Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents (D1AMOND)

Multicenter, Placebo-Controlled, Double-Blind, Randomized, Parallel-Group, Phase 2b Study to Evaluate the Efficacy and Safety of Ecopipam in Children and Adolescents With Tourette's Syndrome

This study evaluates the effect of ecopipam tablets in children and adolescents in the treatment of Tourette's Syndrome (TS). Half of the participants will receive ecopipam tablets, while the other half will receive matching placebo tablets

Study Overview

• Status: Completed
• Conditions: Tourette Syndrome
• Intervention / Treatment: Drug: Placebo; Drug: Ecopipam

Detailed Description

Multicenter, placebo-controlled, double-blind, randomized, parallel-group, Phase 2b study in pediatric subjects (aged greater than equal to 6 to less than 18 years of age) with TS. Following a 28-day Screening period and Baseline visit, eligible subjects will be randomized 1:1 to receive either ecopipam hydrochloride (HCl) or matching placebo for a 12 week treatment period. Doses will be titrated up and down from target dose of 2 mg/kg/day. Follow Up visit will be conducted after the last dose of study medication.

• Study Type: Interventional
• Enrollment (Actual): 153
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Ajax, Ontario, Canada, L1Z 0M1
      • The Kids Clinic Inc
    • Ottawa, Ontario, Canada, K2G 1W2
      • Center for Pediatric Excellence
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • CHU Sainte-Justine
• France
  • Poitiers, France, 86021
    • CHU Poitiers
  • Ile De France
    • Paris, Ile De France, France, 75019
      • Hopital Robert Debre
• Germany
  • Freiburg, Germany, 79104
    • Dept. Child Adolescent Psychiatry Uni. Freiburg
  • Mannheim, Germany, 68159
    • Zentralinstitut fuer Seelische Gesundheit
  • Mittweida, Germany, 09648
    • Pharmakologisches Studienzentrum Chemnitz GmbH
  • Bayern
    • Muenchen, Bayern, Germany, 80336
      • Psychiatric Clinic of Ludwig Maximilians Universitaet Muenchen
  • Niedersachsen
    • Hannover, Niedersachsen, Germany, 30625
      • Department of Psychiatry, Socialpsychiatry and Psychotherapy, Hannover Medical School
• Poland
  • Gdansk, Poland, 80-952
    • Uniwersyteckie Centrum Kliniczne
  • Gdansk, Poland, 80-546
    • Centrum Bada Klinicznych PI-House Sp. z o.o.
  • Gdańsk, Poland, 80-542
    • Gdanskie Centrum Zdrowia Sp z o.o.
  • Katowice, Poland, 40-123
    • NZOZ Wielospecjalistyczna Poradnia Lekarska Synapsis
  • Krakow, Poland, 30-363
    • Centrum Medyczne PLEJADY
  • Krakow, Poland, 31-503
    • Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
  • Poznań, Poland, 60-693
    • Med-Polonia Sp. z o. o.
• United States
  • Alabama
    • Dothan, Alabama, United States, 36303
      • Harmonex Neuroscience Research
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Children's Hospital
  • California
    • Anaheim, California, United States, 92805
      • Advanced Research Center Inc.
    • Los Angeles, California, United States, 90095
      • UCLA
    • San Diego, California, United States, 92108
      • PCSD-Feighner Research
    • Santa Ana, California, United States, 92705
      • Syrentis Clinical Research
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale School of Medicine
  • Florida
    • Gainesville, Florida, United States, 32607
      • Sarkis Clinical Trials
    • Gulf Breeze, Florida, United States, 32561
      • Northwest Florida Clinical Research Group, LLC
    • Hialeah, Florida, United States, 33013
      • Research in Miami Inc.
    • Miami, Florida, United States, 33136
      • University of Miami
    • North Miami, Florida, United States, 33180
      • MedBio Trials
    • Orlando, Florida, United States, 32803
      • APG Research LLC
    • Saint Petersburg, Florida, United States, 33701-4825
      • University of South Florida
    • Tampa, Florida, United States, 33609-4181
      • Pediatric Epilepsy and Neurology Specialists
    • Winter Park, Florida, United States, 32789
      • Pediatric Neurology, PA
  • Georgia
    • Atlanta, Georgia, United States, 30318
      • Rare Disease Research, LLC
    • Savannah, Georgia, United States, 31406
      • Meridian Clinical Research
  • Illinois
    • Chicago, Illinois, United States, 60612-3841
      • Rush University Medical Center
    • Chicago, Illinois, United States, 60637-1447
      • The University of Chicago Hospitals
    • Naperville, Illinois, United States, 60563-6510
      • AMR - Baber Research Inc.
  • Kansas
    • Overland Park, Kansas, United States, 66211
      • Psychiatric Associates
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Michigan Clinical Research Institute PC
    • Bloomfield Hills, Michigan, United States, 48302-1952
      • Neurobehavioral Medicine Group
    • Wyoming, Michigan, United States, 49418
      • Helen DeVos Children's Hospital / Spectrum Health Medical Group
  • Missouri
    • Saint Charles, Missouri, United States, 63304
      • St. Charles Psychiatric Associates dba Midwest Research Group
    • Saint Louis, Missouri, United States, 63110-1093
      • Movement Disorders Center
  • Nebraska
    • Lincoln, Nebraska, United States, 68526
      • Alivation Research, LLC
  • Nevada
    • Las Vegas, Nevada, United States, 89128
      • Center for Psychiatry and Behavioral Medicine Inc.
  • New Jersey
    • Mount Arlington, New Jersey, United States, 07856
      • The NeuroCognitive Institute
    • Voorhees, New Jersey, United States, 08043-1910
      • Clinical Research Center of NJ
  • New York
    • New York, New York, United States, 10003
      • New York Neurology Associates P.C
    • New York, New York, United States, 10019
      • Hapworth Research Inc.
    • New York, New York, United States, 10029-6504
      • Mount Sinai School of Medicine
    • New York, New York, United States, 10036
      • Mood Disorders Consulting Medicine PLLC
    • Rochester, New York, United States, 14618
      • Finger Lakes Clinical Research
  • Ohio
    • Avon Lake, Ohio, United States, 44012-1004
      • Quest Therapeutics of Avon Lake
    • Cincinnati, Ohio, United States, 45229-3026
      • Cincinnati Childrens Hospital Medical Center
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center
    • Middleburg Heights, Ohio, United States, 44130
      • North Star Medical Research LLC
  • Pennsylvania
    • Media, Pennsylvania, United States, 19063
      • Suburban Research Associates
  • South Carolina
    • Charleston, South Carolina, United States, 29414
      • Coastal Pediatric Research
  • Tennessee
    • Nashville, Tennessee, United States, 37203-6502
      • Access Clinical Trials, Inc.
    • Nashville, Tennessee, United States, 37232-0028
      • Vanderbilt University Medical Center
  • Texas
    • Bellaire, Texas, United States, 77401
      • Houston Clinical Trials LLC
    • Dallas, Texas, United States, 75243
      • Relaro Medical Trials
    • Fort Worth, Texas, United States, 76104
      • North Texas Clinical Trials
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine
    • San Antonio, Texas, United States, 78249-3539
      • Road Runner Research Ltd.
  • Utah
    • Springville, Utah, United States, 84663
      • Noetic Psychiatry
  • Virginia
    • Charlottesville, Virginia, United States, 22908-0829
      • University of Virginia
  • Washington
    • Everett, Washington, United States, 98201-4077
      • Eastside Therapeutic Resource Inc dba Core Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• ≥ 6 and < 18 years of age
• ≥ 18 kg (~ 40 lbs.)
• TS diagnosis and both motor and vocal tics that cause impairment with normal routines
• Minimum score of 20 on the YGTSS-Total Tic Score
• May not be taking any medications used to treat motor or vocal tics for at least 14 days prior to Baseline.
• Effective contraception during the study and 30 days after last study dose for sexually active subjects

