Trial Outcomes & Findings for Study of Aripiprazole in the Treatment of Serious Behavioral Problems in Children and Adolescents With Autistic Disorder (AD) (NCT NCT00365859)

NCT ID: NCT00365859

Last Updated: 2013-12-02

Results Overview

Participants with Adverse Events (AEs), Deaths, Serious AEs (SAEs), and AEs leading to study discontinuation. AE is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition. SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires inpatient hospitalization or causes prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a cancer, is a congenital anomaly/birth defect, results in the development of drug dependency or drug abuse, is an important medical event.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

330 participants

Primary outcome timeframe

From Screening (up to 42 days prior to treatment start) through Week 52 (end of study) for SAEs; from Week 0 (Baseline) through Week 52 (End of Study) for AEs

Results posted on

2013-12-02

Participant Flow

109 participants were enrolled in the De Novo arm, 70 in the Rollover Placebo arm, and 174 in the Rollover Aripiprazole arm. 23 participants in the De Novo arm were considered "baseline failures," and did not enter the treatment phase.

Participant milestones

Participant milestones
Measure
De Novo
De novo participants (those who did not participate in protocol (CN138-178 \[NCT00332241\] or CN138-179 \[NCT00337571\]) assigned to open-label aripiprazole (oral tablet), flexibly dosed (2 to 15 mg/day) taken once daily, started at 2 mg/day on Day 1. Target daily dose was 5 mg, 10 mg, or 15 mg; maximum dose, regardless of weight, was 15 mg. Dose increases were incremental (dose levels are 2 mg, 5 mg, 10 mg, and 15 mg), occurring no more often than every 4 days, and were based on assessment of efficacy and tolerability at the current dose. The dosage could be adjusted downward if the patient experienced intolerance at any time to the current dose.
Rollover Placebo
Participants who completed participation in protocol (CN138-178 \[NCT00332241\] or CN138-179 \[NCT00337571\]) on placebo treatment and continued to meet all of the inclusion criteria and none of the exclusion criteria. Assigned in this study to open-label aripiprazole (oral tablet), flexibly dosed (2 to 15 mg/day) taken once daily, started at 2 mg/day on Day 1. Target daily dose was 5 mg, 10 mg, or 15 mg; maximum dose, regardless of weight, was 15 mg. Dose increases were incremental (dose levels are 2 mg, 5 mg, 10 mg, and 15 mg), occurring no more often than every 4 days, and were based on assessment of efficacy and tolerability at the current dose. The dosage could be adjusted downward if the patient experienced intolerance at any time to the current dose.
Rollover Aripiprazole
Participants who completed participation in protocol CN138-178 \[NCT00332241\] or CN138-179 \[NCT00337571\] on aripiprazole treatment and continued to meet all of the inclusion criteria and none of the exclusion criteria. Assigned in this study to open-label aripiprazole (oral tablet), flexibly dosed (2 to 15 mg/day) taken once daily, started at 2 mg/day on Day 1. Target daily dose was 5 mg, 10 mg, or 15 mg; maximum dose, regardless of weight, was 15 mg. Dose increases were incremental (dose levels are 2 mg, 5 mg, 10 mg, and 15 mg), occurring no more often than every 4 days, and were based on assessment of efficacy and tolerability at the current dose. The dosage could be adjusted downward if the patient experienced intolerance at any time to the current dose.
Total
Overall Study
STARTED
86
70
174
330
Overall Study
Safety Population
86
70
174
330
Overall Study
Efficacy Population
84
69
169
322
Overall Study
COMPLETED
55
37
107
199
Overall Study
NOT COMPLETED
31
33
67
131

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Aripiprazole in the Treatment of Serious Behavioral Problems in Children and Adolescents With Autistic Disorder (AD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Total of all reporting groups
Age, Customized
6 to 12 years
69 participants
n=5 Participants
56 participants
n=7 Participants
138 participants
n=5 Participants
263 participants
n=4 Participants
Age, Customized
13 to 17 years
17 participants
n=5 Participants
14 participants
n=7 Participants
36 participants
n=5 Participants
67 participants
n=4 Participants
Age Continuous
9.7 years
STANDARD_DEVIATION 3.13 • n=5 Participants
9.6 years
STANDARD_DEVIATION 2.95 • n=7 Participants
9.5 years
STANDARD_DEVIATION 3.00 • n=5 Participants
9.6 years
STANDARD_DEVIATION 3.02 • n=4 Participants
Sex: Female, Male
Female
16 Participants
n=5 Participants
8 Participants
n=7 Participants
19 Participants
n=5 Participants
43 Participants
n=4 Participants
Sex: Female, Male
Male
70 Participants
n=5 Participants
62 Participants
n=7 Participants
155 Participants
n=5 Participants
287 Participants
n=4 Participants

PRIMARY outcome

Timeframe: From Screening (up to 42 days prior to treatment start) through Week 52 (end of study) for SAEs; from Week 0 (Baseline) through Week 52 (End of Study) for AEs

Population: Safety Population

Participants with Adverse Events (AEs), Deaths, Serious AEs (SAEs), and AEs leading to study discontinuation. AE is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition. SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires inpatient hospitalization or causes prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a cancer, is a congenital anomaly/birth defect, results in the development of drug dependency or drug abuse, is an important medical event.

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Number of Participants With Serious Adverse Events (SAEs), Treatment-Emergent Adverse Events (AEs), Deaths, AEs Leading to Discontinuation, Extra Pyramidal Syndrome (EPS)-Related AEs
Deaths
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Serious Adverse Events (SAEs), Treatment-Emergent Adverse Events (AEs), Deaths, AEs Leading to Discontinuation, Extra Pyramidal Syndrome (EPS)-Related AEs
Treatment-Emergent SAEs
3 Participants
1 Participants
5 Participants
9 Participants
Number of Participants With Serious Adverse Events (SAEs), Treatment-Emergent Adverse Events (AEs), Deaths, AEs Leading to Discontinuation, Extra Pyramidal Syndrome (EPS)-Related AEs
Treatment-Emergent AEs
75 Participants
63 Participants
148 Participants
286 Participants
Number of Participants With Serious Adverse Events (SAEs), Treatment-Emergent Adverse Events (AEs), Deaths, AEs Leading to Discontinuation, Extra Pyramidal Syndrome (EPS)-Related AEs
Treatment-Emergent AEs Leading to Discontinuation
9 Participants
10 Participants
14 Participants
33 Participants
Number of Participants With Serious Adverse Events (SAEs), Treatment-Emergent Adverse Events (AEs), Deaths, AEs Leading to Discontinuation, Extra Pyramidal Syndrome (EPS)-Related AEs
Treatment-Emergent EPS-Related AEs
16 Participants
6 Participants
26 Participants
48 Participants

