| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Children and Adolescent Subjects with Tourette’s Syndrome greater than or equal to 6 and less than or equal to 18 years of age |
|
| E.1.1.1 | Medical condition in easily understood language |
| Tourette's Syndrome is a neurological disorder characterized by motor or vocal tics that begin in childhood and persists over time. |
|
| E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
| MedDRA Classification |
| E.1.3 | Condition being studied is a rare disease | No |
| E.2 Objective of the trial |
| E.2.1 | Main objective of the trial |
| The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in pediatric subjects (aged ≥6 to ≤18 years at Baseline) with Tourette’s Syndrome (TS) that were previously enrolled in the EBS-101-CL-001 study (Phase 2b) and completed the Phase 2b study without a major protocol deviation. |
|
| E.2.2 | Secondary objectives of the trial |
| The secondary objective is to evaluate the durability of effect of ecopipam in pediatric subjects (aged ≥6 to ≤18 years at Baseline) with TS. |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
1) 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) and must be who the
Investigator feels would benefit from continued participation. 2) Adolescent females of childbearing potential who are sexually active must be using highly
effective contraception (i.e., oral contraceptives, intrauterine device, intrauterine hormonereleasing
system, bilateral tubal occlusion, vasectomized male partner) and agree to continue use
of highly effective contraception for the duration of their participation in the study. They must also agree to use highly effective contraception for 30 days after their last dose of study drug. 3) Sexually active male subjects must use a highly effective method of contraception during the
study and agree to continue the use of highly effective contraception for at least 30 days after the
last dose of study drug. 4) Parents or legal guardians of subjects <18 years of age at Baseline must execute a written
informed consent. Subjects ≥18 years of age must execute a written informed consent.
5) Subjects <18 years of age at Baseline |
|
| E.4 | Principal exclusion criteria |
1) Subjects with a clinical presentation and/or history consistent with another neurologic condition
that may have had accompanying abnormal movements (e.g., Huntington’s disease, Parkinson’s
disease, Wilson’s disease, stroke, Restless Legs Syndrome). 2) History of schizophrenia, bipolar disorder, or other psychotic disorders.
3) Any unstable primary mood disorder (DSM-5 criteria) at time of Baseline.
4) Subjects who have unstable medical illness or clinically significant abnormalities on laboratory
tests or ECG at Baseline as determined by the Principal Investigator.
5) Subjects who have moderate to severe renal insufficiency at Screening.
6) Subjects with a major depressive episode in the past 2 years.
7) Subjects with a history of attempted suicide.
8) A significant risk of committing suicide based on history, routine psychiatric status examination,
Investigator’s judgment, or who had an answer of ‘‘yes’’ on any question other than 1–3 (currently or within the past 30 days) on the baseline/screening version of the C-SSRS.
9) Subjects with a history of seizures (excluding febrile seizures that occurred <2 years prior to
Baseline).
10) Subjects with a myocardial infarction within 6 months.
11) Female subjects who are currently pregnant or lactating or planning to get pregnant during the course of the study.
12) Subjects who have a need for medications which would have unfavorable interactions with ecopipam, e.g., dopamine antagonists or agonists [including bupropion], tetrabenazine, monoamine oxidase inhibitors, or St. John’s Wort.
13) Subjects with current or recent (past 3 months) DSM-5 substance use disorder (with the exception of nicotine).
14) Subjects with positive urine drug screen for cocaine, amphetamine, methamphetamine, benzodiazepines, barbiturates, phencyclidine (PCP) or opiates at Baseline, except those receiving stable, prescribed treatment for attention deficit/hyperactivity disorder. Subjects with urine positive only for benzodiazepines will be exclusionary unless medically prescribed.
15) Subjects with a lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any other psychotic disorder.
16) Inability to swallow tablets.
17) Subjects with a known hypersensitivity to ecopipam or any of its excipients.
