Trial Outcomes & Findings for Post-Marketing Surveillance Study of Aripiprazole in Patients With Autism (NCT NCT03179787)
NCT ID: NCT03179787
Last Updated: 2021-09-16
Results Overview
Using caregiver-rated ABC-J, the degree of aberrant behaviors was scored on four levels ranging from 0 'not at all a problem', 1 'slight problem', 2 'moderately serious problem' to 3 'the problem is severe in degree' in irritability subscale (including 15 items describing symptoms of irritability, such as temper tantrums, aggression, mood changes, and self-injury). The irritability subscale score ranging from 0 to 45 was derived from the sum of the 15 items and higher values represent a worse outcome.
Recruitment status
COMPLETED
Target enrollment
528 participants
Primary outcome timeframe
Baseline, Week 4, 8, 16, 24, 52
Results posted on
2021-09-16
Participant Flow
This surveillance was conducted in 528 participants from 100 trial sites in Japan.
Patients newly treated with aripiprazole for irritability associated with autism spectrum disorder (ASD) in children and adolescents (≥6 years and \<18 years) were included.
Participant milestones
| Measure |
Safety Analysis Group
Five hundred and twenty-eight patients were enrolled at 100 sites across Japan, and 526 case reports were collected during the period of April 2017 to September 2019. The safety analysis population consisted of 510 patients. Among excluded patients, 15 patients were lost to follow-up, and one patient was not treated with aripiprazole.
|
|---|---|
|
Overall Study
STARTED
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528
|
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Overall Study
COMPLETED
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510
|
|
Overall Study
NOT COMPLETED
|
18
|
Reasons for withdrawal
| Measure |
Safety Analysis Group
Five hundred and twenty-eight patients were enrolled at 100 sites across Japan, and 526 case reports were collected during the period of April 2017 to September 2019. The safety analysis population consisted of 510 patients. Among excluded patients, 15 patients were lost to follow-up, and one patient was not treated with aripiprazole.
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|---|---|
|
Overall Study
Lost to Follow-up
|
15
|
|
Overall Study
Case report failures
|
2
|
|
Overall Study
Treatment not administered
|
1
|
Baseline Characteristics
Post-Marketing Surveillance Study of Aripiprazole in Patients With Autism
Baseline characteristics by cohort
| Measure |
Safety Analysis Group
n=510 Participants
Five hundred and twenty-eight patients were enrolled at 100 sites across Japan, and 526 case reports were collected during the period of April 2017 to September 2019. The safety analysis population consisted of 510 patients. Among excluded patients, 15 patients were lost to follow-up, and one patient was not treated with aripiprazole.
|
|---|---|
|
Age, Categorical
<=18 years
|
510 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
10.4 years
STANDARD_DEVIATION 3.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
134 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
376 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
510 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Japan
|
510 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 4, 8, 16, 24, 52Population: Twenty-one patients with the protocol deviations (including all effectiveness measures were not assessed after treatment: n = 11, all effectiveness measures were assessed over 7 days after last dose: n = 7, the daily dose was \> 15 mg: n = 2, all effectiveness were assessed after day 393: n = 1) were excluded, and then efficacy sample consisted of all participants who have Baseline and at least one Post-Baseline effectiveness evaluation.
Using caregiver-rated ABC-J, the degree of aberrant behaviors was scored on four levels ranging from 0 'not at all a problem', 1 'slight problem', 2 'moderately serious problem' to 3 'the problem is severe in degree' in irritability subscale (including 15 items describing symptoms of irritability, such as temper tantrums, aggression, mood changes, and self-injury). The irritability subscale score ranging from 0 to 45 was derived from the sum of the 15 items and higher values represent a worse outcome.