PRIMARY outcome

Timeframe: Baseline, Week 8, Week 26, Week 52

Population: Safety Sample - Observed Cases (OC) Data Set and Endpoint - Last Observation Carried Forward (LOCF)

The SAS is a 10-item instrument used to evaluate the presence and severity of parkinsonian symptomatology. It is the most commonly used rating scale for Parkinsonism in clinical trials over the past 25 years. The ten items focus on rigidity rather than bradykinesia, and do not assess subjective rigidity or slowness. Items are rated for severity on a 0-4 scale, with definitions given for each anchor point. The total SAS Score has a possible range from 10 to 50. Negative change scores indicate improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline in Total Simpson-Angus Scale (SAS) At Week 8, Week 26, and Week 52
Baseline (n=79, 63, 153, 295)
10.7 units on a scale
Standard Deviation 1.40
10.6 units on a scale
Standard Deviation 1.17
11.0 units on a scale
Standard Deviation 2.11
10.8 units on a scale
Standard Deviation 1.77
Mean Change From Baseline in Total Simpson-Angus Scale (SAS) At Week 8, Week 26, and Week 52
Change at Week 8 (n=77, 60, 145, 282)
-0.3 units on a scale
Standard Deviation 1.13
0.1 units on a scale
Standard Deviation 1.51
-0.4 units on a scale
Standard Deviation 2.12
-0.2 units on a scale
Standard Deviation 1.78
Mean Change From Baseline in Total Simpson-Angus Scale (SAS) At Week 8, Week 26, and Week 52
Change at Week 26 (n=61, 47, 126, 234)
-0.2 units on a scale
Standard Deviation 1.59
0.2 units on a scale
Standard Deviation 1.42
-0.6 units on a scale
Standard Deviation 1.96
-0.3 units on a scale
Standard Deviation 1.79
Mean Change From Baseline in Total Simpson-Angus Scale (SAS) At Week 8, Week 26, and Week 52
Change at Week 52 (n=48, 36, 91, 175)
-0.6 units on a scale
Standard Deviation 1.44
0.3 units on a scale
Standard Deviation 1.80
-0.5 units on a scale
Standard Deviation 1.82
-0.3 units on a scale
Standard Deviation 1.75
Mean Change From Baseline in Total Simpson-Angus Scale (SAS) At Week 8, Week 26, and Week 52
Change at Endpoint (LOCF) (n=79, 63, 153, 295)
-0.2 units on a scale
Standard Deviation 1.70
0.3 units on a scale
Standard Deviation 1.59
-0.3 units on a scale
Standard Deviation 2.06
-0.1 units on a scale
Standard Deviation 1.89

PRIMARY outcome

Timeframe: Baseline, Week 8, Week 26, Week 52

Population: Safety Sample - Observed Cases (OC) Data Set and Endpoint - Last Observation Carried Forward (LOCF)

The AIMS is an assessment of movement dysfunctions. It is a 12-item instrument assessing abnormal involuntary movements associated with antipsychotic drugs and 'spontaneous' motor disturbance related to the illness itself. Scoring the AIMS consists of rating the severity of movement in 3 main anatomic areas (facial/oral, extremities, and trunk), based on a five-point scale (0=none, 4=severe). The AIMS Total Score has a possible range from 0 to 28. Negative change scores indicate improvement in movement dysfunction.

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline in Abnormal Involuntary Movement Scale (AIMS) At Week 8, Week 26, and Week 52
Baseline (n=79, 67, 155, 301)
0.5 units on a scale
Standard Deviation 1.90
0.5 units on a scale
Standard Deviation 1.02
0.3 units on a scale
Standard Deviation 1.23
0.4 units on a scale
Standard Deviation 1.40
Mean Change From Baseline in Abnormal Involuntary Movement Scale (AIMS) At Week 8, Week 26, and Week 52
Change at Week 8 (n=78, 64, 149, 291)
-0.3 units on a scale
Standard Deviation 1.45
-0.1 units on a scale
Standard Deviation 0.87
-0.1 units on a scale
Standard Deviation 1.15
-0.2 units on a scale
Standard Deviation 1.18
Mean Change From Baseline in Abnormal Involuntary Movement Scale (AIMS) At Week 8, Week 26, and Week 52
Change at Week 26 (n=61, 49, 130, 240)
-0.4 units on a scale
Standard Deviation 1.37
-0.0 units on a scale
Standard Deviation 1.30
-0.2 units on a scale
Standard Deviation 1.26
-0.2 units on a scale
Standard Deviation 1.30
Mean Change From Baseline in Abnormal Involuntary Movement Scale (AIMS) At Week 8, Week 26, and Week 52
Change at Week 52 (n=48, 36, 93, 177)
-0.4 units on a scale
Standard Deviation 1.44
-0.0 units on a scale
Standard Deviation 1.00
-0.1 units on a scale
Standard Deviation 1.13
-0.2 units on a scale
Standard Deviation 1.20
Mean Change From Baseline in Abnormal Involuntary Movement Scale (AIMS) At Week 8, Week 26, and Week 52
Change at Endpoint (LOCF) (n=79, 67, 155, 301)
-0.3 units on a scale
Standard Deviation 1.33
0.0 units on a scale
Standard Deviation 1.44
-0.1 units on a scale
Standard Deviation 1.32
-0.1 units on a scale
Standard Deviation 1.35

PRIMARY outcome

Timeframe: Baseline, Week 8, Week 26, Week 52

Population: Safety Sample - Observed Cases (OC) Data Set and Endpoint - Last Observation Carried Forward (LOCF)

The Barnes Akathisia Rating Scale is a 4-item scale to assess presence and severity of drug-induced akathisia, including both objective items and subjective items, together with a global clinical assessment of akathisia. Global assessment is made on a scale of 0 to 5 with comprehensive definitions provided for each anchor point on scale: 0=absent; 1=questionable; 2=mild akathisia; 3=moderate akathisia; 4=marked akathisia; 5=severe akathisia. Score has a possible range from 0 (absent) to 5 (severe akathisia). Negative change scores indicate improvement in akathisia.

