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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01585207
Recruitment Status : Completed
First Posted : April 25, 2012
Results First Posted : January 19, 2018
Last Update Posted : January 19, 2018
Information provided by (Responsible Party):
Barbara J. Coffey, Icahn School of Medicine at Mount Sinai

Brief Summary:
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 or disease Intervention/treatment Phase
Tourette's Disorder Drug: vigabatrin Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : July 2012
Actual Primary Completion Date : March 2015
Actual Study Completion Date : March 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Vigabatrin

Arm Intervention/treatment
Experimental: Vigabatrin
3 tablets, bid for 8 weeks
Drug: vigabatrin
3 tablets, bid for 8 weeks
Other Name: CPP-109 Vigabatrin

Primary Outcome Measures :
  1. Global Severity Score on the Y-GTSS [ Time Frame: weekly from baseline to end of study (10weeks) ]
    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. Scale from 0- 100. Higher score indicates more impairment.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 35 Years   (Adult)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01585207

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

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Responsible Party: Barbara J. Coffey, Professor, Icahn School of Medicine at Mount Sinai Identifier: NCT01585207    
Other Study ID Numbers: GCO 12-0964
First Posted: April 25, 2012    Key Record Dates
Results First Posted: January 19, 2018
Last Update Posted: January 19, 2018
Last Verified: January 2018
Keywords provided by Barbara J. Coffey, Icahn School of Medicine at Mount Sinai:
Tourette's Disorder
Additional relevant MeSH terms:
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Tourette Syndrome
Pathologic Processes
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
Neurodevelopmental Disorders
Mental Disorders
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs