Trial Outcomes & Findings for Open Label Trial of Aripiprazole in Children and Adolescents With Tourette's Disorder (NCT NCT00241176)

Last Updated: 2016-12-09

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

11 participants

Primary outcome timeframe

8 Weeks

Results posted on

2016-12-09

Participant Flow

Participant milestones

Participant milestones
Measure	Aripiprazole Subjects received initial dose based on body weight at Visit 2: Subjects between 25-50 kg were started on 1.25 mg/day, Subjects between 50-70 kg were started on 2.5 mg/day, Subjects greater than 70 kg were started on 5 mg/day. Dosage was titrated at Visit 3, 5, 6, or 7 based on YGTSS and CGI-TS ratings at the discretion of the investigator. Subjects who showed evidence of response (reduction in CGI-TS by 1-2 points)remained on the same dose. Subjects who did not show evidence of response could be increased: Subjects between 25-50 kg were could be increased 1.25 mg/day at each titration visit, Subjects between 50-70 kg could be increased 2.5 mg/day at each titration visit, and Subjects greater than 70 kg could be increased 5 mg/day at each titration visit.
Overall Study STARTED	11
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Open Label Trial of Aripiprazole in Children and Adolescents With Tourette's Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sample n=11 Participants Sample of children and adolescents that enrolled in study to receive active medication. This was not a placebo controlled study.
Age, Categorical <=18 years	10 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	13.36 years STANDARD_DEVIATION 3.33 • n=5 Participants
Gender Female	1 Participants n=5 Participants
Gender Male	10 Participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants

PRIMARY outcome

Timeframe: 8 Weeks

Outcome measures

Outcome measures
Measure	Group 1 - Baseline n=11 Participants Sample of children and adolescents that enrolled in study to receive active medication at baseline.	Group 1 - Endpoint n=11 Participants Sample of children and adolescents that enrolled in study to receive active medication at endpoint prior to down titration.
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 4.40	9.73 units on a scale Standard Deviation 2.76
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 7.10	7.00 units on a scale Standard Deviation 5.76
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	16.73 units on a scale Standard Deviation 7.54
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	33.73 units on a scale Standard Deviation 15.18

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Group 1 - Baseline n=11 Participants Sample of children and adolescents that enrolled in study to receive active medication at baseline.	Group 1 - Endpoint n=11 Participants Sample of children and adolescents that enrolled in study to receive active medication at endpoint prior to down titration.
Clinical Global Impression Severity Scores	4.45 units on a scale Standard Deviation 0.52	3.18 units on a scale Standard Deviation 0.60

Adverse Events

Sample

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Sample n=11 participants at risk Sample of children and adolescents that enrolled in study to receive active medication. This was not a placebo controlled study.
General disorders Headache	100.0% 11/11 • Number of events 34
Musculoskeletal and connective tissue disorders Muscle, bone, or joint pain condition	90.9% 10/11 • Number of events 31
General disorders Appetite increase/weight gain	63.6% 7/11 • Number of events 27
Gastrointestinal disorders Stomach discomfort	81.8% 9/11 • Number of events 26
General disorders Tiredness/fatigue	72.7% 8/11 • Number of events 23
General disorders Dizziness	81.8% 9/11 • Number of events 18
General disorders Appetite decrease/weight loss	54.5% 6/11 • Number of events 12
General disorders Drowsiness/sedation	63.6% 7/11 • Number of events 13
General disorders Dry mouth	36.4% 4/11 • Number of events 10

Additional Information

Barbara J Coffey, M.D, M.S.

NYU School of Medicine, NYU Child Study Center

Phone: (212)263-3926

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place