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline in Barnes Akathisia Global Clinical Assessment at Week 8, Week 26, Week 52, and Endpoint
Baseline (n=79, 64, 154, 297)
0.1 units on a scale
Standard Deviation 0.57
0.2 units on a scale
Standard Deviation 0.54
0.1 units on a scale
Standard Deviation 0.41
0.1 units on a scale
Standard Deviation 0.49
Mean Change From Baseline in Barnes Akathisia Global Clinical Assessment at Week 8, Week 26, Week 52, and Endpoint
Change at Week 8 (n=78, 60, 149, 287)
-0.1 units on a scale
Standard Deviation 0.55
-0.1 units on a scale
Standard Deviation 0.50
0.0 units on a scale
Standard Deviation 0.35
0.0 units on a scale
Standard Deviation 0.44
Mean Change From Baseline in Barnes Akathisia Global Clinical Assessment at Week 8, Week 26, Week 52, and Endpoint
Change at Week 26 (n=61, 47, 129, 237)
-0.1 units on a scale
Standard Deviation 0.64
-0.1 units on a scale
Standard Deviation 0.48
0.0 units on a scale
Standard Deviation 0.41
0.0 units on a scale
Standard Deviation 0.50
Mean Change From Baseline in Barnes Akathisia Global Clinical Assessment at Week 8, Week 26, Week 52, and Endpoint
Change at Week 52 (n=48, 35, 93, 176)
-0.1 units on a scale
Standard Deviation 0.37
0.0 units on a scale
Standard Deviation 0.49
0.1 units on a scale
Standard Deviation 0.56
0.0 units on a scale
Standard Deviation 0.50
Mean Change From Baseline in Barnes Akathisia Global Clinical Assessment at Week 8, Week 26, Week 52, and Endpoint
Change at Endpoint (LOCF) (n=79, 64, 154, 297)
0.0 units on a scale
Standard Deviation 0.67
0.0 units on a scale
Standard Deviation 0.52
0.1 units on a scale
Standard Deviation 0.58
0.0 units on a scale
Standard Deviation 0.59

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 8, Week 26, Week 52

Population: Safety Sample

Abnormal metabolic criterion values include the following: fasting serum glucose ≥115 mg/dL; non-fasting serum glucose ≥ 200 mg/dL; total cholesterol ≥240 mg/dL; LDL cholesterol ≥160 mg/dL; HDL cholesterol ≤30 mg/dL; triglycerides ≥120 mg/dL (females), ≥160 mg/dL (males)

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Glucose, Fasting Serum
1 Participants
1 Participants
5 Participants
7 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Glucose, Serum
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, HDL Fasting
4 Participants
3 Participants
7 Participants
14 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, HDL Non-Fasting
4 Participants
3 Participants
10 Participants
17 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, HDL Random
7 Participants
6 Participants
16 Participants
29 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, LDL Fasting
0 Participants
0 Participants
2 Participants
2 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, LDL Non-Fasting
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, LDL Random
0 Participants
0 Participants
2 Participants
2 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, Total Fasting
1 Participants
0 Participants
1 Participants
2 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, Total Non-Fasting
0 Participants
0 Participants
1 Participants
1 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Cholesterol, Total Random
1 Participants
0 Participants
2 Participants
3 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Triglycerides, Fasting
10 Participants
8 Participants
20 Participants
38 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Triglycerides, Non-Fasting
15 Participants
12 Participants
33 Participants
60 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Metabolic Abnormalities
Triglycerides, Random
22 Participants
20 Participants
47 Participants
89 Participants

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 8, Week 26, Week 52

Population: Safety Sample

Abnormal hematology criterion values include the following: hematocrit (ages 6-17, males \& females) ≤33%; hemoglobin (ages 6-17, males \& females) \<11.3 g/dL; leukocytes ≤2800 c/mm3 or ≥16000 c/mm3; eosinophils (ages 6-17, males \& females) \>17%; neutrophils \<15%; platelet count ≤75,000 c/mm3 or ≥700,000 c/mm3

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Hematology Abnormalities
Hematocrit
0 Participants
0 Participants
2 Participants
2 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Hematology Abnormalities
Hemoglobin
0 Participants
2 Participants
3 Participants
5 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Hematology Abnormalities
Leukocytes
2 Participants
0 Participants
3 Participants
5 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Hematology Abnormalities
Platelet Count
0 Participants
1 Participants
0 Participants
1 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Hematology Abnormalities
Eosinophils, relative
0 Participants
1 Participants
3 Participants
4 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Hematology Abnormalities
Neutrophils, relative
1 Participants
0 Participants
0 Participants
1 Participants

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 8, Week 26, Week 52

Population: Safety Sample

Abnormal chemistry criterion values include the following: aspartate aminotransferase ≥3x upper limit of normal (ULN); alanine aminotransferase ≥3x ULN; alkaline phosphatase ≥3x ULN; lactate dehydrogenase ≥3x ULN; creatinine ≥2.0 mg/dL; uric acid, ≥10.5 mg/dL (male), ≥8.5 mg/dL (female); bilirubin (Total) ≥2.0 mg/dL; serum prolactin \>ULN; creatine kinase ≥3x ULN; blood urea nitrogen ≥30 mg/dL; sodium ≤126 mEq/L or ≥156 mEq/L; potassium ≤2.5 mEq/L or ≥ 6.5 mEq/L; chloride ≤90 mEq/L or ≥118 mEq/L; calcium ≤8.2 mg/dL or ≥12 mg/dL

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Alanine Aminotransferase
3 Participants
2 Participants
5 Participants
10 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Aspartate Aminotransferase
0 Participants
0 Participants
2 Participants
2 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Alkaline Phosphatase
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Prolactin
2 Participants
0 Participants
0 Participants
2 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Lactate Dehydrogenase
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Creatinine
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Uric Acid
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Bilirubin, Total
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Creatine Kinase
2 Participants
4 Participants
7 Participants
13 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Blood Urea Nitrogen
0 Participants
1 Participants
1 Participants
2 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Sodium, Serum
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Potassium, Serum
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Chloride, Serum
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Laboratory Chemistry Abnormalities
Calcium, Total
0 Participants
0 Participants
0 Participants
0 Participants

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 8, Week 26, Week 52

Population: Safety Sample

These abbreviations are used in the table of ECG measurements: supraventricular (SV), baseline (BL), 1 degree (1°), atrioventricular (A-V), intraventricular (IVT), symmetrical (SYM), corrected QT interval (QTc). QRS complex is a recording of a single heartbeat on ECG corresponding to the depolarization of the right and left ventricles. PR interval is measured from beginning of P wave to beginning of QRS complex. QT interval is a measure of time between start of Q wave and end of T wave in the heart's electrical cycle. ↑ = increase from baseline, ↓ = decrease from baseline, → = "to"

