Children and Adolescent Subjects with Tourette’s Syndrome grater than or equal to 6 and less than 18 years of age.
Conditions
Sponsors
Emalex Biosciences, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion Criteria: 1) Subject’s parent or legal guardian must sign a written informed consent. 2) Subject must sign a written informed assent according to the requirements of the site’s IRB/EC. 3) Subjects must be =6 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Exclusion Criteria: 1) Subjects with any unstable primary mood disorder (DSM-5 criteria) at Screening. 2) Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests, or ECG at Screening as determined by the Principal Investigator. 3) Subjects with 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 Columbia Suicide Severity Rating Scale (C-SSRS). 4) Subjects with a clinical presentation at Screening 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). 5) Female subjects who are currently pregnant or lactating or planning to become pregnant during the course of the study. 6) Subjects who have moderate to severe renal insufficiency at Screening. 7) Subjects who have hepatic impairment at Screening 8) Subjects with current or recent (past 3 months) history of DSM-5 substance use disorder (with the exception of nicotine). 9) Subjects with positive urine drug screen for cocaine, amphetamine, methamphetamine, benzodiazepines, barbiturates, phencyclidine (PCP), or opiates at Screening, except those receiving stable, prescribed treatment for attention deficit/hyperactivity disorder (ADHD) 10) Subjects with a > 25% difference in the absolute change in YGTSS-TTS score between the Screening visit and the Baseline visit 11) Subjects with a lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any other psychotic disorder. 12) Subjects with a major depressive episode in the past 2 years. 13) Subjects with a history of attempted suicide. 14) Subjects with a history of seizures (excluding febrile seizures that occurred >2 years prior to Screening). 15) Subjects with a history of neuroleptic malignant syndrome. 16) Subjects with a myocardial infarction within 6 months. 17) Subjects who have had previous treatment with ecopipam. 18) Subjects who have had previous treatment with: o investigational medication within 1 month prior to Screening o depot neuroleptics within 3 months prior to Screening o oral neuroleptics within 4 weeks prior to Screening 19) Subjects receiving anti-depressant, anti-anxiety or anti-ADHD medications unless the dosage has been stable for a minimum of 2 weeks prior to Screening and not prescribed to relieve the neurological symptoms related to TS. 20) 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. 21) Initiation or changes in behavioral therapies for TS during the course of the study (i.e., Habit Reversal Training or Comprehensive Behavioral Intervention for Tics) as well as deep brain stimulation. 22) Subjects who have initiated new behavioral therapies to treat TS fewer than 10 weeks prior to Baseline visit 23) Subjects unable to swallow tablets 24) Subjects with a known hypersensitivity to ecopipam or any of its excipients.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary: The primary objective of this study is to evaluate the efficacy of ecopipam tablets in pediatric subjects (aged =6 to <18 years) with Tourette’s Syndrome (TS).;Secondary Objective: Secondary: The secondary objectives of this study are to evaluate the safety of ecopipam tablets in pediatric subjects (aged =6 to <18 years) with TS and characterize the pharmacokinetics (PK) of ecopipam.;Primary end point(s): Efficacy: Primary Efficacy Endpoint: The primary efficacy endpoint is the change in the Yale Global Tic Severity Scale – Total Tic Score (YGTSS-TTS, i.e., sum of the motor and phonic tic scores) from baseline (YGTSS-TTS score from Baseline visit) to end of therapy (YGTSS-TTS score from Week 12).;Timepoint(s) of evaluation of this end point: From baseline (YGTSS-TTS score from Baseline visit) to end of therapy (YGTSS-TTS score from Week 12). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Key Secondary Efficacy Endpoint: • Change in Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) from Baseline to Week 12 Other Secondary efficacy endpoints include: • Change in Clinician Global Impression Tourette Syndrome of Improvement (CGI-TS-I) from Baseline to Week 12 • Change in YGTSS-Global Score (GS) from Baseline to Week 12 • Change in Caregiver Global Impression of Change (CaGI-C) from Baseline to Week 12 • Change in Gilles de la Tourette Syndrome–Quality of Life Scale for Children and Adolescents (C&A-GTS-QOL) from Baseline to Week12 • Percentage of subjects with a 25% improvement on the YGTSS-TTS • Percentage of subjects with complete remission of tics on the YGTSS-TTS • Change in YGTSS-TTS from Baseline to Weeks 4, 6, and 8 • Change in CGI-TS-S from Baseline to Weeks 4, 6, and 8 • Change in CGI-TS-I from Baseline to Weeks 4, 6, and 8 • Change in YGTSS-GS from Baseline to Weeks 4, 6, and 8 • Change in CaGI-C from Baseline to Weeks 4, 6, and 8 • Change in the C&A-GTS-QOL from Baseline to Weeks 4, 6, and 8 Rank order of hierarchy of the secondary endpoints will be outlined in the statistical analysis plan.;Timepoint(s) of evaluation of this end point: from Baseline to Week 12 | — |
Countries
Canada, France, Germany, Hungary, Italy, Poland, United States
Contacts
Public ContactClinical contact
Emalex Biosciences, Inc.
Outcome results
None listed