Tourette's DisorderPhase 1/2NCT01585207
Proof-of-Concept Safety Study of CPP-109 (Vigabatrin) for Treatment Refractory Tourette's Disorder
A Phase 1/2 study testing vigabatrin in people with Tourette's Disorder, run by Barbara J. Coffey at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 to 35 years
- Conditions the study is for
- Tourette's Disorder
- What is being tested?
- vigabatrin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2012to Mar 2015
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 35 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Tics and Tourette's Clinical and Research Program
New York, New York, 10029
About this study
Official title: 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.
Read the 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.
- Conditions
- Tourette's Disorder
- Being tested
- vigabatrin
- Keywords
- Tourette's Disorder, vigabatrin
- Medical subject headings
- Tourette Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vigabatrinDrug
Also called CPP-109 Vigabatrin
3 tablets, bid for 8 weeks
| Study group | Role | What they receive |
|---|---|---|
| Vigabatrin | Experimental | 3 tablets, bid for 8 weeks Receives: Drug: vigabatrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: weekly from baseline to end of study (10weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2012 (estimated)
- Study startJul 2012
- Primary completionMar 2015
- Study completionMar 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Tourette's DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Barbara J. Coffey
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Barbara J. Coffey, Icahn School of Medicine at Mount Sinai
- Investigators
- Jonathan D Brodie, PhD,MD, Principal investigator, NYU School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01585207
- Sponsor's protocol ID
- GCO 12-0964
- Other IDs
- 11-01864
- Status last verified
- Jan 2018
- First submitted
- 24 Apr 2012
- Last updated
- 19 Jan 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Safety and Tolerability Trial of CPP-109 (Vigabatrin) in Adults With Treatment Refractory Tourette's Disorder
- ClinicalTrials.gov ID
- NCT01585207
- Acronym / sponsor ID
- GCO 12-0964
- Conditions
- Tourette's Disorder
- Keywords
- Tourette's Disorder, vigabatrin
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Barbara J. CoffeyUniversity, hospital or other
- Study start
- Jul 2012
- Primary completion
- Mar 2015 (actual)
- Study completion
- Mar 2015 (actual)
- First posted
- 25 Apr 2012 (estimated)
- Results
- Posted 19 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vigabatrin | Experimental | Drug: vigabatrin3 tablets, bid for 8 weeks |
Who may qualify
- Ages
- 18 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- 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).
Key exclusion criteria (14 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| vigabatrinAlso: CPP-109 Vigabatrin3 tablets, bid for 8 weeks | Drug | Vigabatrin |
Primary outcome
Global Severity Score on the Y-GTSS
Time frame: 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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jonathan D Brodie, PhD,MD principal investigator
NYU School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing vigabatrin in people with Tourette's Disorder, run by Barbara J. Coffey at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Jan 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Global Severity Score on the Y-GTSS (time frame: weekly from baseline to end of study (10weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jan 2018.