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Right Bundle Branch Block (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Sinus Tachycardia (≥140 bpm & ↑ ≥15 bpm)
2 Participants
0 Participants
2 Participants
4 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Sinus Bradycardia (≤50 bpm and ↓ ≥15 bpm)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
SV Premature Beat (≥2per10sec & ↑ over BL)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Ventricular Premature Beat(≥1per10sec & ↑ over BL)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
SV Tachycardia (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Ventricular Tachycardia (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Atrial Fibrillation (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Atrial Flutter (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
1° A-V Block (PR ≥ 0.20 sec and ↑ ≥ 0.05 sec)
0 Participants
0 Participants
1 Participants
1 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
2° A-V block (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
3° A-V block (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Left Bundle Branch Block (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Pre-Excitation Syndrome (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Other ITV Block (QRS ≥ 0.10 sec & ↑ 0.02 sec)
0 Participants
0 Participants
3 Participants
3 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Old Infarction (not present-present at ≥12 weeks)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Acute/Subacute Infarction (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Myocardial Ischemia (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
SYM T-Wave Inversion (not present → present)
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
QTcB interval (>475 msec & elevation 10% over BL)
1 Participants
0 Participants
2 Participants
3 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
QTcF interval (>475 msec & elevation 10% over BL)
1 Participants
0 Participants
0 Participants
1 Participants
Number of Participants With Potentially Clinically Relevant Eletrocardiograph (ECG) Abnormalities
Other Abnormality
3 Participants
3 Participants
6 Participants
12 Participants

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 0 (Baseline), Week 1, Week 2, Week 4, Week 8, Week 14, Week 20, Week 26, Week 34, Week 42, Week 52 (Endpoint)

Population: Safety Sample

Clinically significantly abnormal vital signs met age-appropriate (heart rate cohorts: ages 5-14 \& ages 15+; blood pressure cohorts: ages 6-12 \& ages 13-17) criterion AND represented change from pretreatment value of at least the following magnitudes: systolic blood pressure (≥130 mmHg \& ≥144 mmHg w/ increase of ≥20 mmHg) or (≤117 mmHg \& ≤120 mmHg w/ decrease of ≥20 mmHg); diastolic blood pressure (≥86 mmHg \& ≥92 mmHg w/ increase of ≥15 mmHg) or (≤75 mm Hg \& ≤80 mmHg w/ decrease of ≥15 mmHg); heart rate (140 bpm and 120 bpm w/ increase of ≥15 bpm) or (50 bpm and 50 bpm w/ decrease of ≥15 bpm).

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Supine Systolic Blood Pressure Increase
5 Participants
2 Participants
18 Participants
25 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Supine Systolic Blood Pressure Decrease
14 Participants
15 Participants
27 Participants
56 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Standing Systolic Blood Pressure Increase
6 Participants
7 Participants
13 Participants
26 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Standing Systolic Blood Pressure Decrease
16 Participants
22 Participants
25 Participants
63 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Sitting Systolic Blood Pressure Increase
3 Participants
1 Participants
6 Participants
10 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Sitting Systolic Blood Pressure Decrease
11 Participants
8 Participants
13 Participants
32 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Supine Diastolic Blood Pressure Increase
6 Participants
5 Participants
11 Participants
22 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Supine Diastolic Blood Pressure Decrease
15 Participants
19 Participants
38 Participants
72 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Standing Diastolic Blood Pressure Increase
3 Participants
8 Participants
17 Participants
28 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Standing Diastolic Blood Pressure Decrease
22 Participants
20 Participants
36 Participants
78 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Sitting Diastolic Blood Pressure Increase
2 Participants
2 Participants
1 Participants
5 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Sitting Diastolic Blood Pressure Decrease
14 Participants
8 Participants
9 Participants
31 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Supine Heart Rate Increase
1 Participants
1 Participants
2 Participants
4 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Supine Heart Rate Decrease
0 Participants
0 Participants
0 Participants
0 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Standing Heart Rate Increase
5 Participants
3 Participants
4 Participants
12 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Standing Heart Rate Decrease
0 Participants
0 Participants
1 Participants
1 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Sitting Heart Rate Increase
1 Participants
0 Participants
1 Participants
2 Participants
Number of Potentially Clinically Relevant Vital Sign Abnormalities
Sitting Heart Rate Decrease
0 Participants
0 Participants
0 Participants
0 Participants

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 0 (Baseline), Week 1, Week 2, Week 4, Week 8, Week 14, Week 20, Week 26, Week 34, Week 42, Week 52 (Endpoint)

Population: Safety Sample - Observed Cases (OC) Data Set and Endpoint - Last Observation Carried Forward (LOCF)

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline in Patient Weight
Change at Week 4 (n=80, 67, 165, 312)
0.8 kg
Standard Deviation 1.26
0.5 kg
Standard Deviation 1.60
1.2 kg
Standard Deviation 1.43
1.0 kg
Standard Deviation 1.45
Mean Change From Baseline in Patient Weight
Baseline (n=84, 69, 169, 322)
42.5 kg
Standard Deviation 23.17
45.1 kg
Standard Deviation 20.37
45.4 kg
Standard Deviation 21.79
44.6 kg
Standard Deviation 21.83
Mean Change From Baseline in Patient Weight
Change at Week 1 (n=78, 58, 149, 285)
0.2 kg
Standard Deviation 0.76
-0.1 kg
Standard Deviation 1.06
0.4 kg
Standard Deviation 0.94
0.3 kg
Standard Deviation 0.94
Mean Change From Baseline in Patient Weight
Change at Week 2 (n=81, 67, 165, 313)
0.2 kg
Standard Deviation 0.99
0.0 kg
Standard Deviation 1.47
0.8 kg
Standard Deviation 1.15
0.5 kg
Standard Deviation 1.23
Mean Change From Baseline in Patient Weight
Change at Week 8 (n=77, 65, 149, 291)
1.6 kg
Standard Deviation 1.99
1.5 kg
Standard Deviation 2.29
1.9 kg
Standard Deviation 2.09
1.8 kg
Standard Deviation 2.11
Mean Change From Baseline in Patient Weight
Change at Week 14 (n=71, 55, 142, 268)
3.3 kg
Standard Deviation 2.81
2.8 kg
Standard Deviation 2.79
3.1 kg
Standard Deviation 2.80
3.1 kg
Standard Deviation 2.80
Mean Change From Baseline in Patient Weight
Change at Week 20 (n=66, 50, 136, 252)
4.0 kg
Standard Deviation 3.39
3.3 kg
Standard Deviation 4.73
4.2 kg
Standard Deviation 3.34
4.0 kg
Standard Deviation 3.67
Mean Change From Baseline in Patient Weight
Change at Week 26 (n=61, 49, 130, 240)
5.0 kg
Standard Deviation 4.41
4.7 kg
Standard Deviation 4.36
4.9 kg
Standard Deviation 3.99
4.9 kg
Standard Deviation 4.16
Mean Change From Baseline in Patient Weight
Change at Week 34 (n=60, 46, 123, 229)
6.0 kg
Standard Deviation 5.80
5.8 kg
Standard Deviation 4.90
6.1 kg
Standard Deviation 4.68
6.0 kg
Standard Deviation 5.02
Mean Change From Baseline in Patient Weight
Change at Week 42 (n=58, 43, 115, 216)
7.0 kg
Standard Deviation 6.46
6.4 kg
Standard Deviation 5.90
7.1 kg
Standard Deviation 5.11
6.9 kg
Standard Deviation 5.64
Mean Change From Baseline in Patient Weight
Change at Week 52 (n=48, 36, 94, 178)
8.7 kg
Standard Deviation 6.77
7.7 kg
Standard Deviation 6.44
7.9 kg
Standard Deviation 5.90
8.1 kg
Standard Deviation 6.23
Mean Change From Baseline in Patient Weight
Change at Endpoint (LOCF) (n=84, 69, 169, 322)
6.3 kg
Standard Deviation 6.71
5.5 kg
Standard Deviation 5.50
6.6 kg
Standard Deviation 5.72
6.3 kg
Standard Deviation 5.94

