A Phase 3 interventional study of Brexpiprazole and Placebo in Irritability Associated With Autism Spectrum Disorder (ASD), sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc.. Completed at 1 site in United States. Open to participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2025-08-28.
Sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc. · Phase 3, Interventional, and Treatment
The purpose of this study is to find out about the potential benefits and safety of brexpiprazole in children and adolescent participants, aged 5 to 17, with irritability associated with autism spectrum disorder.
1,344 studies on the registry are indexed under Autistic Disorder; 334 are open to participants now.
This study's enrollment of 119 is above the median of 45 across 1,044 interventional studies indexed under Autistic Disorder.
Browse Autistic Disorder studies →Otsuka Pharmaceutical Development & Commercialization, Inc. is the lead sponsor of 289 studies on the registry; 18 are open to participants now.
Of its 104 completed or terminated interventional studies of FDA-regulated products, 69 (66%) have results posted.
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Key Inclusion Criteria:
Key Exclusion Criteria:
Participants received flexible doses of brexpiprazole 0.25 to 3 milligram per day (mg/day), orally, once daily (QD) up to Week 8. For participants with body weight \< 50 kilograms (kg) the dose was titrated up from 0.25 mg/day on Days 1 to 3, followed by 0.5 mg on Days 4 to 7, and to 1 mg on Days 8 to 14. Based on the investigator's judgment the dose was increased to 1.5 mg/day after Day 15. The dose was fixed after Week 6 and administration continued for another 2 weeks until Week 8. For participants with body weight ≥ 50 kg the dose was titrated up from 0.5 mg/day on Days 1 to 3, followed by 1.5 mg on Days 4 to 7, and to 2 mg on Days 8 to 14. Based on the investigator's judgment the dose was increased to 3 mg/day after Day 15. The dose was fixed after Week 6 and administration continued for another 2 weeks until Week 8.
Drug: Brexpiprazole
Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8.
Drug: Placebo
Oral tablet; take once daily
Also known as: brexpiprazole, OPC-34712 and LuAF41156
Administered orally daily for up to Week 8.
Mean Change From Baseline to Week 8 in Aberrant Behavior Checklist - Irritability (ABC-I) Subscale Score
The ABC is a parent-reported rating scale designed to assess treatment effects on problem behavior in participants with intellectual disabilities. The ABC scale has 58 items, which divide into 5 subscales as follows: (1) Irritability, Agitation; (2) Lethargy, Social Withdrawal; (3) Stereotypic Behavior; (4) Hyperactivity, Noncompliance; and (5) Inappropriate Speech. Each of the 58 ABC items is rated on a 4-point scale (0=not at all a problem; 1=the behavior is a problem, but slight in degree;2=the problem is moderately serious; 3=the problem is severe in degree). ABC-I measures emotional and behavioral symptoms of ASD, including aggression toward others, deliberate self-injuriousness, temper tantrums, and quickly changing moods. ABC-I total score is the sum of the ratings over 15 ABC items. Individual scores were summed, thus the ABC-I total score ranges from 0 to 45. Higher scores represent the worst condition. A negative change from baseline indicates improvement.
Time frame: Baseline to Week 8
Mean Change From Baseline to Week 8 in Clinical Global Impression - Severity (CGI-S) Score
The CGI-S scale is a clinician-rated assessment that evaluates the severity of a participant's condition with a focus on symptoms of irritability on a 7-point scale. The investigator (or rater) answered the following question: "Considering your total clinical experience with this particular population, how ill was the participant at this time with regard to symptoms of irritability?" Response choices were 0 = not assessed; 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; and 7 = among the most extremely ill patients. Higher scores indicate more severe illness. A negative change from baseline indicates improvement.
Time frame: Baseline to Week 8
Participants took part in the study at 40 sites in the United States from 30 October 2019 to 9 September 2022.
