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

• Sponsor: Barbara J. Coffey (Other)
• Overall Status: Completed
• CT.gov ID: NCT01585207
• Collaborator: (none)
• Enrollment: 4
• Location: 1
• Arm: 1
• Duration (Months): 32
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 4 participants
• Allocation: N/A
• 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: Jul 1, 2012
• Actual Primary Completion Date: Mar 1, 2015
• Actual Study Completion Date: Mar 1, 2015

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Global Severity Score on the Y-GTSS [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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 35 Years
• 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Tics and Tourette's Clinical and Research Program	New York	New York	United States	10029

Sponsors and Collaborators

• Barbara J. Coffey

Investigators

• Principal Investigator: Jonathan D Brodie, PhD,MD, NYU School of Medicine

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Barbara J. Coffey, Professor, Icahn School of Medicine at Mount Sinai
• ClinicalTrials.gov Identifier: NCT01585207
• Other Study ID Numbers: GCO 12-0964; 11-01864
• First Posted: Apr 25, 2012
• Last Update Posted: Jan 19, 2018
• Last Verified: Jan 1, 2018

• Keywords provided by Barbara J. Coffey, Professor, Icahn School of Medicine at Mount Sinai: Tourette's Disorder; vigabatrin
• Additional relevant MeSH terms: Tourette Syndrome; Disease; Vigabatrin

Study Results

Participant Flow

Arm/Group Title	Vigabatrin
Arm/Group Description	3 tablets, bid for 8 weeks
Period Title: Overall Study
STARTED	4
COMPLETED	4
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Vigabatrin
Arm/Group Description	3 tablets, bid for 8 weeks
Overall Participants	4
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	34.75
Sex: Female, Male (Count of Participants)
Female	1 25%
Male	3 75%
YGTSS (total tic) (total tics) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [total tics]	39 (3.67)

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Vigabatrin
Arm/Group Description	3 tablets, bid for 8 weeks
Measure Participants	4
Baseline	80.25
Week 0	78.75
Week 1	61
Week 2	69.75
Week 3	61
Week 4	59
Week 6	58.75
Week 8	60.88
Week 10	70.33

Adverse Events

Arm/Group Title	Vigabatrin
Arm/Group Description	3 tablets, bid for 8 weeks
All Cause Mortality
	Vigabatrin
	Affected / at Risk (%)	# Events
Total	0/4 (0%)
Serious Adverse Events
	Vigabatrin
	Affected / at Risk (%)	# Events
Total	0/4 (0%)
Other (Not Including Serious) Adverse Events
	Vigabatrin
	Affected / at Risk (%)	# Events
Total	4/4 (100%)
Eye disorders
Blurry Vision	1/4 (25%)
General disorders
Fatigue	3/4 (75%)
Appetite Increase	1/4 (25%)
Sleep Difficulties	1/4 (25%)
Dry mouth	1/4 (25%)
Daytime tiredness	1/4 (25%)
Vivid dreams	1/4 (25%)
Possible weight gain	1/4 (25%)
Nervous system disorders
Increase in OCD symptoms	1/4 (25%)
Mood swings	1/4 (25%)
Memory impairment	1/4 (25%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.