Trial Outcomes & Findings for Study of Aripiprazole in the Treatment of Children and Adolescents With Autistic Disorder (AD). (NCT NCT00332241)
NCT ID: NCT00332241
Last Updated: 2026-05-13
Results Overview
The ABC is a 58-item informant-based assessment of problem behaviors in children/adolescents with mental retardation. Items are rated on a 4-point scale (0=no problem, 3=severe problem), and resolve into 5 domain subscales. A decrease in score indicates improvement.
COMPLETED
PHASE3
98 participants
Week 8
2026-05-13
Participant Flow
Participant milestones
| Measure |
Placebo
oral placebo once daily (QD)
|
Aripiprazole
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Overall Study
STARTED
|
51
|
47
|
|
Overall Study
COMPLETED
|
36
|
39
|
|
Overall Study
NOT COMPLETED
|
15
|
8
|
Reasons for withdrawal
| Measure |
Placebo
oral placebo once daily (QD)
|
Aripiprazole
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Overall Study
Lack of Efficacy
|
6
|
1
|
|
Overall Study
Adverse Event
|
3
|
5
|
|
Overall Study
Withdrawal by Subject
|
2
|
1
|
|
Overall Study
Lost to Follow-up
|
4
|
1
|
Baseline Characteristics
Study of Aripiprazole in the Treatment of Children and Adolescents With Autistic Disorder (AD).
Baseline characteristics by cohort
| Measure |
Placebo
n=51 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=47 Participants
oral aripiprazole 2 to 15 mg QD
|
Total
n=98 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Race, Customized
Asian
|
0 participants
n=1512 Participants
|
2 participants
n=504 Participants
|
2 participants
n=2016 Participants
|
|
Race, Customized
Native Hawaiian or Other Pacific Islander
|
0 participants
n=1512 Participants
|
1 participants
n=504 Participants
|
1 participants
n=2016 Participants
|
|
Race, Customized
Black or African American
|
7 participants
n=1512 Participants
|
11 participants
n=504 Participants
|
18 participants
n=2016 Participants
|
|
Race, Customized
White
|
41 participants
n=1512 Participants
|
32 participants
n=504 Participants
|
73 participants
n=2016 Participants
|
|
Race, Customized
Other
|
3 participants
n=1512 Participants
|
1 participants
n=504 Participants
|
4 participants
n=2016 Participants
|
|
Ethnicity, Customized
Hispanic/Latino
|
8 participants
n=1512 Participants
|
6 participants
n=504 Participants
|
14 participants
n=2016 Participants
|
|
Ethnicity, Customized
Not Hispanic/Latino
|
43 participants
n=1512 Participants
|
41 participants
n=504 Participants
|
84 participants
n=2016 Participants
|
|
Weight Group
<40 kilograms
|
32 Participants
n=1512 Participants
|
26 Participants
n=504 Participants
|
58 Participants
n=2016 Participants
|
|
Weight Group
≥40 kilograms
|
19 Participants
n=1512 Participants
|
21 Participants
n=504 Participants
|
40 Participants
n=2016 Participants
|
|
Body Mass Index
|
19.96 kg/cm2
STANDARD_DEVIATION 5.392 • n=1512 Participants
|
21.08 kg/cm2
STANDARD_DEVIATION 5.394 • n=504 Participants
|
20.50 kg/cm2
STANDARD_DEVIATION 5.395 • n=2016 Participants
|
|
Height
|
138.6 centimeters
STANDARD_DEVIATION 14.7 • n=1512 Participants
|
140.9 centimeters
STANDARD_DEVIATION 18.80 • n=504 Participants
|
139.7 centimeters
STANDARD_DEVIATION 16.74 • n=2016 Participants
|
|
Weight
|
40.6 kilograms
STANDARD_DEVIATION 18.92 • n=1512 Participants
|
43.9 kilograms
STANDARD_DEVIATION 19.15 • n=504 Participants
|
42.2 kilograms
STANDARD_DEVIATION 19.01 • n=2016 Participants
|
|
Age, Customized
6 to 12 years
|
46 participants
n=1512 Participants
|
37 participants
n=504 Participants
|
83 participants
n=2016 Participants
|
|
Age, Customized
13 to 17 years
|
5 participants
n=1512 Participants
|
10 participants
n=504 Participants
|
15 participants
n=2016 Participants
|
|
Sex/Gender, Customized
Female
|
7 Participants
n=1512 Participants
|
5 Participants
n=504 Participants
|
12 Participants
n=2016 Participants
|
|
Sex/Gender, Customized
Male
|
44 Participants
n=1512 Participants
|
42 Participants
n=504 Participants
|
86 Participants
n=2016 Participants
|
PRIMARY outcome
Timeframe: Week 8Population: Efficacy population=all randomized participants minus 2 patients in the placebo group (1 lost to follow-up, 1 withdrew consent) and 1 participant in the aripiprazole group who discontinued due to AE on Day 2. Data set is LOCF.
