Tic Disorders, Tourette's Syndrome
Conditions
Keywords
Psychiatric, Clinical Trial, Pediatrics, Tourette's Disorder, Tic Disorder
Brief summary
The purpose of this study is to determine if Abilify will reduce tics (repetitive, uncontrollable movements or vocalizations) in children and adolescents ages 7-18 with Tourette's Disorder (TD) or a chronic motor tic disorder (either repetitive, uncontrollable movements or vocalizations).
Detailed description
The aims of this study are to obtain systematic data regarding dosing and safety of aripiprazole (Abilify) in the treatment of youth with Tourette's Disorder (TD). Tourette's Disorder is characterized by multiple motor (more than one uncontrollable movement) and vocal tics (vocal outbursts) which have been present for more than 1 year, with onset before the age of 18. The disorder causes marked distress in social, occupational or other important areas of functioning. Abilify has been approved by the United States Food and Drug Administration (FDA) to treat adults with schizophrenia but has not been approved to treat Tourette's Disorder (TD) so it is considered experimental or investigational in this study.
Interventions
DRUGAripiprazole
Baseline Visit 2: Subjects btw 25-50kg start on 1.25mg/day, btw 50-70kg start on 2.5mg/day, greater than 70kg start on 5mg/day Visit 3: Titrated based on YGTSS & CGI-TS ratings at investigator discretion. Subjects who show evidence of response (reduction in CGI-TS by 1-2 points) may remain on same dose. Subjects who show no response may increase as follows: btw 25-50kg increase to 2.5mg/day, btw 50-70kg increase to 3.75mg/day, greater than 70kg increase to 7.5mg/day Visit 5: Subjects who show no response may increase as follows: btw 25-50kg increase to 3.75mg/day, btw 50-70kg increase to 5mg/day, greater than 70kg increase to 10mg/day Visit 6: Subjects who show no response may be increase as follows: btw 25-50kg increase to 5mg/day, btw 50-70kg increase to 7.5mg/day, greater than 70kg increase to 12.5mg/day Visit 7: Subjects who how no response may be increase as follows: btw 25-50kg increase to 7.5mg/day, btw 50-70kg increase to 10mg/day, greater than 70kg increase to 15mg/day
Sponsors
NYU Langone Health
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Child or adolescent must be 7 to 18 years of age (inclusive) when informed consent is obtained. * Must meet full criteria for Tourette's Disorder or chronic motor tic disorder. * Must have failed to respond to an adequate trial, as determined by the investigator, of clonidine, guanfacine, or neuroleptic medication in the past. * Tics are causing significant distress or impairment, as determined by parent/subject and principal investigator, on 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). * Must be able to swallow pills. * Must be of normal intelligence in the judgment of the investigator. * Must possess an educational level, degree of understanding and command of the English language to enable them to communicate suitably with the investigator and study coordinator, and to understand the nature of the study. * Subjects and their legal representatives must be considered reliable. * Written informed consent of parents and subjects (ages 18 and above) and assent of subjects ages 7-17 will be obtained.
