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

NCT ID: NCT04174365

Last Updated: 2025-08-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 119 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-10-30
• Study Completion Date: 2022-09-09

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)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Brexpiprazole

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.

Group Type: EXPERIMENTAL

Brexpiprazole

Intervention Type: DRUG

Oral tablet; take once daily

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Administered orally daily for up to Week 8.

Interventions

Brexpiprazole

Oral tablet; take once daily

Intervention Type: DRUG

Placebo

Administered orally daily for up to Week 8.

Intervention Type: DRUG

Other Intervention Names

brexpiprazole, OPC-34712 and LuAF41156

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• Primary diagnosis of bipolar I disorder, including any DSM-5 current diagnosis of bipolar II disorder, schizophrenia, schizoaffective disorder, major depressive episode, and post-traumatic stress disorder (PTSD). Attention-deficit/hyperactivity disorder (ADHD) maybe exclusionary if it is the primary disorder, or is not stable or adequately treated.
• 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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

H. Lundbeck A/S

INDUSTRY

Sponsor Role: collaborator

Otsuka Pharmaceutical Development & Commercialization, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

For additional information regarding sites

Princeton, New Jersey, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

331-201-00148

Identifier Type: -

Identifier Source: org_study_id