Outcome measures
| Measure |
Safety Analysis Group
n=396 Participants
Five hundred and twenty-eight patients were enrolled at 100 sites across Japan, and 526 case reports were collected during the period of April 2017 to September 2019. The safety analysis population consisted of 510 patients. Among excluded patients, 15 patients were lost to follow-up, and one patient was not treated with aripiprazole.
|
|---|---|
|
Mean Changes of Aberrant Behavior Checklist-Japanese Version (ABC-J) Irritability Subscale Scores From Baseline.
Baseline
|
19.8 score on a scale
Standard Deviation 9.5
|
|
Mean Changes of Aberrant Behavior Checklist-Japanese Version (ABC-J) Irritability Subscale Scores From Baseline.
Week 4
|
14.7 score on a scale
Standard Deviation 8.8
|
|
Mean Changes of Aberrant Behavior Checklist-Japanese Version (ABC-J) Irritability Subscale Scores From Baseline.
Week 8
|
12.9 score on a scale
Standard Deviation 8.1
|
|
Mean Changes of Aberrant Behavior Checklist-Japanese Version (ABC-J) Irritability Subscale Scores From Baseline.
Week 16
|
12.7 score on a scale
Standard Deviation 8.3
|
|
Mean Changes of Aberrant Behavior Checklist-Japanese Version (ABC-J) Irritability Subscale Scores From Baseline.
Week 24
|
13.4 score on a scale
Standard Deviation 8.9
|
|
Mean Changes of Aberrant Behavior Checklist-Japanese Version (ABC-J) Irritability Subscale Scores From Baseline.
Week 52
|
13.1 score on a scale
Standard Deviation 9.1
|
|
Mean Changes of Aberrant Behavior Checklist-Japanese Version (ABC-J) Irritability Subscale Scores From Baseline.
End-point (LOCF)
|
13.0 score on a scale
Standard Deviation 9.0
|
Adverse Events
Safety Analysis Group
Serious events: 3 serious events
Other events: 82 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Safety Analysis Group
n=510 participants at risk
Five hundred and twenty-eight patients were enrolled at 100 sites across Japan, and 526 case reports were collected during the period of April 2017 to September 2019. The safety analysis population consisted of 510 patients. Among excluded patients, 15 patients were lost to follow-up, and one patient was not treated with aripiprazole.
|
|---|---|
|
Nervous system disorders
Epilepsy
|
0.20%
1/510 • Number of events 1 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Nervous system disorders
Partial seizures
|
0.20%
1/510 • Number of events 1 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Renal and urinary disorders
Renal impairment
|
0.20%
1/510 • Number of events 1 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Psychiatric disorders
Insomnia
|
0.20%
1/510 • Number of events 1 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Psychiatric disorders
Impulsive behaviour
|
0.20%
1/510 • Number of events 1 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
Other adverse events
| Measure |
Safety Analysis Group
n=510 participants at risk
Five hundred and twenty-eight patients were enrolled at 100 sites across Japan, and 526 case reports were collected during the period of April 2017 to September 2019. The safety analysis population consisted of 510 patients. Among excluded patients, 15 patients were lost to follow-up, and one patient was not treated with aripiprazole.
|
|---|---|
|
Nervous system disorders
Somnolence
|
9.4%
48/510 • Number of events 48 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Investigations
Weight increased
|
3.5%
18/510 • Number of events 18 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Nervous system disorders
Headache
|
1.6%
8/510 • Number of events 8 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Gastrointestinal disorders
Nausea
|
1.4%
7/510 • Number of events 7 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Metabolism and nutrition disorders
Increased appetite
|
1.2%
6/510 • Number of events 6 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
|
Metabolism and nutrition disorders
Obesity
|
0.98%
5/510 • Number of events 5 • Adverse events were observed from first dose until follow-up for 52 weeks.
The adverse events (if any occurred), onset date, seriousness, outcome, date of outcome, causality to aripiprazole, other possible causal factors, and treatment for adverse events during the observation period were recorded. Seriousness was determined by the physicians.
|
Additional Information
Section Chief of Pharmacovigilance
Otsuka Pharmaceutical Co., Ltd.
Phone: +81-3-6717-1400
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place