The ABC is a 58-item informant-based assessment of problem behaviors in children/adolescents with mental retardation. Items are rated on a 4-point scale (0=no problem, 3=severe problem), and resolve into 5 domain subscales. A decrease in score indicates improvement.
Outcome measures
| Measure |
Placebo
n=49 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=46 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Mean Change (Week 8 - Baseline) in the Autistic Behavior Checklist (ABC) Irritability Subscale Score
|
-5.0 units on a scale
Standard Error 1.43
|
-12.9 units on a scale
Standard Error 1.44
|
SECONDARY outcome
Timeframe: Week 8Population: Efficacy population=all randomized participants minus 2 patients in the placebo group (1 lost to follow-up, 1 withdrew consent) and 1 participant in the aripiprazole group who discontinued due to AE on Day 2. Data set is LOCF.
The CGI scale is a clinician-rated global assessment of a patient's improvement over time. Baseline assessment rated a patient's condition on a 7-point scale (1=no symptoms, 7=very severe symptoms). Subsequent assessed improvement relative to baseline symptoms on a 7-point CGI-I item scale (1=very much improved, 7=very much worse).
Outcome measures
| Measure |
Placebo
n=49 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=46 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Mean Clinical Global Impressions Improvement Scale (CGI-I) Score
|
3.6 units on a scale
Standard Error 0.18
|
2.2 units on a scale
Standard Error 0.18
|
SECONDARY outcome
Timeframe: Week 8Population: Efficacy population=all randomized participants minus 2 patients in the placebo group (1 lost to follow-up, 1 withdrew consent) and 1 participant in the aripiprazole group who discontinued due to AE on Day 2. Data set is LOCF.
Response defined as a ≥ 25% reduction from baseline to endpoint in the ABC Irritability Subscale score and a CGI-I score of 1 or 2 at endpoint
Outcome measures
| Measure |
Placebo
n=49 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=46 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Number of Participants With Response at Week 8
|
7 participant
|
24 participant
|
SECONDARY outcome
Timeframe: Week 8Population: Efficacy population=all randomized participants minus 2 patients in the placebo group (1 lost to follow-up, 1 withdrew consent) and 1 participant in the aripiprazole group who discontinued due to AE on Day 2. Data set is LOCF.
CY-BOCS=10-item assessment of obsessive-compulsive symptoms in patients \<18 years. 5 items pertaining to compulsions rate symptoms (time spent, interference with functioning, distress, resistance, control) on a 5-point scale (0=no symptoms/minimum severity, 4=extreme symptoms/maximum severity). A decrease in value indicates improvement.
Outcome measures
| Measure |
Placebo
n=44 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=43 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Mean Change (Week 8 - Baseline) in the Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS; Compulsion Scale Only)
|
-0.8 units on a scale
Standard Error 0.52
|
-3.8 units on a scale
Standard Error 0.50
|
SECONDARY outcome
Timeframe: Week 8Population: Efficacy population=all randomized participants minus 2 patients in the placebo group (1 lost to follow-up, 1 withdrew consent) and 1 participant in the aripiprazole group who discontinued due to AE on Day 2. Data set is LOCF.
Mean change (Week 8 - baseline) in the other ABC subscale scores (lethargy/social withdrawal; stereotypic behavior; hyperactivity/ noncompliance; inappropriate speech). A decrease in value indicates improvement
Outcome measures
| Measure |
Placebo
n=49 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=46 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Mean Change (Week 8 - Baseline) in the Other ABC Subscale Scores
ABC Hyperactivity Subscale Score
|
-2.8 units of a scale
Standard Error 1.5
|
-12.7 units of a scale
Standard Error 1.52
|
|
Mean Change (Week 8 - Baseline) in the Other ABC Subscale Scores
ABC Stereotypy Subscale Score
|
-2.0 units of a scale
Standard Error 0.62
|
-4.8 units of a scale
Standard Error 0.63
|
|
Mean Change (Week 8 - Baseline) in the Other ABC Subscale Scores
ABC Inappropriate Speech Subscale Score
|
-0.4 units of a scale
Standard Error 0.39
|
-2.5 units of a scale
Standard Error 0.39
|
|
Mean Change (Week 8 - Baseline) in the Other ABC Subscale Scores
ABC Social Withdrawal Subscale Score
|
-6.2 units of a scale
Standard Error 1.13
|
-7.9 units of a scale
Standard Error 1.15
|
SECONDARY outcome
Timeframe: Week 8Population: Efficacy population=all randomized participants minus 2 patients in the placebo group (1 lost to follow-up, 1 withdrew consent) and 1 participant in the aripiprazole group who discontinued due to AE on Day 2. Data set is LOCF.
