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

NCT01585207 | Completed Phase 1 Results DMC

• 2 other identifiers: GCO 12-096411-01864
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

Tourette's Disorder

Keywords

Tourette's Disorder; vigabatrin

Outcome Measures

Primary Outcomes (1)

• 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)

Study Arms (1)

Vigabatrin

EXPERIMENTAL

3 tablets, bid for 8 weeks

Drug: vigabatrin

Interventions

vigabatrin DRUG

3 tablets, bid for 8 weeks

Also known as: CPP-109 Vigabatrin

Vigabatrin

Eligibility Criteria

• Age: 18 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Subjects must be between 18 and 35 years of age (inclusive) when informed consent is obtained.
• 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.
• 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.
• Tics are causing significant distress or impairment, as determined by the subject and principal investigator, on the current treatment regimen.
• Laboratory results, including serum chemistries, hematology, and urinalysis, must show no significant abnormalities (significant is defined as laboratory values requiring acute medical intervention).
• Subjects will not undergo formal IQ testing, but must be of normal intelligence in the judgment of the investigator.
• 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.
• Subjects must be considered reliable.
• Written informed consent of subjects is obtained.

You may not qualify if:

• Subjects with organic brain disease, for example, traumatic brain injury residua.
• Subjects with a preexisting ophthalmologic condition.
• 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.
• Subjects meeting criteria for mental retardation as defined by the DSM-IV-TR.
• Subjects with a history of seizure disorder (other than febrile seizure).
• Subjects with history of Sydenham's Chorea.
• Subjects with autism, schizophrenia, other psychotic disorder, or bipolar disorder.
• Subjects with a primary diagnosis of a major mood disorder that requires ongoing psychiatric treatment.
• Subjects with a neurological disorder other than a tic disorder.
• Subjects with a major medical illness.
• Female subjects who are unwilling to use birth control or who are pregnant, as determined by serum pregnancy test at baseline assessment, or lactating.
• 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.
• Subjects who have any clinically significant abnormal laboratory result at baseline screening including EKG, or blood tests.
• Subjects who, in the opinion of the investigator, are unsuitable in any other way to participate in this study.

Sponsors & Collaborators

• Barbara J. Coffey: lead

Study Sites (1)

Tics and Tourette's Clinical and Research Program

New York, New York, 10029, United States

Location

MeSH Terms

Conditions

Tourette Syndrome

Interventions

Vigabatrin

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

gamma-Aminobutyric Acid; Aminobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Dr. Barbara J. Coffey
• Organization: Icahn School of Medicine at Mount Sinai

Barbara.coffey@mssm.edu

212-659-1663

Study Officials

• Jonathan D Brodie, PhD,MD

  NYU School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 24, 2012
• First Posted: April 25, 2012
• Study Start: July 1, 2012
• Primary Completion: March 1, 2015
• Study Completion: March 1, 2015
• Last Updated: January 19, 2018
• Results First Posted: January 19, 2018

Record last verified: 2018-01

Locations

US (1)