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
• Hepatic insufficiency
• Positive urine drug screen
• Unstable doses for drugs to treat anxiety, depression, Attention Deficit Hyperactivity Disorder
• Certain medications that would lead to drug interactions
• Recent behavioral therapy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Matching Placebo tablets taken orally in the evening	Drug: Placebo; Matching Placebo
Experimental: Ecopipam HCI 2 mg/kg/day; Ecopipam HCl 12.5-, 50-, 75- and 100-mg tablets; 2 mg/kg/day target dose; oral administration daily in evenings	Drug: Ecopipam; Ecopipam HCI tablets administered PO to establish 2 mg/kg/day

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at Week 12	The YGTSS was a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0=none to 5=severe for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe) with higher score represent more severe symptoms. A negative change from baseline indicates improvement.	Baseline, Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) at Week 12	Clinical Global Impression (CGI) scale was used to assess overall severity on a 7-point Likert scale consisted of 2 reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms. The CGI severity scale ranges from 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill subject. 1 = "normal, not ill at all" to 7 = "extremely ill." A negative change indicates improvement in the condition.	Baseline, Week 12

Collaborators and Investigators

• Sponsor: Emalex Biosciences Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 28, 2019
• Primary Completion (Actual): September 23, 2021
• Study Completion (Actual): September 23, 2021

Study Registration Dates

• First Submitted: July 2, 2019
• First Submitted That Met QC Criteria: July 2, 2019
• First Posted (Actual): July 5, 2019

Study Record Updates

• Last Update Posted (Actual): October 4, 2023
• Last Update Submitted That Met QC Criteria: September 8, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Disease; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Neurodevelopmental Disorders; Tic Disorders; Syndrome; Tourette Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Antipsychotic Agents; Tranquilizing Agents; Psychotropic Drugs; Dopamine Agents; Dopamine Antagonists; Ecopipam
• Other Study ID Numbers: EBS-101-CL-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No