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 0 (Baseline), Week 1, Week 2, Week 4, Week 8, Week 14, Week 20, Week 26, Week 34, Week 42, Week 52 (Endpoint)

Population: Safety Sample. Subjects included in the period evaluation had a baseline and at least 1 measurement within the time period that was assessed. Patients are only counted once in each time period but can appear in multiple time periods. For those with multiple records in 1 time period, only the last record in that period is included in calculations.

The Z-Score indicates how many standard deviations a person is from the population norm values. The body weight z-scores are designed to take into account the amount of weight gain that would be expected due to normal growth in children and adolescents. The body weight z-scores are by age and gender standardized values (corresponding to a normal distribution with mean 0 and a standard deviation of 1) of the actual weight measurements, based on the Growth Charts provided by the Centers for Disease Control (CDC; see Links section of this record).

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline by Time Period in Body Weight Z-Score
Baseline (n=84, 70, 169, 323)
0.47 Standard Deviations away from Population
Standard Deviation 1.687
0.95 Standard Deviations away from Population
Standard Deviation 1.112
0.98 Standard Deviations away from Population
Standard Deviation 1.392
0.84 Standard Deviations away from Population
Standard Deviation 1.434
Mean Change From Baseline by Time Period in Body Weight Z-Score
<= 3 Months (n=84, 70, 169, 323)
0.13 Standard Deviations away from Population
Standard Deviation 0.283
0.10 Standard Deviations away from Population
Standard Deviation 0.204
0.09 Standard Deviations away from Population
Standard Deviation 0.300
0.10 Standard Deviations away from Population
Standard Deviation 0.277
Mean Change From Baseline by Time Period in Body Weight Z-Score
3-6 Months (n=73, 56, 145, 274)
0.24 Standard Deviations away from Population
Standard Deviation 0.433
0.26 Standard Deviations away from Population
Standard Deviation 0.331
0.20 Standard Deviations away from Population
Standard Deviation 0.327
0.22 Standard Deviations away from Population
Standard Deviation 0.359
Mean Change From Baseline by Time Period in Body Weight Z-Score
6-9 Months (n=63, 49, 131, 243)
0.31 Standard Deviations away from Population
Standard Deviation 0.570
0.28 Standard Deviations away from Population
Standard Deviation 0.367
0.23 Standard Deviations away from Population
Standard Deviation 0.407
0.26 Standard Deviations away from Population
Standard Deviation 0.448
Mean Change From Baseline by Time Period in Body Weight Z-Score
>9 Months (n=59, 44, 115, 218)
0.33 Standard Deviations away from Population
Standard Deviation 0.580
0.23 Standard Deviations away from Population
Standard Deviation 0.399
0.24 Standard Deviations away from Population
Standard Deviation 0.465
0.26 Standard Deviations away from Population
Standard Deviation 0.486

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 0 (Baseline), Week 1, Week 2, Week 4, Week 8, Week 14, Week 20, Week 26, Week 34, Week 42, Week 52 (Endpoint)

Population: Safety Sample: Endpoint - Last Observation Carried Forward (LOCF)

The body mass index (BMI) is a statistical measurement which compares a person's weight and height. Though it does not actually measure the percentage of body fat, it is used to estimate a healthy body weight based on subject height.

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline in Patient Body Mass Index (BMI)
Baseline (n=84, 69, 169, 322)
20.1 kg/m2
Standard Deviation 6.31
21.0 kg/m2
Standard Deviation 5.15
21.6 kg/m2
Standard Deviation 6.38
21.1 kg/m2
Standard Deviation 6.13
Mean Change From Baseline in Patient Body Mass Index (BMI)
Change at Endpoint (LOCF) (n=84, 69, 169, 322)
1.7 kg/m2
Standard Deviation 2.56
1.4 kg/m2
Standard Deviation 2.07
1.8 kg/m2
Standard Deviation 2.51
1.7 kg/m2
Standard Deviation 2.43

PRIMARY outcome

Timeframe: At screening (up to 42 days prior to treatment start), Week 0 (Baseline), Week 1, Week 2, Week 4, Week 8, Week 14, Week 20, Week 26, Week 34, Week 42, Week 52 (Endpoint)

Population: Safety Sample. Subjects included in the period evaluation had a baseline and at least 1 measurement within the time period that was assessed. Patients are only counted once in each time period but can appear in multiple time periods. For those with multiple records in 1 time period, only the last record in that period is included in calculations.

The Z-Score indicates how many standard deviations a person is from the population norm values. The BMI z-scores are designed to take into account the BMI that would be expected due to normal growth in children and adolescents. The BMI z-scores are by age and gender standardized values (corresponding to a normal distribution with mean 0 and a standard deviation of 1) of the actual BMI measurements, based on the Growth Charts provided by the Centers for Disease Control (CDC; see Links section of this record).

