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| Name | Class |
|---|---|
| H. Lundbeck A/S | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Placebo | Placebo Comparator | Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Brexpiprazole | Drug | Oral tablet; take once daily |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| For additional information regarding sites | Princeton | New Jersey | 08540 | United States |
A total of 260 participants were screened, of which 119 participants were randomized to receive brexpiprazole or placebo.
Participants took part in the study at 40 sites in the United States from 30 October 2019 to 9 September 2022.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 7, 2022 | Aug 26, 2025 |
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| Placebo | Drug | Administered orally daily for up to Week 8. |
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| Baseline to Week 8 |
| FG001 | Placebo | Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8. |
| Randomized Sample | Randomized Sample included all participants who were randomized into the trial. |
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| Safety Sample | Safety Sample included all enrolled participants who received at least 1 dose of brexpiprazole or placebo. |
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| Efficacy Sample | Efficacy sample included all randomized participants who took at least 1 dose of brexpiprazole or placebo and had baseline and at least 1 post-baseline assessment of the primary efficacy variable Aberrant Behavior Checklist - Irritability (ABC-I) subscale score during the double-blind treatment phase. |
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| COMPLETED |
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| NOT COMPLETED |
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Randomized sample included all participants who were randomized into the trial.
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| ID | Title | Description |
|---|---|---|
| BG000 | Brexpiprazole | Participants received flexible doses of brexpiprazole 0.25 to 3 mg/day, orally, QD up to Week 8. For participants with body weight < 50 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. |
| BG001 | Placebo | Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Aberrant Behavior Checklist - Irritability (ABC-I) Subscale Score | 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. 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). The Irritability subscale (ABC-I) measures emotional and behavioral symptoms of ASD. ABC-I total score is the sum of 15 ABC items, where the total score ranges from 0 to 45. | Randomized Sample included all participants who were randomized into the trial. 'Number analyzed' indicates the number of participants with data available for analysis at Baseline. | Mean | Standard Deviation | score on a scale |
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| Clinical Global Impression - Severity (CGI-S) Score | CGI-S scale is a clinician-rated assessment that evaluates the severity of irritation in a participant on a 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. | Randomized Sample included all participants who were randomized into the trial. | Mean | Standard Deviation | score on a scale |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Efficacy sample included all randomized participants who took at least 1 dose of brexpiprazole or placebo and had baseline and at least 1 post-baseline assessment of the primary efficacy variable ABC-I subscale score during the double-blind treatment phase. 'Overall number of participants analyzed' indicates the number of participants with data available for analysis at the specified time point. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to Week 8 |
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| Secondary | 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. | Efficacy sample included all randomized participants who took at least 1 dose of brexpiprazole or placebo and had baseline and at least 1 post-baseline assessment of the primary efficacy variable ABC-I subscale score during the double-blind treatment phase. 'Overall number of participants analyzed' indicates the number of participants with data available for analysis at the specified time point. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to Week 8 |
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From first dose through 21 days after last dose of study drug (up to Week 11)
Safety sample included all enrolled participants who received at least 1 dose of brexpiprazole or placebo.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Brexpiprazole | Participants received flexible doses of brexpiprazole 0.25 to 3 mg/day, orally, QD up to Week 8. For participants with body weight < 50 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. | 0 | 58 | 0 | 58 | 13 | 58 |
| EG001 | Placebo | Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8. | 0 | 57 | 0 | 57 | 5 | 57 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
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| Vomiting | Gastrointestinal disorders | 22.0 | Systematic Assessment |
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| Headache | Nervous system disorders | 22.0 | Systematic Assessment |
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| Somnolence | Nervous system disorders | 22.0 | Systematic Assessment |
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Sponsor reserves the right to review results publications prior to public release and can delay such publications for a period greater than 60 days but no more than 120 days from the date that the publication is submitted to the Sponsor for review. Sponsor can require changes to the publication to protect Sponsor's intellectual property rights and/or confidential information and reserves the right to limit publication timing and scope of data published based on the number of study locations.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Global Clinical Development | Otsuka Pharmaceutical Development & Commercialization, Inc. | 1-609-524-6788 | clinicaltransparency@otsuka-us.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 28, 2022 | Aug 26, 2025 | SAP_003.pdf |
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| ID | Term |
|---|---|
| D001321 | Autistic Disorder |
| D000067877 | Autism Spectrum Disorder |
| ID | Term |
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| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
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| C000591922 | brexpiprazole |
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| OG001 | Placebo | Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8. |
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