| Milestone | Brexpiprazole | Placebo |
|---|---|---|
| Started | 60 | 59 |
| Randomized sample | 60 | 59 |
| Safety sample | 58 | 57 |
| Efficacy sample | 58 | 56 |
| Completed | 52 | 52 |
| Not completed | 8 | 7 |
| Withdrew: Lost to follow-up | 1 | 2 |
| Withdrew: Adverse event | 2 | 1 |
| Withdrew: Subject withdrew consent to participate | 1 | 0 |
| Withdrew: Subject was withdrawn from participation by parent/guardian | 2 | 1 |
| Withdrew: Protocol deviation | 1 | 0 |
| Withdrew: Lack of efficacy | 0 | 2 |
| Withdrew: Disease relapse | 1 | 0 |
| Withdrew: Reason not specified | 0 | 1 |
The ABC is a parent-reported rating scale designed to assess treatment effects on problem behavior in participants with intellectual disabilities. The ABC scale has 58 items, which divide into 5 subscales as follows: (1) Irritability, Agitation; (2) Lethargy, Social Withdrawal; (3) Stereotypic Behavior; (4) Hyperactivity, Noncompliance; and (5) Inappropriate Speech. Each of the 58 ABC items is rated on a 4-point scale (0=not at all a problem; 1=the behavior is a problem, but slight in degree;2=the problem is moderately serious; 3=the problem is severe in degree). ABC-I measures emotional and behavioral symptoms of ASD, including aggression toward others, deliberate self-injuriousness, temper tantrums, and quickly changing moods. ABC-I total score is the sum of the ratings over 15 ABC items. Individual scores were summed, thus the ABC-I total score ranges from 0 to 45. Higher scores represent the worst condition. A negative change from baseline indicates improvement.
| score on a scale | Brexpiprazole | Placebo |
|---|---|---|
| Mean Change From Baseline to Week 8 in Aberrant Behavior Checklist - Irritability (ABC-I) Subscale Score | -10.1 ± 1.28 | -8.87 ± 1.25 |
The CGI-S scale is a clinician-rated assessment that evaluates the severity of a participant's condition with a focus on symptoms of irritability on a 7-point scale. The investigator (or rater) answered the following question: "Considering your total clinical experience with this particular population, how ill was the participant at this time with regard to symptoms of irritability?" Response choices were 0 = not assessed; 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; and 7 = among the most extremely ill patients. Higher scores indicate more severe illness. A negative change from baseline indicates improvement.
| score on a scale | Brexpiprazole | Placebo |
|---|---|---|
| Mean Change From Baseline to Week 8 in Clinical Global Impression - Severity (CGI-S) Score | -1.16 ± 0.15 | -1.09 ± 0.15 |
Collected over From first dose through 21 days after last dose of study drug (up to Week 11). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Brexpiprazole | 0/58 (0%) | 0/58 (0%) | 13/58 (22.4%) |
| Placebo | 0/57 (0%) | 0/57 (0%) | 5/57 (8.8%) |
| Event | Brexpiprazole | Placebo |
|---|---|---|
| SomnolenceNervous system disorders | 7/58 | 3/57 |
| HeadacheNervous system disorders | 6/58 | 1/57 |
| VomitingGastrointestinal disorders | 3/58 | 1/57 |
Randomized sample included all participants who were randomized into the trial.
| Age, Continuous(years) | Brexpiprazole | Placebo | Total |
|---|---|---|---|
| Mean | 9.8 ± 2.9 | 10.0 ± 3.2 | 9.9 ± 3.0 |
| Sex: Female, Male(Participants) | Brexpiprazole | Placebo | Total |
|---|---|---|---|
| Female | 6 | 9 | 15 |
| Male | 54 | 50 | 104 |
| Ethnicity (NIH/OMB)(Participants) | Brexpiprazole | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 8 | 7 | 15 |
| Not Hispanic or Latino | 52 | 52 | 104 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race/Ethnicity, Customized(Participants) | Brexpiprazole | Placebo | Total |
|---|---|---|---|
| Race — White | 49 | 45 | 94 |
| Race — Black or African American | 3 | 10 | 13 |
| Race — American Indian or Alaska Native | 0 | 0 | 0 |
| Race — Asian | 2 | 3 | 5 |
| Race — Native Hawaiian or Other Pacific Islander | 1 | 0 | 1 |
| Race — Other | 5 | 1 | 6 |
| Region of Enrollment(participants) | Brexpiprazole | Placebo | Total |
|---|---|---|---|
| United States | 60 | 59 | 119 |
| Aberrant Behavior Checklist - Irritability (ABC-I) Subscale Score(score on a scale) | Brexpiprazole | Placebo | Total |
|---|---|---|---|
| Mean | 29.7 ± 8.0 | 28.6 ± 7.6 | 29.1 ± 7.8 |
| Clinical Global Impression - Severity (CGI-S) Score(score on a scale) | Brexpiprazole | Placebo | Total |
|---|---|---|---|
| Mean | 4.9 ± 0.7 | 4.7 ± 0.5 | 4.8 ± 0.7 |
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Otsuka Pharmaceutical Development & Commercialization, Inc.