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline By Time Period in BMI Z-Score
Baseline (n=84, 70, 169, 323)
0.44 Standard Deviations away from Population
Standard Deviation 1.591
0.98 Standard Deviations away from Population
Standard Deviation 0.997
1.01 Standard Deviations away from Population
Standard Deviation 1.236
0.86 Standard Deviations away from Population
Standard Deviation 1.313
Mean Change From Baseline By Time Period in BMI Z-Score
<=3 Months (n=84, 70, 169, 323)
0.17 Standard Deviations away from Population
Standard Deviation 0.436
0.12 Standard Deviations away from Population
Standard Deviation 0.268
0.09 Standard Deviations away from Population
Standard Deviation 0.500
0.12 Standard Deviations away from Population
Standard Deviation 0.443
Mean Change From Baseline By Time Period in BMI Z-Score
3-6 Months (n=73, 56, 145, 274)
0.29 Standard Deviations away from Population
Standard Deviation 0.625
0.28 Standard Deviations away from Population
Standard Deviation 0.388
0.18 Standard Deviations away from Population
Standard Deviation 0.549
0.23 Standard Deviations away from Population
Standard Deviation 0.543
Mean Change From Baseline By Time Period in BMI Z-Score
6-9 Months (n=62, 49, 129, 240)
0.36 Standard Deviations away from Population
Standard Deviation 0.647
0.24 Standard Deviations away from Population
Standard Deviation 0.438
0.18 Standard Deviations away from Population
Standard Deviation 0.643
0.24 Standard Deviations away from Population
Standard Deviation 0.611
Mean Change From Baseline By Time Period in BMI Z-Score
>9 Months (n=59, 44, 115, 218)
0.33 Standard Deviations away from Population
Standard Deviation 0.768
0.15 Standard Deviations away from Population
Standard Deviation 0.476
0.14 Standard Deviations away from Population
Standard Deviation 0.775
0.19 Standard Deviations away from Population
Standard Deviation 0.725

SECONDARY outcome

Timeframe: Week 0 (Baseline), Week 52 (Endpoint, LOCF)

Population: Efficacy Sample, LOCF. The Efficacy Sample includes all patients that had a baseline and at least one post-baseline measurement of the same scale. A total of 322 patients met this criterion based on CGI-S evaluations.

CGI scale is a global evaluation of improvement over time. CGI-Severity (CGI-S) is a clinician-rated assessment that evaluates the severity of a patient's condition on a 7-point scale ranging from 1 (no symptoms) to 7 (very severe symptoms). CGI-Severity score is from 1 to 7. A negative change score signifies improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=84 Participants
As previously described in Participant Flow
Rollover Placebo
n=69 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=169 Participants
As previously described in Participant Flow
Total
n=322 Participants
Mean Change From Baseline in Clinical Global Impression (CGI)-Severity Score at Week 52 (Endpoint, LOCF)
Baseline
4.8 units on a scale
Standard Deviation 0.99
4.2 units on a scale
Standard Deviation 0.99
3.9 units on a scale
Standard Deviation 1.05
4.2 units on a scale
Standard Deviation 1.08
Mean Change From Baseline in Clinical Global Impression (CGI)-Severity Score at Week 52 (Endpoint, LOCF)
Change at Endpoint (LOCF)
-0.8 units on a scale
Standard Deviation 0.85
-0.4 units on a scale
Standard Deviation 1.06
-0.0 units on a scale
Standard Deviation 1.01
-0.3 units on a scale
Standard Deviation 1.04

SECONDARY outcome

Timeframe: Week 52 (Endpoint, LOCF)

Population: Efficacy Sample. The Efficacy Sample includes all patients that had a baseline and at least one post-baseline measurement of the same scale. A total of 322 patients met this criterion based on CGI-S evaluations.

CGI scale is a global evaluation of improvement over time. CGI-Improvement (CGI-I) is a clinician rated assessment that evaluates improvement relative to symptoms at baseline on a a 7-point scale ranging from 1 (very much improved) to 7 (very much worse). CGI-I Score is from 1 to 7. A lower score signifies larger improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=84 Participants
As previously described in Participant Flow
Rollover Placebo
n=69 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=169 Participants
As previously described in Participant Flow
Total
n=322 Participants
CGI-Improvement Score at Week 52 (Endpoint, LOCF)
2.7 units on a scale
Standard Deviation 1.30
2.4 units on a scale
Standard Deviation 1.24
2.5 units on a scale
Standard Deviation 1.20
2.5 units on a scale
Standard Deviation 1.23

SECONDARY outcome

Timeframe: Week 0 (Baseline), Week 52 (Endpoint, LOCF)

Population: Efficacy Sample, LOCF. The Efficacy Sample includes all patients that had a baseline and at least one post-baseline measurement of the same scale. A total of 322 patients met this criterion based on CGI-S evaluations. Of those, 8 patients had baseline ABC (all subscales) evaluations but no post-baseline.

The ABC is an informant-based symptom checklist for assessing and classifying problem behaviors of children and adolescents with mental retardation. The 58 items are rated on a 4-point scale (0 = not at all a problem to 3 = the problem is severe in degree), and resolve into 5 subscales: (1) irritability and agitation; (2) lethargy and social withdrawal; (3) stereotypic behavior; (4) hyperactivity and noncompliance, and (5) inappropriate speech. ABC Irritability Subscale Score is from 0 to 45. A negative change signifies improvement

Outcome measures

Outcome measures
Measure
De Novo
n=80 Participants
As previously described in Participant Flow
Rollover Placebo
n=68 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=166 Participants
As previously described in Participant Flow
Total
n=314 Participants
Mean Change From Baseline in Aberrant Behavior Checklist (ABC) Irritability Score at Week 52 (Endpoint, LOCF)
Baseline
23.2 units on a scale
Standard Deviation 8.88
21.5 units on a scale
Standard Deviation 9.82
15.0 units on a scale
Standard Deviation 9.20
18.5 units on a scale
Standard Deviation 9.96
Mean Change From Baseline in Aberrant Behavior Checklist (ABC) Irritability Score at Week 52 (Endpoint, LOCF)
Change at Endpoint (LOCF)
-6.5 units on a scale
Standard Deviation 11.12
-6.1 units on a scale
Standard Deviation 11.25
0.7 units on a scale
Standard Deviation 9.72
-2.6 units on a scale
Standard Deviation 10.98

SECONDARY outcome

Timeframe: Week 0 (Baseline), Week 52 (Endpoint, LOCF)

Population: Efficacy Sample, LOCF.The Efficacy Sample includes all patients that had a baseline and at least one post-baseline measurement of the same scale. A total of 322 patients met this criterion based on CGI-S evaluations. Of those, 8 patients had baseline ABC (all subscales) evaluations but no post-baseline.