Exclusion criteria
* Organic brain disease, for example, traumatic brain injury residua. * Mental retardation as defined by the DSM-IV-TR. * A history of seizure disorder (other than febrile seizure). * A history of Sydenham's Chorea. * Autism, schizophrenia, other psychotic disorder, or bipolar disorder. * A primary diagnosis of a major mood disorder that requires ongoing psychiatric treatment. * A neurological disorder other than a tic disorder. * A major medical illness. * Females who are of child bearing age who are unwilling to use birth control or who are pregnant, as determined by serum pregnancy test at baseline assessment, or lactating. * Have a past or current history of substance dependence and/or a current history of substance abuse or who fail baseline toxic screen. * Have any clinically significant abnormal laboratory result at baseline screening including EKG, or blood tests. * Have a history of ongoing or previously undisclosed child abuse (risk of removal from home would not allow for consistent caretaker ratings).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | 8 Weeks | The Yale Global Tic Severity Scale (YGTSS) is a clinical rating instrument that was designed for use in studies of Tourette's syndrome and other tic disorders. The YGTSS provides an evaluation of the number, frequency, intensity, complexity, and interference of motor and phonic symptoms. The maximum YGTSS Global score is 100, while the maximum motor score is 25, the maximum vocal score is 25, and the maximum impairment score is 50. Higher scores indicate more severe tics. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Global Impression Severity Scores | 24 Months | The Clinical Global Impression scale (CGI) is a classic instrument for making global assessments. This scale yields three different measures: 1. Severity of illness (7-point scale, with 7 being the most impaired; assessment of patient's current symptom severity, referred to here as CGIs), 2. Global improvement (7-point scale, with 7 being the most impaired; comparison of patient's baseline condition with his/her current condition, referred to here as CGIi), 3. Efficacy index (4 point x 4 point rating scale, comparison of patient's baseline condition with a ratio of current therapeutic benefit to severity of side effects) |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Sample Sample of children and adolescents that enrolled in study to receive active medication. This was not a placebo controlled study. | 11 |
| Total | 11 |
Baseline characteristics
| Characteristic | Sample |
|---|---|
| Age, Categorical <=18 years | 10 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Age, Continuous | 13.36 years STANDARD_DEVIATION 3.33 |
| Gender Female | 1 Participants |
| Gender Male | 10 Participants |
| Region of Enrollment United States | 11 participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 11 / 11 |
| serious Total, serious adverse events | 0 / 11 |
Outcome results
Primary
Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales)
The Yale Global Tic Severity Scale (YGTSS) is a clinical rating instrument that was designed for use in studies of Tourette's syndrome and other tic disorders. The YGTSS provides an evaluation of the number, frequency, intensity, complexity, and interference of motor and phonic symptoms. The maximum YGTSS Global score is 100, while the maximum motor score is 25, the maximum vocal score is 25, and the maximum impairment score is 50. Higher scores indicate more severe tics.
Time frame: 8 Weeks
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1 - Baseline | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Global Severity | 61.82 units on a scale | Standard Deviation 13.49 |
| Group 1 - Baseline | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Motor Tic | 15.82 units on a scale | Standard Deviation 4.4 |
| Group 1 - Baseline | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Vocal Tic | 12.36 units on a scale | Standard Deviation 7.1 |
| Group 1 - Baseline | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Total Tic | 28.18 units on a scale | Standard Deviation 7.74 |
| Group 1 - Endpoint | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Total Tic | 16.73 units on a scale | Standard Deviation 7.54 |
| Group 1 - Endpoint | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Global Severity | 33.73 units on a scale | Standard Deviation 15.18 |
| Group 1 - Endpoint | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Vocal Tic | 7.00 units on a scale | Standard Deviation 5.76 |
| Group 1 - Endpoint | Calculating Difference Between Means (Baseline and Endpoint Scores on the Yale Global Tic Severity Scale Subscales) | YGTSS Motor Tic | 9.73 units on a scale | Standard Deviation 2.76 |
Comparison: Group 1 Baseline vs. Endpointp-value: <0.05Wilcoxon (Mann-Whitney)
Secondary
Clinical Global Impression Severity Scores
The Clinical Global Impression scale (CGI) is a classic instrument for making global assessments. This scale yields three different measures: 1. Severity of illness (7-point scale, with 7 being the most impaired; assessment of patient's current symptom severity, referred to here as CGIs), 2. Global improvement (7-point scale, with 7 being the most impaired; comparison of patient's baseline condition with his/her current condition, referred to here as CGIi), 3. Efficacy index (4 point x 4 point rating scale, comparison of patient's baseline condition with a ratio of current therapeutic benefit to severity of side effects)
Time frame: 24 Months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1 - Baseline | Clinical Global Impression Severity Scores | 4.45 units on a scale | Standard Deviation 0.52 |
| Group 1 - Endpoint | Clinical Global Impression Severity Scores | 3.18 units on a scale | Standard Deviation 0.6 |
Comparison: Mean scores baseline to endpointp-value: <0.05Wilcoxon (Mann-Whitney)