Ecopipam Treatment of Tourette's Syndrome in Subjects 7-17 Years

NCT02102698 | Completed Phase 2 DMC

• 1 other identifier: PSY302
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 11

Brief Summary

Tourette's Syndrome is a neurological disease characterized by motor and vocal tics. It has been hypothesized that abnormal interactions of dopamine with its receptors may cause the tics. The purpose of this study is to test the hypothesis that a drug (ecopipam) that selectively blocks dopamine D1/D5 receptors can reduce the frequency and severity of the tics.

Conditions

Tourette's Syndrome

Keywords

Tourette's Syndrome; Ecopipam; D1 Receptor; Phase 2

Outcome Measures

Primary Outcomes (1)

• Yale Global Tic Severity Scale

  The Yale Global Tic Severity Scale (YGTSS) is the standard rating scale used to assess the effects of a new treatment on the symptoms of Tourette's Syndrome.

  30 days

Secondary Outcomes (6)

• Adverse events (SAE), clinical laboratory values, regular measurement of vital signs, electrocardiograms, C-SSRS, Children's Depression Inventory and Physical Exams.

  30 days

• DuPaul ADHD rating scale-IV

  30 days

• Child Yale-Brown Obsessive Compulsive Scale

  30 days

• Children's Depression Inventory

  30 days

• Clinical Global Impression - Improvement and Severity Scales

  30 days

Study Arms (2)

Ecopipam

EXPERIMENTAL

Ecopipam is a selective antagonist of the dopamine D1/D5 receptor family that is being studied as a treatment for Tourette's Syndrome

Drug: Ecopipam

Placebo

PLACEBO COMPARATOR

Placebo is the inactive comparator

Drug: Ecopipam

Interventions

Ecopipam DRUG

Ecopipam is a selective antagonist of the dopamine D1 receptor family.

Also known as: SCH 39166, PSYRX101

Ecopipam; Placebo

Eligibility Criteria

• Age: 7 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subjects must exhibit both motor and vocal tics.
• Subjects must have a minimum score of 20 at both Screening and Baseline (just prior to the first treatment) on the Yale Global Tic Severity Scale.
• Subjects must be age (≥ 7 to \< 18 years of age)
• Subjects must weigh ≥ 20 kg (45 lbs)
• Adolescent females of childbearing potential who are sexually active must be using effective contraception (i.e., oral contraceptives, intrauterine device, double barrier method of condom and spermicide) and agree to continue use of contraception for the duration of their participation in the study. They must also agree to use contraception for 30 days after their last dose of study drug.
• Sexually active male subjects must use a barrier method of contraception during the study and agree to continue the use of male contraception for at least 30 days after the last dose of study drug.
• Subject's parent or legal guardian must execute a written informed consent.
• Subject must execute a written informed assent.

You may not qualify if:

• Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests, or ECG at Screening.
• Subjects with a major depressive episode in the past 2 years
• Subjects with a history of attempted suicide
• Subjects with clinically significant suicidality (based on the Columbia Suicide Rating Scale (C-SSRS)
• Subjects with a first-degree relative with a major depressive episode that resulted in any psychiatric hospitalization, or attempted/ completed suicide with the exception of a hospitalization for post-partum depression.
• Subjects with a history of seizures (excluding febrile seizures that occurred \>2 years in the past)
• Subjects with a myocardial infarction within 6 months.
• Girls who are currently pregnant or lactating.
• Subjects who have a need for medication (other than ecopipam) with possible effects on TS symptoms (i.e., lithium, psychostimulants)
• Subjects who have a need for medications which would have unfavorable interactions with ecopipam, e.g., dopamine antagonists or agonists \[including bupropion\], tetrabenazine, or monoamine oxidase inhibitors.
• Subjects with a lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any psychotic disorder determined by the Structured Clinical Interview for Diagnostic and Statistical Manual of Mental Disorders - 4th Edition (DSM-IV) Axis-I Disorders (SCID).
• Subjects with current or recent (past 3 months) DSM-IV substance abuse or dependence (with the exception of nicotine).
• Subjects with positive urine drug screen (cocaine, amphetamine, methamphetamine, tetrahydrocannabinol (THC), benzodiazepines, barbiturates, phencyclidine (PCP), opiates) at Screening. Subjects with urine positive only for benzodiazepines and/or marijuana (i.e., a user but not an abuser as based on DSM-IV criteria) may be eligible.
• Subjects who have had previous treatment with ecopipam.
• Subjects who have had treatment with:

Sponsors & Collaborators

• Emalex Biosciences Inc.: lead
• Psyadon Pharma: collaborator

Study Sites (11)

UCLA

Los Angeles, California, 90024, United States

Location

University of South Florida/Rothman Center for Neuropsychiatry

St. Petersburg, Florida, 33701, United States

Location

Emory University School Of Medicine

Atlanta, Georgia, 30322, United States

Location

University of Chicago Medical School

Chicago, Illinois, 60637, United States

Location

Children's Mercy Kansas City

Kansas City, Kansas, 64108, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Overlook Hospital

Summit, New Jersey, 07902, United States

Location

North Shore-Long Island Jewish Hosptial

Manhasset, New York, 11030, United States

Location

Weill Cornell Medical School

New York, New York, 10065, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45228, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Publications (2)

• Gilbert DL, Budman CL, Singer HS, Kurlan R, Chipkin RE. A D1 receptor antagonist, ecopipam, for treatment of tics in Tourette syndrome. Clin Neuropharmacol. 2014 Jan-Feb;37(1):26-30. doi: 10.1097/WNF.0000000000000017.

  PMID: 24434529 BACKGROUND

• Gilbert DL, Murphy TK, Jankovic J, Budman CL, Black KJ, Kurlan RM, Coffman KA, McCracken JT, Juncos J, Grant JE, Chipkin RE. Ecopipam, a D1 receptor antagonist, for treatment of tourette syndrome in children: A randomized, placebo-controlled crossover study. Mov Disord. 2018 Aug;33(8):1272-1280. doi: 10.1002/mds.27457. Epub 2018 Sep 7.

  PMID: 30192018 DERIVED

MeSH Terms

Conditions

Tourette Syndrome

Interventions

ecopipam

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Donald Gilbert, MD

  Children's Hospital Medical Center, Cincinnati

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2014
• First Posted: April 3, 2014
• Study Start: March 1, 2014
• Primary Completion: December 1, 2019
• Study Completion: December 1, 2019
• Last Updated: April 19, 2024

Record last verified: 2016-11

Locations

US (11)