The ABC is an informant-based symptom checklist for assessing and classifying problem behaviors of children and adolescents with mental retardation. The 58 items are rated on a 4-point scale (0 = not at all a problem to 3 = the problem is severe in degree), and resolve into 5 subscales: (1) irritability and agitation; (2) lethargy and social withdrawal; (3) stereotypic behavior; (4) hyperactivity and noncompliance, and (5) inappropriate speech. ABC Hyperactivity Subscale Score is from 0 to 48. A negative change score signifies improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=80 Participants
As previously described in Participant Flow
Rollover Placebo
n=68 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=166 Participants
As previously described in Participant Flow
Total
n=314 Participants
Mean Change From Baseline in ABC Hyperactivity Subscale Score at Week 52 (Endpoint, LOCF)
Baseline
28.4 units on a scale
Standard Deviation 10.87
25.8 units on a scale
Standard Deviation 13.18
18.4 units on a scale
Standard Deviation 12.03
22.5 units on a scale
Standard Deviation 12.79
Mean Change From Baseline in ABC Hyperactivity Subscale Score at Week 52 (Endpoint, LOCF)
Change at Endpoint (LOCF)
-10.0 units on a scale
Standard Deviation 10.55
-8.3 units on a scale
Standard Deviation 10.85
0.3 units on a scale
Standard Deviation 11.18
-4.2 units on a scale
Standard Deviation 11.92

SECONDARY outcome

Timeframe: Week 0 (Baseline), Week 52 (Endpoint, LOCF)

Population: Efficacy Sample, LOCF. The Efficacy Sample includes all patients that had a baseline and at least one post-baseline measurement of the same scale. A total of 322 patients met this criterion based on CGI-S evaluations. Of those, 8 patients had baseline ABC (all subscales) evaluations but no post-baseline.

The ABC is an informant-based symptom checklist for assessing and classifying problem behaviors of children and adolescents with mental retardation. The 58 items are rated on a 4-point scale (0 = not at all a problem to 3 = the problem is severe in degree), and resolve into 5 subscales: (1) irritability and agitation; (2) lethargy and social withdrawal; (3) stereotypic behavior; (4) hyperactivity and noncompliance, and (5) inappropriate speech. ABC Stereotypy Subscale Score is from 0 to 21. A negative change score signifies improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=80 Participants
As previously described in Participant Flow
Rollover Placebo
n=68 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=166 Participants
As previously described in Participant Flow
Total
n=314 Participants
Change From Baseline in ABC Stereotypy Subscale Score at Week 52 (Endpoint, LOCF)
Baseline
8.1 units on a scale
Standard Deviation 5.18
8.1 units on a scale
Standard Deviation 5.57
6.4 units on a scale
Standard Deviation 5.46
7.2 units on a scale
Standard Deviation 5.46
Change From Baseline in ABC Stereotypy Subscale Score at Week 52 (Endpoint, LOCF)
Change at Endpoint (LOCF)
-2.5 units on a scale
Standard Deviation 4.67
-1.9 units on a scale
Standard Deviation 4.46
0.1 units on a scale
Standard Deviation 4.58
-1.0 units on a scale
Standard Deviation 4.71

SECONDARY outcome

Timeframe: Week 0 (Baseline), Week 52 (Endpoint, LOCF)

Population: Efficacy Sample, LOCF. The Efficacy Sample includes all patients that had a baseline and at least one post-baseline measurement of the same scale. A total of 322 patients met this criterion based on CGI-S evaluations. Of those, 8 patients had baseline ABC (all subscales) evaluations but no post-baseline.

The ABC is an informant-based symptom checklist for assessing and classifying problem behaviors of children and adolescents with mental retardation. The 58 items are rated on a 4-point scale (0 = not at all a problem to 3 = the problem is severe in degree), and resolve into 5 subscales: (1) irritability and agitation; (2) lethargy and social withdrawal; (3) stereotypic behavior; (4) hyperactivity and noncompliance, and (5) inappropriate speech. ABC Social Withdrawal Subscale Score is from 0 to 48. A negative change score signifies improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=80 Participants
As previously described in Participant Flow
Rollover Placebo
n=68 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=166 Participants
As previously described in Participant Flow
Total
n=314 Participants
Mean Change From Baseline in ABC Social Withdrawal Scale At Week 52 (Endpoint, LOCF)
Baseline
14.6 units on a scale
Standard Deviation 8.62
11.3 units on a scale
Standard Deviation 9.24
10.4 units on a scale
Standard Deviation 8.86
11.7 units on a scale
Standard Deviation 9.03
Mean Change From Baseline in ABC Social Withdrawal Scale At Week 52 (Endpoint, LOCF)
Change at Endpoint (LOCF)
-5.4 units on a scale
Standard Deviation 9.07
-3.0 units on a scale
Standard Deviation 6.96
-1.8 units on a scale
Standard Deviation 6.09
-3.0 units on a scale
Standard Deviation 7.28

SECONDARY outcome

Timeframe: Week 0 (Baseline), Week 52 (Endpoint, LOCF)

Population: Efficacy Sample, LOCF

The ABC is an informant-based symptom checklist for assessing and classifying problem behaviors of children and adolescents with mental retardation. The 58 items are rated on a 4-point scale (0 = not at all a problem to 3 = the problem is severe in degree), and resolve into 5 subscales: (1) irritability and agitation; (2) lethargy and social withdrawal; (3) stereotypic behavior; (4) hyperactivity and noncompliance, and (5) inappropriate speech. ABC Inappropriate Speech Subscale score is from 1 to 12. A negative change score signifies improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=86 Participants
As previously described in Participant Flow
Rollover Placebo
n=70 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=174 Participants
As previously described in Participant Flow
Total
n=330 Participants
Mean Change From Baseline in ABC Inappropriate Speech Subscale Score at Week 52 (Endpoint, LOCF)
Baseline
5.8 units on a scale
Standard Deviation 3.15
5.7 units on a scale
Standard Deviation 4.23
4.2 units on a scale
Standard Deviation 3.61
4.9 units on a scale
Standard Deviation 3.72
Mean Change From Baseline in ABC Inappropriate Speech Subscale Score at Week 52 (Endpoint, LOCF)
Change at Endpoint (LOCF)
-1.9 units on a scale
Standard Deviation 2.66
-1.8 units on a scale
Standard Deviation 2.94
-0.3 units on a scale
Standard Deviation 2.50
-1.0 units on a scale
Standard Deviation 2.75

SECONDARY outcome

Timeframe: Week 0 (Baseline), Week 52 (Endpoint, LOCF)

Population: Efficacy Sample, LOCF. The Efficacy Sample includes all patients that had a baseline and at least one post-baseline measurement of the same scale. A total of 322 patients met this criterion based on CGI-S evaluations. Of those, 96 patients did not have post-baseline evaluations for CY-BOCS, and were excluded from the efficacy analyses.

CY-BOCS is a 10-item, clinician-rated scale designed to measure the severity of obsessive-compulsive symptoms in patients below the age of 18. The scale contains 5 items pertaining to obsessions (which were not used in this trial) and 5 items pertaining to compulsions, which rated each symptom domain in terms of time spent, interference with functioning, distress, resistance, and control. Each item was rated on a 5-point scale, from 0 (no symptoms or minimum severity) to 4 (extreme symptoms or maximum severity). CY-BOCS Score is from 0 to 20. A negative change score signifies improvement.

