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The goal of the current trial is to determine efficacy and safety of once-weekly aripiprazole in reducing Total Tic Severity (TTS) score in children and adolescents with Tourette's Disorder.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Matching Placebo | Placebo Comparator | Participants received aripiprazole matching placebo, tablets, orally, once weekly for 8 weeks in the Double-blind Treatment Period. |
|
| Aripiprazole 52.5 mg | Experimental | Participants received aripiprazole 52.5 mg, tablets, orally, once weekly for 8 weeks in the Double-blind Treatment Period. |
|
| Aripiprazole 77.5 mg | Experimental | Participants received aripiprazole 77.5 mg, tablets, orally, once weekly for 8 weeks in the Double-blind Treatment Period. |
|
| Aripiprazole 110 mg | Experimental | Participants received aripiprazole 110 mg, tablets, orally, once weekly for 8 weeks in the Double-blind Treatment Period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Aripiprazole | Drug | Aripiprazole tablets once weekly for 8 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline to Week 8 in Yale Global Tic Severity Scale (YGTSS) Total Tic Score | The YGTSS is a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0 to 5 for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe) with higher score represent more severe symptoms (greater reduction from baseline for greater improvement). | Baseline and Week 8 |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline in Clinical Global Impressions Scale for Tourette's Syndrome (CGI-TS) Score | The severity of illness and efficacy of study medication for each participant were rated using the CGI-TS scale. The study physician rated the participants total improvement whether or not it is due to study treatment. All responses were compared to the participants condition at Baseline (Day 0). Response choices include: 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. A negative change from Baseline indicates improvement. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Eva Kohegyi, MD | Otsuka Pharmaceutical Development & Commercialization, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Dothan | Alabama | 36303 | United States | |||
Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.
Data will be available after marketing approval in global markets, or beginning 1-3 years following article publication. There is no end date to the availability of the data.
Otsuka will share data on the Vivli data sharing platform which can be found here: https://vivli.org/ourmember/Otsuka/.
This study consisted of 2 distinct phases: a Pre-treatment Phase and a Treatment Phase. The Pre-treatment Phase consisted of a Screening period, a Washout period, and a Baseline visit. This was followed by an 8-week Treatment Phase. There was a follow-up period (30 days) for those participants who did not roll-over into the Open-label Study 31-10-274 (NCT01416441).
A total of 105 participants were screened and 83 were randomized to the treatment. The randomized participants were recruited at 38 sites in the following 5 countries: Bulgaria, Germany, Romania, Ukraine, and the US.
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| ID | Title | Description |
|---|---|---|
| FG000 | Aripiprazole 52.5 mg | Participants received aripiprazole 52.5 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| FG001 | Aripiprazole 77.5 mg | Participants received aripiprazole 77.5 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Placebo | Drug | Aripiprazole matching-placebo once-weekly for 8 weeks. |
|
| Baseline and Week 8 |
| Mean Change From Baseline in Gilles de la Tourette Quality of Life (GTS-QOL) Overall Score at Week 8 | The GTS-QOL is a disease-specific patient-reported scale for the measurement of health-related quality of life in participants with Tourette's Disorder, taking into account the complexity of the clinical picture of the disease. The questionnaire consists of a 27-item Tourette's Disorder-specific scale with 4 subscales (psychological, physical, obsessional, and cognitive). The GTS-QOL total score ranged from 0 (extremely dissatisfied with life) and 100 (extremely satisfied with life). A positive change from Baseline indicates improvement. | Baseline and Week 8 |
| San Francisco |
| California |
| 94143 |
| United States |
| Gainesville | Florida | 32610 | United States |
| Miami | Florida | 33126 | United States |
| Atlanta | Georgia | 30308 | United States |
| Atlanta | Georgia | 30329 | United States |
| Chicago | Illinois | 60612 | United States |
| Omaha | Nebraska | 68131 | United States |
| Manhasset | New York | 11030 | United States |
