Clinical trial NCT01585207: Proof-of-Concept Safety Study of CPP-109 (Vigabatrin) for Treatment Refractory Tourette's Disorder
Official title: Open Label Safety and Tolerability Trial of CPP-109 (Vigabatrin) in Adults With Treatment Refractory Tourette's Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Tourette's Disorder
Keywords: Tourette's Disorder
vigabatrin
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
First posted: 2012-04-25
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Maximum age (years): 35
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Global Severity Score on the Y-GTSS
timeFrame: weekly from baseline to end of study (10weeks)
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Tics and Tourette's Clinical and Research Program
state: New York
Study officials:
affiliation: NYU School of Medicine
name: Jonathan D Brodie, PhD,MD
role: PRINCIPAL_INVESTIGATOR
References: