurological	1 Participants
Number of Participants With Clinically Significant Abnormal Physical Examination Findings Other	1 Participants

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=119 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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 7.10
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
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
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
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 outcome

Timeframe: Baseline up to Months 1, 3, 6, 9 and 12

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=119 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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
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
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
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 outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=119 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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
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
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
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 outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=119 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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 1.10
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 1.10
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
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 outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=120 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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
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
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
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
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

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=117 Participants Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
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 11.30
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 12.30
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
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
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

Adverse Events

Ecopipam HCI 2 mg/kg/Day

Serious events: 2 serious events

Other events: 84 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ecopipam HCI 2 mg/kg/Day n=121 participants at risk Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Gastrointestinal disorders Abdominal pain upper	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Nausea	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
General disorders Non-cardiac chest pain	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Rib fracture	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Skin laceration	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Musculoskeletal and connective tissue disorders Arthralgia	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Loss of consciousness	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Obsessive thoughts	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Respiratory, thoracic and mediastinal disorders Pneumothorax	0.83% 1/121 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)

Other adverse events

Other adverse events
Measure	Ecopipam HCI 2 mg/kg/Day n=121 participants at risk Participants received ecopipam HCl tablets at a targeted dose of 2 mg/kg/day, orally, once daily for up to 52 weeks.
Congenital, familial and genetic disorders Tourette's disorder	0.83% 1/121 • Number of events 3 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Ear and labyrinth disorders Vertigo	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Eye disorders Vision blurred	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Eye disorders Visual impairment	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Diarrhoea	7.4% 9/121 • Number of events 10 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Nausea	5.0% 6/121 • Number of events 6 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Abdominal pain upper	4.1% 5/121 • Number of events 5 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Vomiting	4.1% 5/121 • Number of events 6 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Abdominal pain	3.3% 4/121 • Number of events 7 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Gastrooesophageal reflux disease	2.5% 3/121 • Number of events 3 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Constipation	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Tooth impacted	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Abdominal discomfort	0.83% 1/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Gastrointestinal disorders Tooth resorption	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
General disorders Pyrexia	5.8% 7/121 • Number of events 9 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
General disorders Fatigue	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
General disorders Malaise	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
General disorders Non-cardiac chest pain	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Nasopharyngitis	14.0% 17/121 • Number of events 28 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Upper respiratory tract infection	5.8% 7/121 • Number of events 7 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations COVID-19	5.0% 6/121 • Number of events 6 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Influenza	3.3% 4/121 • Number of events 4 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Rhinitis	3.3% 4/121 • Number of events 8 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Pharyngitis	2.5% 3/121 • Number of events 4 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Bronchitis	1.7% 2/121 • Number of events 3 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Sinusitis	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Viral infection	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Adenoiditis	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Body tinea	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Folliculitis	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Otitis media	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Paronychia	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Pharyngitis streptococcal	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Pneumonia	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Staphylococcal infection	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Tonsillitis	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Urinary tract infection	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Infections and infestations Varicella	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Arthropod bite	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Contusion	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Fall	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Ligament sprain	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Skin abrasion	1.7% 2/121 • Number of events 3 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Skin laceration	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Foot fracture	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Joint injury	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Limb injury	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Penis injury	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Testicular injury	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Injury, poisoning and procedural complications Tooth injury	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Investigations Weight decreased	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Investigations Hepatic enzyme increased	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Metabolism and nutrition disorders Decreased appetite	4.1% 5/121 • Number of events 7 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Musculoskeletal and connective tissue disorders Myalgia	1.7% 2/121 • Number of events 3 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Musculoskeletal and connective tissue disorders Pain in extremity	1.7% 2/121 • Number of events 3 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Musculoskeletal and connective tissue disorders Arthritis	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Musculoskeletal and connective tissue disorders Joint lock	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Headache	7.4% 9/121 • Number of events 9 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Somnolence	6.6% 8/121 • Number of events 8 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Dizziness	2.5% 3/121 • Number of events 3 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Syncope	2.5% 3/121 • Number of events 4 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Migraine	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Paraesthesia	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Tremor	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Akathisia	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Balance disorder	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Formication	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Narcolepsy	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Postural tremor	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Nervous system disorders Tongue biting	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Anxiety	9.1% 11/121 • Number of events 12 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Insomnia	7.4% 9/121 • Number of events 9 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Depression	4.1% 5/121 • Number of events 8 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Tic	4.1% 5/121 • Number of events 6 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Aggression	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Agitation	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Depressed mood	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Depressive symptom	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Dissociative disorder	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Suicidal ideation	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Attention deficit hyperactivity disorder	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Bruxism	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Compulsions	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Initial insomnia	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Intentional self-injury	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Irritability	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Major depression	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Mental disorder	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Mood altered	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Mood swings	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Obsessive-compulsive disorder	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Panic attack	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Restlessness	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Sleep disorder	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Social anxiety disorder	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Suicidal behaviour	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Psychiatric disorders Terminal insomnia	0.83% 1/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Reproductive system and breast disorders Amenorrhoea	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Reproductive system and breast disorders Polycystic ovaries	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Respiratory, thoracic and mediastinal disorders Nasal congestion	2.5% 3/121 • Number of events 4 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Respiratory, thoracic and mediastinal disorders Cough	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Respiratory, thoracic and mediastinal disorders Paranasal sinus hypersecretion	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Skin and subcutaneous tissue disorders Rash	1.7% 2/121 • Number of events 2 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Skin and subcutaneous tissue disorders Dermatitis allergic	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Skin and subcutaneous tissue disorders Dermatitis contact	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Skin and subcutaneous tissue disorders Skin striae	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Vascular disorders Flushing	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Vascular disorders Hot flush	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)
Vascular disorders Vein collapse	0.83% 1/121 • Number of events 1 • From Baseline up to 30 days after last dose of the study drug (Up to Month 13)

Additional Information

Sr. Director of Clinical Operations

Emalex Biosciences, Inc.

Phone: 1847

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee PI must consult their clinical trial agreement. In summary, PI shall have the right to publish, present or otherwise use the results for their research publication objectives, provided that such Publication does not disclose Confidential Information. PI shall submit in writing to Sponsor any material at least 90 days for review and comment. Sponsor shall advise PI of any information which is Confidential Information or which may impair Sponsor's ability to obtain patent protection.
Publication restrictions are in place

Restriction type: OTHER