Phase 2
N=124
Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension
Tourette Syndrome in Children · Tourette Syndrome in Adolescence
Bottom Line
View on ClinicalTrials.gov: NCT04114539 ↗Enrolled (actual)
124
Serious AEs
1.7%
Results posted
Dec 2023
Primary outcome: Primary: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) — 84; 2; 44; 27 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ecopipam (Drug)
- Age
- Pediatric, Adult · 6+ yrs
- Sex
- All
- Sponsor
- Emalex Biosciences Inc.
- Primary completion
- Nov 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) and Their Relationship (Unrelated, Possibly Related, or Probably Related) |
84; 2; 44; 27; 13; 1 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Basophils to Leukocytes Ratio Reported in Percentage of Cells |
-0.1 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Eosinophils to Leukocytes Ratio Reported in Percentage of Cells |
0.1 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Erythrocytes |
0.04 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Hematocrit |
0.01 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Hemoglobin |
0.08 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Leukocytes and Platelets |
0.15; 4.1 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Lymphocytes to Leukocytes Ratio Reported in Percentage of Cells |
0.2 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Monocytes to Leukocytes Ratio Reported in Percentage of Cells |
0.2 | — |
| PRIMARY Change From Baseline in Hematology Parameters: Neutrophils to Leukocytes Ratio Reported in Percentage of Cells |
-0.3 | — |
| PRIMARY Change From Baseline in Serum Chemistry Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactase Dehydrogenase |
2.3; -4.7; -0.3; -6.2 | — |
| PRIMARY Change From Baseline in Serum Chemistry Parameters: Albumin, Globulin and Protein |
0.6; 1.2; 1.9 | — |
| PRIMARY Change From Baseline in Serum Chemistry Parameters: Bicarbonate, Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglyceride and Urea Nitrogen |
-0.6; 0.02; -0.2; 0.20; -0.06; -0.02 | — |
| PRIMARY Change From Baseline in Serum Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin |
-0.6; 6.2; -0.1 | — |
| PRIMARY Change From Baseline in Hemoglobin A1c (HbA1c) |
0.03 | — |
| PRIMARY Change From Baseline in Vital Signs Parameter: Diastolic Blood Pressure and Systolic Blood Pressure |
0.9; 0.6; 1.6; 0.3; 1.8; -1.1 | — |
| PRIMARY Change From Baseline in Vital Signs Parameter: Pulse Rate |
0.4; 0.6; 1.8 | — |
| PRIMARY Change From Baseline in Vital Signs Parameter: Body Mass Index [BMI] |
1.0 | — |
| PRIMARY Change From Baseline in Vital Signs Parameter: Height |
4.0 | — |
| PRIMARY Change From Baseline in Vital Signs Parameter: Weight |
5.4 | — |
| PRIMARY Change From Baseline in Electrocardiogram (ECG) Values Parameters: Aggregate PR Interval, Aggregate QRS Duration, Aggregate QT Interval, and Aggregate QTc Interval |
-5.6; -0.6; 5.5; 0.2 | — |
| PRIMARY Number of Participants With Clinically Significant Abnormal Physical Examination Findings |
1; 1 | — |
| SECONDARY Change From Baseline in the Yale Global Tic Severity Scale -Total Tic Score (YGTSS-TTS) at Months 1, 3, 6, 9 and 12 |
-6.5; -7.5; -10.6; -11.5; -11.7 | — |
| SECONDARY Change From Baseline in the Yale Global Tic Severity Scale - Impairment (YGTSS-I) at Months 1, 3, 6, 9 and 12 |
-7.2; -8.3; -10.4; -12.5; -12.1 | — |
| SECONDARY Change From Baseline in the Yale Global Tic Severity Scale - Global Score (YGTSS-GS) at Months 1, 3, 6, 9 and 12 |
-13.8; -15.9; -21.0; -24.0; -23.8 | — |
| SECONDARY Change From Baseline in Clinical Global Impression of Tourette Syndrome of Severity (CGI-TS-S) at Months 1, 3, 6, 9, 12 |
-0.8; -0.8; -1.0; -1.2; -1.2 | — |
| SECONDARY Clinical Global Impression Tourette Syndrome of Improvement (CGI-TS-I) Scores at Months 1, 3, 6, 9 and 12 |
2.7; 2.5; 2.2; 2.0; 2.0 | — |
| SECONDARY Change From Baseline in Gilles de la Tourette Syndrome-Quality of Life Scale for Children and Adolescents (C&A-GTS-QOL) Total Score at Months 1, 3, 6, 9 and 12 |
-4.0; -4.5; -7.7; -6.7; -8.0 | — |
Summary
This study was an international, multicenter, open-label, long term extension study evaluating the safety of ecopipam tablets for the treatment of children and adolescent subjects with Tourette Syndrome.
Eligibility Criteria
Inclusion Criteria
- Subjects must have completed the EBS-101-CL-001 study through the Day 14 Follow Up Visit within the last 30 days (or longer with permission of the medical monitor) without a major reportable protocol deviation and must be someone the Investigator feels would benefit from continued participation.
Exclusion Criteria
- Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder).
- Unstable medical illness or clinically significant lab abnormalities.
- Risk of suicide.
- Pregnant or lactating women.
- Moderate to severe renal insufficiency.
- Positive urine drug screen.
- Certain medications that would lead to drug interactions.
Data sourced from ClinicalTrials.gov (NCT04114539). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.