Brexpiprazole in Treatment of Children and Adolescents With Irritability Associated With Autism Spectrum Disorder

NCT04174365 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: 331-201-00148
• Study Type: interventional
• Target: 119
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Irritability Associated With Autism Spectrum Disorder (ASD)

Keywords

Autism; Irritability; Autism Spectrum Disorder; ASD

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline to Week 8

Secondary Outcomes (1)

• Mean Change From Baseline to Week 8 in Clinical Global Impression - Severity (CGI-S) Score

  Baseline to Week 8

Study Arms (2)

Brexpiprazole

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8.

Drug: Placebo

Interventions

Brexpiprazole DRUG

Oral tablet; take once daily

Also known as: brexpiprazole, OPC-34712 and LuAF41156

Brexpiprazole

Placebo DRUG

Administered orally daily for up to Week 8.

Placebo

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Primary Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) diagnosis of ASD
• Aberrant Behavior checklist - Irritability (ABC-I) subscale score of ≥ 18
• Clinical Global Impressions - Severity (CGI-S) scale score pertaining to irritability ≥ 4
• Mental age of ≥ 2 years as determined by Investigator based upon school participation, social history or medical records
• Ability for parent/caregiver to follow all protocol procedures
• Able to swallow tablets
• Able to discontinue all prohibited concomitant medications to meet protocol required washouts prior to and during the trial period

You may not qualify if:

• current or historical diagnosis of Fragile-X Syndrome or Rett's Disorder
• history of neuroleptic malignant syndrome
• a significant risk of committing violent acts, serious self-harm, or suicide
• epilepsy, a history of seizures, or a history of severe head trauma or stroke, or have a history or current evidence of other unstable medical conditions
• current hypothyroidism or hyperthyroidism
• uncontrolled Type I or Type II diabetes
• uncontrolled hypertension or symptomatic hypotension, or orthostatic hypotension
• Weight \< 15 kg
• Previous exposure to brexpiprazole
• Sexually active males or females, who could become pregnant, not agreeing to practice 2 different methods of birth control or remain abstinent during the trial and for 30 days at the end of the study

Sponsors & Collaborators

• Otsuka Pharmaceutical Development & Commercialization, Inc.: lead
• H. Lundbeck A/S: collaborator

Study Sites (1)

For additional information regarding sites

Princeton, New Jersey, 08540, United States

Location

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Interventions

brexpiprazole

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Global Clinical Development
• Organization: Otsuka Pharmaceutical Development & Commercialization, Inc.

clinicaltransparency@otsuka-us.com

1-609-524-6788

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2019
• First Posted: November 22, 2019
• Study Start: October 30, 2019
• Primary Completion: August 8, 2022
• Study Completion: September 9, 2022
• Last Updated: August 28, 2025
• Results First Posted: August 28, 2025

Record last verified: 2025-08

Locations

US (1)