| Cincinnati | Ohio | 45229-3039 | United States |
| Oklahoma City | Oklahoma | 73112 | United States |
| Charlottesville | Virginia | 22903 | United States |
| La Crosse | Wisconsin | 54601 | United States |
| Rousse | 7004 | Bulgaria |
| Sofia | 1431 | Bulgaria |
| Varna | 9010 | Bulgaria |
| Aachen | 52074 | Germany |
| Dresden | 01307 | Germany |
| Hanover | 30625 | Germany |
| München | 80336 | Germany |
| Ulm | 89075 | Germany |
| Bucharest | 041914 | Romania |
| Cluj-Napoca | 400660 | Romania |
| Craiova | 200620 | Romania |
| Iași | 700282 | Romania |
| Donetsk | 83008 | Ukraine |
| Kharkiv | 61068 | Ukraine |
| Kharkiv | 61153 | Ukraine |
| Kyiv | 01030 | Ukraine |
| Kyiv | 03049 | Ukraine |
| Kyiv | 04209 | Ukraine |
| Luhansk | 91045 | Ukraine |
| Poltava | 36006 | Ukraine |
| Stepanovka | 73488 | Ukraine |
| Vinnytsia | 21005 | Ukraine |
| FG002 | Aripiprazole 110 mg | Participants received aripiprazole 110 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| FG003 | Matching Placebo | Participants received aripiprazole matching placebo, tablets, orally, once weekly for the 8-week Double-blind Treatment Period. |
| COMPLETED |
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| NOT COMPLETED |
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The Intention-to-Treat (ITT) Sample included participants randomly assigned to the double-blind treatment (regardless of whether or not they were dosed with study drug).
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| ID | Title | Description |
|---|---|---|
| BG000 | Aripiprazole 52.5 mg | Participants received aripiprazole 52.5 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| BG001 | Aripiprazole 77.5 mg | Participants received aripiprazole 77.5 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| BG002 | Aripiprazole 110 mg | Participants received aripiprazole 110 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| BG003 | Matching Placebo | Participants received aripiprazole matching placebo tablets, orally, once weekly for the 8-week Double-blind Treatment Period. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Number | Participants |
| ||||||||||||||||
| Weight | Mean | Standard Deviation | Kilogram |
| |||||||||||||||
| Height | Mean | Standard Deviation | centimeter |
| |||||||||||||||
| BMI (Body Mass Index) | Mean | Standard Deviation | kilogram per meter squared (kg/m^2) |
| |||||||||||||||
| Time since first diagnosis for Tourette's Disorder | Time since first diagnosis for Tourette's Disorder (years) = (date of screening assessment - date of first diagnosis + 1)/365.25 | Mean | Standard Deviation | years |
| ||||||||||||||
| Yale Global Tic Severity Scale Total Tic Score (YGTSS TTS) | The YGTSS is a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0 to 5 for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe). | Mean | Standard Deviation | score on a scale |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Mean Change From Baseline to Week 8 in Yale Global Tic Severity Scale (YGTSS) Total Tic Score | The YGTSS is a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0 to 5 for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe) with higher score represent more severe symptoms (greater reduction from baseline for greater improvement). | The Intention-to-Treat (ITT) Sample included participants randomly assigned to the double-blind treatment (regardless of whether or not they were dosed with study drug). Overall number of participants analyzed are participants with data available for analyses. | Posted | Mean | Standard Deviation | score on scale | Baseline and Week 8 |
|
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in Clinical Global Impressions Scale for Tourette's Syndrome (CGI-TS) Score | The severity of illness and efficacy of study medication for each participant were rated using the CGI-TS scale. The study physician rated the participants total improvement whether or not it is due to study treatment. All responses were compared to the participants condition at Baseline (Day 0). Response choices include: 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. A negative change from Baseline indicates improvement. | The Intention-to-Treat (ITT) Sample included participants randomly assigned to the double-blind treatment (regardless of whether or not they were dosed with study drug). Overall number of participants analyzed are participants with data available for analyses. | Posted | Mean | Standard Deviation | score on scale | Baseline and Week 8 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in Gilles de la Tourette Quality of Life (GTS-QOL) Overall Score at Week 8 | The GTS-QOL is a disease-specific patient-reported scale for the measurement of health-related quality of life in participants with Tourette's Disorder, taking into account the complexity of the clinical picture of the disease. The questionnaire consists of a 27-item Tourette's Disorder-specific scale with 4 subscales (psychological, physical, obsessional, and cognitive). The GTS-QOL total score ranged from 0 (extremely dissatisfied with life) and 100 (extremely satisfied with life). A positive change from Baseline indicates improvement. | The Intention-to-Treat (ITT) Sample included participants randomly assigned to the double-blind treatment (regardless of whether or not they were dosed with study drug). Overall number of participants analyzed are participants with data available for analyses. | Posted | Mean | Standard Deviation | score on scale | Baseline and Week 8 |
|
From first dose up to 30 days post last dose (Up to approximately 12 weeks)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Aripiprazole 52.5 mg | Participants received aripiprazole 52.5 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. | 0 | 20 | 0 | 20 | 8 | 20 |
| EG001 | Aripiprazole 77.5 mg | Participants received aripiprazole 77.5 mg, tablet, orally once weekly for the 8-week Double-blind Treatment Period. | 0 | 21 | 0 | 21 | 4 | 21 |
| EG002 | Aripiprazole 110 mg | Participants received aripiprazole 110 mg, tablet, orally once weekly for the 8-week Double-blind Treatment Period. | 0 | 21 | 0 | 21 | 16 | 21 |
| EG003 | Matching Placebo | Participants received aripiprazole matching placebo, tablets, orally, once weekly for the 8-week Double-blind Treatment Period. | 0 | 21 | 0 | 21 | 4 | 21 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tachycardia | Cardiac disorders | MedDra 16.0 | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDra 16.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDra 16.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDra 16.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDra 16.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDra 16.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDra 16.0 | Systematic Assessment |
| |
| Drug Prolactin Decreased | Investigations | MedDra 16.0 | Systematic Assessment |
| |
| Weight Increased | Investigations | MedDra 16.0 | Systematic Assessment |
| |
| Decreased Appetite | Metabolism and nutrition disorders | MedDra 16.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDra 16.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDra 16.0 | Systematic Assessment |
| |
| Sedation | Nervous system disorders | MedDra 16.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDra 16.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDra 16.0 | Systematic Assessment |
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Limitations of the trial such as small numbers of subjects analyzed or technical problems leading to unreliable data.
Sponsor reserves the right to review results publications prior to public release and can delay such publications for a period greater than 60 days but no more than 120 days from the date that the publication is submitted to the Sponsor for review. Sponsor can require changes to the publication to protect Sponsor's intellectual property rights and/or confidential information and reserves the right to limit publication timing and scope of data published based on the number of study locations.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Global Clinical Development | Otsuka Pharmaceutical Development & Commercialization, Inc. | 1-609-524-6788 | clinicaltransparency@otsuka-us.com |
| ID | Term |
|---|---|
| D005879 | Tourette Syndrome |
| D013981 | Tic Disorders |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D000068180 | Aripiprazole |
| ID | Term |
|---|---|
| D010879 | Piperazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D015363 | Quinolones |
| D011804 | Quinolines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
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| 13 to 17 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black or African American |
|
Participants received aripiprazole 110 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| OG003 | Matching Placebo | Participants received aripiprazole matching placebo, tablets, orally, once weekly for the 8-week Double-blind Treatment Period. |
|
|
Participants received aripiprazole 110 mg, tablet, orally, once weekly for the 8-week Double-blind Treatment Period. |
| OG003 | Matching Placebo | Participants received aripiprazole matching placebo, tablets, orally, once weekly for the 8-week Double-blind Treatment Period. |
|
|