18) Any subject who in the opinion of the investigator is not a suitable candidate for the study. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
| Safety will be monitored throughout the study at all visits by repeated adverse event (AE) monitoring, vital signs, the Columbia-Suicide Severity Rating Scale (C-SSRS), the Abnormal Involuntary Movement Scale (AIMS), the Barnes Akathisia Rating Scale (BARS), the Pediatric Anxiety Rating Scale (PARS) and Children's Depression Rating Scale-Revised (CDRS-R). In addition to these assessments, at Baseline, Months 1, 3, 6, 9 and 12 safety will also be monitored by clinical and laboratory evaluations and electrocardiogram (ECG) monitoring. At the Follow Up visit, AE monitoring, the C-SSRS, clinical and laboratory evaluations and vital signs will be assessed. Laboratory evaluations will include hematology, chemistry, urine values and HbA1c. A Follow Up phone call will be conducted 30 days after the last dose of study medication to assess AEs. Additional assessments (all visits except the follow up visit) will include the AIMS, the BARS, the CDRS-R and the PARS. C-SSRS will also be assessed (all visits including the Follow Up visit). |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
| Adverse event (AE) monitoring, vital signs, the Columbia-Suicide Severity Rating Scale (C-SSRS), the Abnormal Involuntary Movement Scale (AIMS), the Barnes Akathisia Rating Scale (BARS), the Pediatric Anxiety Rating Scale (PARS) and Children's Depression Rating Scale-Revised (CDRS-R) will be followed from Baseline to Month 12. |
|
| E.5.2 | Secondary end point(s) |
Efficacy Assessments: The YGTSS, CGI-TS-S, and C&A-GTS-QOL will be assessed at Baseline, Months 1, 3, 6, 9 and 12. The CGI-TS-I will be assessed at Months 1, 3, 6, 9 and 12.
|
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
| Efficacy Assessments: The YGTSS, CGI-TS-S, and C&A-GTS-QOL will be assessed at Baseline, Months 1, 3, 6, 9 and 12. The CGI-TS-I will be assessed at Months 1, 3, 6, 9 and 12. |
|
| E.6 and E.7 Scope of the trial |
| E.6 | Scope of the trial |
| E.6.1 | Diagnosis | Yes |
| E.6.2 | Prophylaxis | No |
| E.6.3 | Therapy | Yes |
| E.6.4 | Safety | Yes |
| E.6.5 | Efficacy | Yes |
| E.6.6 | Pharmacokinetic | No |
| E.6.7 | Pharmacodynamic | No |
| E.6.8 | Bioequivalence | No |
| E.6.9 | Dose response | Yes |
| E.6.10 | Pharmacogenetic | No |
| E.6.11 | Pharmacogenomic | No |
| E.6.12 | Pharmacoeconomic | No |
| E.6.13 | Others | No |
| E.7 | Trial type and phase |
| E.7.1 | Human pharmacology (Phase I) | No |
| E.7.1.1 | First administration to humans | No |
| E.7.1.2 | Bioequivalence study | No |
| E.7.1.3 | Other | No |
| E.7.1.3.1 | Other trial type description | |
| E.7.2 | Therapeutic exploratory (Phase II) | Yes |
| E.7.3 | Therapeutic confirmatory (Phase III) | No |
| E.7.4 | Therapeutic use (Phase IV) | No |
| E.8 Design of the trial |
| E.8.1 | Controlled | No |
| E.8.1.1 | Randomised | No |
| E.8.1.2 | Open | Yes |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | No |
| E.8.1.5 | Parallel group | No |
| E.8.1.6 | Cross over | No |
| E.8.1.7 | Other | No |
| E.8.2 | Comparator of controlled trial |
| E.8.2.1 | Other medicinal product(s) | No |
| E.8.2.2 | Placebo | No |
| E.8.2.3 | Other | No |
| E.8.2.4 | Number of treatment arms in the trial | 1 |
| E.8.3 |
The trial involves single site in the Member State concerned
| No |
| E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
| E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
| E.8.5 | The trial involves multiple Member States | Yes |
| E.8.5.1 | Number of sites anticipated in the EEA | 13 |
| E.8.6 Trial involving sites outside the EEA |
| E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
| E.8.6.2 | Trial being conducted completely outside of the EEA | No |
| E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
| Canada |
| France |
| Germany |
| Poland |
| United States |
|
| E.8.7 | Trial has a data monitoring committee | No |
| E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 2 |
| E.8.9.1 | In the Member State concerned months | 0 |
| E.8.9.1 | In the Member State concerned days | 0 |
| E.8.9.2 | In all countries concerned by the trial years | 2 |
| E.8.9.2 | In all countries concerned by the trial months | 0 |
| E.8.9.2 | In all countries concerned by the trial days | 0 |
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