Ecopipam Tablets to Study Tourette’s Disorder in Children, Adolescents and Adults (D1AMOND)
- Conditions
- Tourette’s DisorderMedDRA version: 20.0Level: PTClassification code: 10044126Term: Tourette's disorder Class: 100000004850Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2023-503494-38-00
- Lead Sponsor
- Emalex Biosciences Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 239
Subjects must be = 6 years of age at time of screening; Subjects must weigh at least =18 kg (39.6 lbs.); Subjects must have TD based on the Diagnostic and Statistical Manual for Mental Disorders – 5th Edition-Text Revision (DSM-5-TR) diagnostic criteria for TD; Subjects must exhibit both motor and vocal tics that cause impairment with normal routines and have TD as their primary disorder; Subjects must have a minimum score of 20 on the YGTSS-TTS at Screening and at Baseline visits with tic symptoms causing impairment in the subject’s normal routines, including academic achievement, and/or occupational functioning, and/or social activities, and/or relationships or whose symptoms distress the subject to the extent that treatment is appropriate; Female participants of childbearing potential must agree to use a highly effective method of contraception (i.e., pregnancy rate of less than 1% per year) during the study and for 30 days after the discontinuation of the IMP. Adequate contraceptive methods include combined hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception with inhibition of ovulation (oral, injectable, implantable), intrauterine devices (IUDs), intrauterine hormone-releasing system (IUS), true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant), bilateral tubal occlusion, vasectomized male partner or a female participant who is not of childbearing potential. Female participants and female partners of male study participants using a hormonal contraceptive must also use a barrier method (i.e., condom or occlusive cap [diaphragm or cervical/vault caps]) and should have been stable on their hormonal contraceptive treatment for at least 4 weeks prior to Screening. For sites in the EU only, the subject must have received an adequate trial of non-pharmacological therapy without adequate response prior to study enrollment.
Subjects with any unstable mood disorder (DSM-5-TR criteria) at Screening or Baseline; Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests, or ECG at Screening as determined by the Principal Investigator; Subjects with a significant risk of attempting 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); Subjects with a clinical presentation at Screening or Baseline 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); Subjects who have moderate to severe renal impairment; Subjects who have hepatic impairment at Screening;, Subjects with a =25% difference in the absolute change in YGTSS-TTS between the Screening visit and the Baseline visit; Subjects with an onset of a major depressive episode in the past 6 months; Subjects who have had previous treatment with investigational medication within 4 weeks prior to Screening, oral neuroleptics within 4 weeks prior to Screening, and/or depot neuroleptics within 3 months prior to Screening (e.g., risperidone microspheres) or 6 months prior to screening (e.g., paliperidone palmitate); Subjects receiving any other medication to treat motor or vocal tics for at least 14 days prior to Baseline; Subjects receiving unstable doses or excluded medications to treat depression, anxiety or ADHD during the 4 weeks prior to Screening; ; Subjects with known hypersensitivity to any excipients of ecopipam tablets, including subjects with confirmed lactose intolerance; Subjects who have initiated new psychological therapies or deep brain stimulation within 10 weeks prior to Baseline visit.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this study is to evaluate the maintenance of efficacy of ecopipam tablets in children (= 6 and < 12 years of age), adolescents (= 12 and < 18 years of age) with Tourette’s Disorder (TD).;Secondary Objective: The secondary objectives of this study are to evaluate the safety and tolerability of ecopipam dosed at 1.8 mg/kg/day (2 mg/kg/day ecopipam HCl) in children (= 6 to <12 years of age), adolescents (= 12 and < 18 years of age), and adults (= 18 years of age), to evaluate the maintenance of efficacy of ecopipam in adults (? 18 years of age), with TD and to characterize the pharmacokinetics (PK) of ecopipam in this patient population.;Primary end point(s): Time from Randomization (Week 12) to Relapse in subjects = 6 and < 18 years for ecopipam compared to placebo during the double-blind, R/WD period.
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Time from Randomization (Week 12) to Relapse in all subjects irrespective of age for ecopipam compared to placebo during the double-blind R/WD period.