Proof-of-Concept Safety Study of CPP-109 (Vigabatrin) for Treatment Refractory Tourette's Disorder

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2014 by Mount Sinai School of Medicine
Information provided by (Responsible Party):
Barbara J. Coffey, Mount Sinai School of Medicine Identifier:
First received: April 24, 2012
Last updated: October 20, 2014
Last verified: October 2014

The purpose of this study is to determine if vigabatrin, an unusual anti-seizure medication, will diminish the Tourette Disorder outbursts in young adults whose symptoms have persisted into adulthood and have not responded to usual treatment.

Condition Intervention Phase
Tourette's Disorder
Drug: vigabatrin
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open Label Safety and Tolerability Trial of CPP-109 (Vigabatrin) in Adults With Treatment Refractory Tourette's Disorder

Resource links provided by NLM:

Further study details as provided by Mount Sinai School of Medicine:

Primary Outcome Measures:
  • Global Severity Score on the Y-GTSS [ Time Frame: weekly from baseline to end of study (8weeks) ] [ Designated as safety issue: No ]
    The Global Severity score is the sum of the Total Tic score and the TD Impairment score. It is rated by the Investigator on the Yale Global Tic Severity Score ( Y-GTSS, a widely accepted measure of drug efficacy in TD

Estimated Enrollment: 10
Study Start Date: July 2012
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Vigabatrin
3 tablets, bid for 8 weeks
Drug: vigabatrin
3 tablets, bid for 8 weeks
Other Name: CPP-109 Vigabatrin

Detailed Description:

The aims of this study are to 1) explore proof of concept that CPP-109 will reduce tics, and 2) to obtain systematic data regarding dosing, safety and tolerability of CPP-109 in adults with treatment refractory TD. We will obtain preliminary data on estimate of effect size for tics using Cohen's d, calculating the difference between the two means (baseline and endpoint scores on the YGTSS), divided by the standard deviation of the difference.


Ages Eligible for Study:   18 Years to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Subjects must be between 18 and 35 years of age (inclusive) when informed consent is obtained.
  2. Subjects must meet full DSM-IV diagnostic criteria for TD by clinical interview on examination by a physician investigator, and confirmed by the Structured Clinical Interview for DSM (SCID-CT) for clinical trials.
  3. Subjects will have failed to respond to an adequate trial, as determined by the investigator, of clonidine, guanfacine, and a first generation (typical) and second-generation (atypical) neuroleptic medication in the past.
  4. Tics are causing significant distress or impairment, as determined by the subject and principal investigator, on the current treatment regimen.
  5. Laboratory results, including serum chemistries, hematology, and urinalysis, must show no significant abnormalities (significant is defined as laboratory values requiring acute medical intervention).
  6. Subjects will not undergo formal IQ testing, but must be of normal intelligence in the judgment of the investigator.
  7. Subjects must possess an educational level, degree of understanding and command of the English language to enable them to communicate suitably with the investigators and study coordinator, and to understand the nature of the study.
  8. Subjects must be considered reliable.
  9. Written informed consent of subjects is obtained.

Exclusion Criteria:

  1. Subjects with organic brain disease, for example, traumatic brain injury residua.
  2. Subjects with a preexisting ophthalmologic condition.
  3. Subjects with or at high risk of other types of irreversible vision loss or who require other drugs associated with serious adverse ophthalmic effects such as retinopathy or glaucoma.
  4. Subjects meeting criteria for mental retardation as defined by the DSM-IV-TR.
  5. Subjects with a history of seizure disorder (other than febrile seizure).
  6. Subjects with history of Sydenham's Chorea.
  7. Subjects with autism, schizophrenia, other psychotic disorder, or bipolar disorder.
  8. Subjects with a primary diagnosis of a major mood disorder that requires ongoing psychiatric treatment.
  9. Subjects with a neurological disorder other than a tic disorder.
  10. Subjects with a major medical illness.
  11. Female subjects who are unwilling to use birth control or who are pregnant, as determined by serum pregnancy test at baseline assessment, or lactating.
  12. Subjects who have a past or current history of substance dependence and/or a current history of substance abuse or who fail baseline toxicology screen.
  13. Subjects who have any clinically significant abnormal laboratory result at baseline screening including EKG, or blood tests.
  14. Subjects who, in the opinion of the investigator, are unsuitable in any other way to participate in this study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01585207

Contact: Jonathan Brodie, MD (212) 263-6365

United States, New York
Tics and Tourette's Clinical and Research Program Recruiting
New York, New York, United States, 10029
Principal Investigator: Barbara Coffey, MD         
Sponsors and Collaborators
Barbara J. Coffey
Principal Investigator: Jonathan D Brodie, PhD,MD NYU School of Medicine
  More Information

No publications provided

Responsible Party: Barbara J. Coffey, Professor, Mount Sinai School of Medicine Identifier: NCT01585207     History of Changes
Other Study ID Numbers: GCO 12-0964, 11-01864
Study First Received: April 24, 2012
Last Updated: October 20, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Mount Sinai School of Medicine:
Tourette's Disorder

Additional relevant MeSH terms:
Tourette Syndrome
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Mental Disorders
Mental Disorders Diagnosed in Childhood
Movement Disorders
Nervous System Diseases
Neurodegenerative Diseases
Tic Disorders
Central Nervous System Agents
Enzyme Inhibitors
GABA Agents
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on March 26, 2015