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

Open Label Safety and Tolerability Trial of CPP-109 (Vigabatrin) in Adults With Treatment Refractory Tourette's Disorder

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.

Study Overview

• Status: Completed
• Conditions: Tourette's Disorder
• Intervention / Treatment: Drug: 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.

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10029
      • Tics and Tourette's Clinical and Research Program

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 35 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Vigabatrin; 3 tablets, bid for 8 weeks	Drug: vigabatrin; 3 tablets, bid for 8 weeks; Other Names: CPP-109 Vigabatrin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Global Severity Score on the Y-GTSS	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.	weekly from baseline to end of study (10weeks)

Collaborators and Investigators

• Sponsor: Barbara J. Coffey
• Investigators: Principal Investigator: Jonathan D Brodie, PhD,MD, NYU School of Medicine

Study record dates

Study Major Dates

• Study Start: July 1, 2012
• Primary Completion (Actual): March 1, 2015
• Study Completion (Actual): March 1, 2015

Study Registration Dates

• First Submitted: April 24, 2012
• First Submitted That Met QC Criteria: April 24, 2012
• First Posted (Estimate): April 25, 2012

Study Record Updates

• Last Update Posted (Actual): January 19, 2018
• Last Update Submitted That Met QC Criteria: January 17, 2018
• Last Verified: January 1, 2018

More Information

Terms related to this study

• Keywords: Tourette's Disorder; vigabatrin
• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Neurodevelopmental Disorders; Tic Disorders; Disease; Tourette Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; GABA Agents; Anticonvulsants; Vigabatrin
• Other Study ID Numbers: GCO 12-0964; 11-01864