Study of Cariprazine Oral Capsules or Solution to Assess Adverse Events and Change in Irritability Due to Autism Spectrum Disorder (ASD) in Participants Aged 5-17 Years With ASD

An 8-Week, Multicenter, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of Cariprazine in the Treatment of Pediatric Subjects (5 to 17 Years of Age) With Autism Spectrum Disorder

Autism spectrum disorder (ASD) consists of deficits in social, communication, and cognitive development, repetitive and stereotypic behaviors. Many ASD patients show notably high levels of irritability, including verbal and physical aggression, self injury, and/or property destruction. Autistic infants tend to avoid eye contact and show little interest in others. This study will assess how safe and effective cariprazine is in treating pediatric participants (5 to 17 years of age) with ASD. Adverse events and change in disease activity will be assessed.

Cariprazine is an investigational drug being developed for the treatment of irritability due to ASD. This study is double-blinded means that neither the participants nor the study doctors will know who will be given cariprazine and who will be given placebo (does not contain treatment drug). Study doctors put the participants in 1 of the 2 groups, called treatment arms. Each group receives a different treatment. There is a 1 in 2 chance that participants will be assigned to placebo. Approximately 152 participants diagnosed with ASD will be enrolled in approximately 40 sites globally.

Participants will receive oral capsules or oral solution of cariprazine or placebo once daily for 8-weeks and will undergo a 4-week safety follow-up period.

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, checking for side effects and completing questionnaires.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: Cariprazine; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 161
• Phase: Phase 3

Contacts and Locations

Study Locations

• Puerto Rico
  • Caguas, Puerto Rico, 00727
    • Dr. Samuel Sanchez PSC /ID# 245739
  • San Juan, Puerto Rico, 00917-3104
    • GCM Medical Group PSC /ID# 245735
• United States
  • California
    • Anaheim, California, United States, 92805
      • Advanced Research Center /ID# 245049
    • Imperial, California, United States, 92251-9401
      • Sun Valley Research Center /ID# 243133
    • Orange, California, United States, 92868-4203
      • CHOC Children's Hospital /ID# 245577
    • Upland, California, United States, 91786
      • Pacific Clinical Research Management Group /ID# 243019
  • Florida
    • Doral, Florida, United States, 33122
      • D&H Doral Research Center-Doral /ID# 255457
    • Gainesville, Florida, United States, 32607
      • Sarkis Clinical Trials /ID# 242868
    • Homestead, Florida, United States, 33030-4613
      • Advanced Research Institute of Miami /ID# 243179
    • Lake Mary, Florida, United States, 32746
      • Sandhill Research LLC /ID# 245079
    • Loxahatchee Groves, Florida, United States, 33470
      • Axcess Medical Research /ID# 244952
    • Miami, Florida, United States, 33126-2018
      • G+C Research Group, LLC /ID# 252016
    • Orange City, Florida, United States, 32763
      • Medical Research Group of Central Florida /ID# 243170
    • Orlando, Florida, United States, 32751
      • K2 Medical Research - Orlando - South Orlando Avenue /ID# 248694
    • Orlando, Florida, United States, 32803
      • APG Research, LLC /ID# 243130
    • Orlando, Florida, United States, 32829-8070
      • Nona Pediatric Center /ID# 245078
    • Spring Hill, Florida, United States, 34609-5692
      • Asclepes Research Centers - Spring Hill /ID# 248682
    • Tamarac, Florida, United States, 33321-2979
      • D&H Tamarac Research Center /ID# 250436
  • Georgia
    • Atlanta, Georgia, United States, 30331
      • Atlanta Center for Medical Research /ID# 243124
    • Dunwoody, Georgia, United States, 30338
      • Atlanta Behavioral Research, LLC /ID# 243082
    • Savannah, Georgia, United States, 31405
      • iResearch Savannah /ID# 254582
  • Nebraska
    • Lincoln, Nebraska, United States, 68526-9474
      • Alivation Research /ID# 242915
  • New York
    • Jackson Heights, New York, United States, 11372-3922
      • Dr. Hosneara, M.D. LLC /ID# 252129
    • Staten Island, New York, United States, 10314-1607
      • Richmond Behavioral Associates An Evolution Research Group Portfolio Company /ID# 259578
  • North Carolina
    • Kinston, North Carolina, United States, 28501-1603
      • New Dawn Psychiatric Services PLLC /ID# 243697
  • Ohio
    • Avon Lake, Ohio, United States, 44012
      • Quest Therapeutics of Avon Lake /ID# 252013
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital /ID# 243968
    • West Chester, Ohio, United States, 45069
      • CincyScience /ID# 242993
  • Texas
    • Austin, Texas, United States, 78759
      • BioBehavioral Research of Austin /ID# 255460
    • Dallas, Texas, United States, 75243
      • Relaro Medical Trials /ID# 243126
    • Dallas, Texas, United States, 75251-2202
      • Cedar Health Research /ID# 248683
    • Plano, Texas, United States, 75093
      • AIM Trials /ID# 243120
    • The Woodlands, Texas, United States, 77381
      • Family Psychiatry of The Woodlands /ID# 242867
  • Utah
    • Ogden, Utah, United States, 84405
      • Advanced Research Institute /ID# 243098
  • Washington
    • Bellevue, Washington, United States, 98007
      • Northwest Clinical Research Center /ID# 243083
    • Everett, Washington, United States, 98201
      • Core Clinical Research /ID# 243084