A CGI-S assessment (a 7-point scale to evaluate the severity of symptoms) was performed at baseline (1=no symptoms; 7=very severe symptoms). The patient's improvement relative to the symptoms at baseline on were assessed on a 7-point CGI-I (1=very much improved; 7=very much worse). A decrease in value indicates improvement.
Outcome measures
| Measure |
Placebo
n=40 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=40 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Mean Change (Week 8 - Baseline) in CGI-Severity (CGI-S)
|
-0.4 units on a scale
Standard Deviation 0.15
|
-1.2 units on a scale
Standard Deviation 0.14
|
SECONDARY outcome
Timeframe: continuous throughout the studyPopulation: Safety population=all randomized participants minus 1 patient in the placebo group lost to follow-up. Data set is LOCF.
Deaths, Adverse Events (AEs), Serious AEs (SAEs), Treatment-Emergent AEs and AEs leading to discontinuation
Outcome measures
| Measure |
Placebo
n=50 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=47 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Summary of Safety
Treatment-emergent extrapyramidal symptom AEs
|
4 participants
|
7 participants
|
|
Summary of Safety
AEs leading to discontinuation
|
3 participants
|
5 participants
|
|
Summary of Safety
Treatment-emergent AEs overall
|
36 participants
|
43 participants
|
|
Summary of Safety
Treatment-emergent AEs realted to study drug
|
25 participants
|
39 participants
|
|
Summary of Safety
Deaths
|
0 participants
|
0 participants
|
|
Summary of Safety
Treatment-emergent SAEs
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Week 8Population: Safety population=all randomized participants minus 1 patient in the placebo group lost to follow-up. Includes all participants with weight measurement at baseline and timepoint. Data set is LOCF.
Adjusted mean change (Week 8 - baseline) in body weight
Outcome measures
| Measure |
Placebo
n=49 Participants
oral placebo once daily (QD)
|
Aripiprazole
n=45 Participants
oral aripiprazole 2 to 15 mg QD
|
|---|---|---|
|
Change From Baseline in Body Weight
|
0.8 kilograms
Standard Error 0.29
|
2.0 kilograms
Standard Error 0.30
|
Adverse Events
Aripiprazole
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Aripiprazole
n=47 participants at risk
|
Placebo
n=50 participants at risk
|
|---|---|---|
|
Psychiatric disorders
INSOMNIA
|
6.4%
3/47
|
8.0%
4/50
|
|
Psychiatric disorders
AGGRESSION
|
2.1%
1/47
|
8.0%
4/50
|
|
Nervous system disorders
TREMOR
|
8.5%
4/47
|
0.00%
0/50
|
|
Nervous system disorders
DROOLING
|
8.5%
4/47
|
0.00%
0/50
|
|
Nervous system disorders
HEADACHE
|
6.4%
3/47
|
16.0%
8/50
|
|
Nervous system disorders
SEDATION
|
10.6%
5/47
|
2.0%
1/50
|
|
Nervous system disorders
SOMNOLENCE
|
17.0%
8/47
|
4.0%
2/50
|
|
Gastrointestinal disorders
VOMITING
|
14.9%
7/47
|
4.0%
2/50
|
|
Gastrointestinal disorders
DIARRHOEA
|
8.5%
4/47
|
10.0%
5/50
|
|
Infections and infestations
NASOPHARYNGITIS
|
4.3%
2/47
|
6.0%
3/50
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
2.1%
1/47
|
10.0%
5/50
|
|
Renal and urinary disorders
ENURESIS
|
6.4%
3/47
|
8.0%
4/50
|
|
Metabolism and nutrition disorders
INCREASED APPETITE
|
14.9%
7/47
|
10.0%
5/50
|
|
Respiratory, thoracic and mediastinal disorders
NASAL CONGESTION
|
6.4%
3/47
|
2.0%
1/50
|
|
General disorders
FATIGUE
|
21.3%
10/47
|
4.0%
2/50
|
|
General disorders
PYREXIA
|
8.5%
4/47
|
2.0%
1/50
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Bristol-Myers Squibb Co. agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing, but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
- Publication restrictions are in place
Restriction type: OTHER