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

Tourette's Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01585207
• Other study ID #: GCO 12-0964
• Secondary ID: 11-01864
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: April 24, 2012
• Last updated: January 17, 2018
• Start date: July 2012
• Est. completion date: March 2015

Study information

• Verified date: January 2018
• Source: Icahn School of Medicine at Mount Sinai
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: March 2015
• Est. primary completion date: March 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 35 Years

Eligibility

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.

Related Conditions & MeSH terms

• Disease
• Tourette Syndrome
• Tourette's Disorder

Intervention

Drug:
• vigabatrin
3 tablets, bid for 8 weeks

Locations

Country	Name	City	State
United States	Tics and Tourette's Clinical and Research Program	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Barbara J. Coffey

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)