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participants at the time of screening must have a Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) diagnosis of ASD, confirmed by Kiddie Schedule for Affective Disorders and Schizophrenia-Present and Lifetime (K-SADS-PL) administered at screening (Visit 1) by a trained clinician.
• Participants must have an Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) subscale score ≥ 18 (Visits 1 and 2).
• Participants must have a Clinical Global Impressions - Severity - Irritability (CGI-S Irritability) score ≥ 4 at screening (Visits 1 and 2).

Exclusion Criteria:

• Participants with diagnosis of intellectual disability (Intelligence quotient < 25).
• No history of major depressive disorder, bipolar disorder, schizophrenia, schizoaffective disorder, schizophreniform disorder, brief psychotic disorder, or psychotic disorder due to another medical condition, as excluded by administration of Kiddie Schedule for Affective Disorders and Schizophrenia-Present and Lifetime (K-SADS-PL).
• History of ASD that is associated with Rett Disorder, Fragile-X Syndrome, or Childhood Disintegrative Disorder.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cariprazine; Participants will receive age-and weight dependent flexible doses of cariprazine once daily for 8-weeks.	Drug: Cariprazine; Oral Capsules or Oral Solution; Other Names: VRAYLAR; AGN-241780
Placebo Comparator: Placebo; Participants will receive placebo once daily for 8-weeks.	Drug: Placebo; Oral capsules or Oral Solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline in Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) Subscale Score	The Aberrant Behavior Checklist (ABC) - Community version is a 58-item, caregiver-rated scale designed to measure inappropriate and maladaptive behavior of people with developmental disabilities (including intellectual disability and ASD). The ABC has 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactivity and Inappropriate Speech). The caregiver rates the child's behavior from 0=not at all a problem to 3= the problem is severe in degree. The range for Irritability is 0 to 45. Higher scores indicate greater severity.	Baseline (Week 0) to Week 8

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants with responder status of "Very Much Improved" or "Much Improved" on the Clinical Global Impression- Change Irritability (CGI-C Irritability) Scale	The Clinical Global Impression - Change-Irritability (CGI-C Irritability) is a single-item rating scale that assesses change in the severity of a participant's irritability from the clinician's perspective. The CGI-C Irritability 7-point graded response scale (1 = "Very much improved," 2 = "Much improved," 3 = "Minimally improved," 4 = "No change," 5 = "Minimally worse," 6 = "Much worse," or 7 = "Very much worse").	Week 8
Change from Baseline in Parent-Rated Anxiety Scale for Youth with Autism Spectrum Disorder (PRAS-ASD) Total Score	PRAS-ASD is a 25-item caregiver-reported measure of anxiety in participants aged 3 to 17 with ASD. All items use a 4-point response scale: 0 = "None (not present);" 1 = "Mild (present sometimes, not a real problem);" 2 = "Moderate (often present and a problem);" 3 = "Severe (very frequent and a major problem)." Total score ranges from 0 to 75, with higher scores indicating greater severity of anxiety.	Baseline (Week 0) to Week 8
Change from Baseline in Caregiver Strain Questionnaire Short Form 7-Item (CGSQ SF-7) total score	CGSQ SF-7 is a caregiver-reported 7-item measure of self-reported strain that focuses on the impact of caring for a child with emotional and behavioral problems. Responses are scored on a 5-point scale (1 = "Not at all," 2 = "A little;" 3 = "Somewhat;" 4 = "Quite a bit;" and 5 "Very much") with higher scores indicating greater caregiver strain.	Baseline (Week 0) to Week 8

Collaborators and Investigators

• Sponsor: AbbVie
• Investigators: Study Director: ABBVIE INC., AbbVie

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): July 7, 2022
• Primary Completion (Actual): October 4, 2024
• Study Completion (Actual): October 4, 2024

Study Registration Dates

• First Submitted: June 28, 2022
• First Submitted That Met QC Criteria: June 28, 2022
• First Posted (Actual): June 30, 2022

Study Record Updates

• Last Update Posted (Actual): April 4, 2025
• Last Update Submitted That Met QC Criteria: April 2, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: Autism Spectrum Disorder; Cariprazine; VRAYLAR; AGN-241780
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autism Spectrum Disorder; Autistic Disorder; Child Development Disorders, Pervasive; Physiological Effects of Drugs; Central Nervous System Depressants; Tranquilizing Agents; Psychotropic Drugs; Antipsychotic Agents; Cariprazine
• Other Study ID Numbers: M21-465

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: AbbVie is committed to responsible clinical trial data sharing. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information.
• IPD Sharing Time Frame: For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
• IPD Sharing Access Criteria: To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No