Outcome measures

Outcome measures
Measure
De Novo
n=62 Participants
As previously described in Participant Flow
Rollover Placebo
n=49 Participants
As previously described in Participant Flow
Rollover Aripiprazole
n=115 Participants
As previously described in Participant Flow
Total
n=226 Participants
Change From Baseline in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) Score at Week 52 (Endpoint, LOCF)
Baseline
12.6 units on a scale
Standard Deviation 4.60
12.1 units on a scale
Standard Deviation 3.96
10.4 units on a scale
Standard Deviation 3.87
11.4 units on a scale
Standard Deviation 4.20
Change From Baseline in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) Score at Week 52 (Endpoint, LOCF)
Change at Endpoint (LOCF)
-2.0 units on a scale
Standard Deviation 3.68
-2.4 units on a scale
Standard Deviation 5.06
0.2 units on a scale
Standard Deviation 3.81
-0.9 units on a scale
Standard Deviation 4.23

Adverse Events

De Novo

Serious events: 3 serious events
Other events: 73 other events
Deaths: 0 deaths

Roll Over Aripiprazole

Serious events: 5 serious events
Other events: 136 other events
Deaths: 0 deaths

Roll Over Placebo

Serious events: 1 serious events
Other events: 61 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
De Novo
n=86 participants at risk
Roll Over Aripiprazole
n=174 participants at risk
Roll Over Placebo
n=70 participants at risk
Psychiatric disorders
AGGRESSION
1.2%
1/86
0.57%
1/174
0.00%
0/70
Psychiatric disorders
SUICIDAL IDEATION
1.2%
1/86
0.00%
0/174
0.00%
0/70
Psychiatric disorders
IMPULSIVE BEHAVIOUR
0.00%
0/86
0.57%
1/174
0.00%
0/70
Hepatobiliary disorders
CHOLECYSTITIS
0.00%
0/86
0.57%
1/174
0.00%
0/70
Hepatobiliary disorders
CHOLELITHIASIS
0.00%
0/86
0.57%
1/174
0.00%
0/70
Nervous system disorders
CONVULSION
0.00%
0/86
0.00%
0/174
1.4%
1/70
Gastrointestinal disorders
DYSPHAGIA
0.00%
0/86
0.57%
1/174
0.00%
0/70
Infections and infestations
SINUSITIS
0.00%
0/86
0.57%
1/174
0.00%
0/70
Infections and infestations
SKIN INFECTION
1.2%
1/86
0.00%
0/174
0.00%
0/70
Infections and infestations
OTITIS MEDIA ACUTE
0.00%
0/86
0.57%
1/174
0.00%
0/70
Infections and infestations
PHARYNGOTONSILLITIS
0.00%
0/86
0.57%
1/174
0.00%
0/70
Respiratory, thoracic and mediastinal disorders
ASTHMA
0.00%
0/86
0.57%
1/174
0.00%
0/70

Other adverse events

Other adverse events
Measure
De Novo
n=86 participants at risk
Roll Over Aripiprazole
n=174 participants at risk
Roll Over Placebo
n=70 participants at risk
Investigations
WEIGHT INCREASED
23.3%
20/86
23.0%
40/174
22.9%
16/70
Psychiatric disorders
TIC
0.00%
0/86
1.1%
2/174
8.6%
6/70
Psychiatric disorders
ANXIETY
3.5%
3/86
5.7%
10/174
0.00%
0/70
Psychiatric disorders
INSOMNIA
9.3%
8/86
9.8%
17/174
11.4%
8/70
Psychiatric disorders
AGITATION
9.3%
8/86
6.3%
11/174
2.9%
2/70
Psychiatric disorders
AGGRESSION
9.3%
8/86
8.6%
15/174
8.6%
6/70
Nervous system disorders
DROOLING
3.5%
3/86
9.2%
16/174
4.3%
3/70
Nervous system disorders
HEADACHE
8.1%
7/86
10.3%
18/174
10.0%
7/70
Nervous system disorders
LETHARGY
8.1%
7/86
1.1%
2/174
1.4%
1/70
Nervous system disorders
SEDATION
9.3%
8/86
5.2%
9/174
14.3%
10/70
Nervous system disorders
DYSKINESIA
5.8%
5/86
1.7%
3/174
0.00%
0/70
Nervous system disorders
SOMNOLENCE
4.7%
4/86
1.7%
3/174
8.6%
6/70
Gastrointestinal disorders
NAUSEA
3.5%
3/86
1.1%
2/174
5.7%
4/70
Gastrointestinal disorders
VOMITING
19.8%
17/86
19.5%
34/174
15.7%
11/70
Gastrointestinal disorders
DIARRHOEA
8.1%
7/86
8.6%
15/174
11.4%
8/70
Gastrointestinal disorders
CONSTIPATION
7.0%
6/86
4.0%
7/174
5.7%
4/70
Infections and infestations
SINUSITIS
8.1%
7/86
5.7%
10/174
2.9%
2/70
Infections and infestations
EAR INFECTION
3.5%
3/86
8.6%
15/174
2.9%
2/70
Infections and infestations
NASOPHARYNGITIS
14.0%
12/86
12.6%
22/174
14.3%
10/70
Infections and infestations
GASTROENTERITIS VIRAL
5.8%
5/86
4.6%
8/174
1.4%
1/70
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
12.8%
11/86
9.2%
16/174
15.7%
11/70
Renal and urinary disorders
ENURESIS
5.8%
5/86
3.4%
6/174
5.7%
4/70
Metabolism and nutrition disorders
DECREASED APPETITE
7.0%
6/86
4.0%
7/174
2.9%
2/70
Metabolism and nutrition disorders
INCREASED APPETITE
18.6%
16/86
10.9%
19/174
11.4%
8/70
Skin and subcutaneous tissue disorders
RASH
5.8%
5/86
4.0%
7/174
4.3%
3/70
Respiratory, thoracic and mediastinal disorders
COUGH
11.6%
10/86
8.6%
15/174
8.6%
6/70
Respiratory, thoracic and mediastinal disorders
EPISTAXIS
9.3%
8/86
5.7%
10/174
4.3%
3/70
Respiratory, thoracic and mediastinal disorders
NASAL CONGESTION
11.6%
10/86
5.7%
10/174
0.00%
0/70
Respiratory, thoracic and mediastinal disorders
RESPIRATORY TRACT CONGESTION
1.2%
1/86
1.1%
2/174
5.7%
4/70
General disorders
FATIGUE
8.1%
7/86
5.2%
9/174
10.0%
7/70
General disorders
PYREXIA
7.0%
6/86
13.2%
23/174
14.3%
10/70
General disorders
IRRITABILITY
4.7%
4/86
5.7%
10/174
1.4%
1/70

Additional Information

BMS Study Director

Bristol-Myers Squibb

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