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

NCT05439616 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: M21-465
• Study Type: interventional
• Target: 161
• Locations: 2 countries
• Sites: 36

Brief Summary

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.

Conditions

Autism Spectrum Disorder

Keywords

Autism Spectrum Disorder; Cariprazine; VRAYLAR; AGN-241780

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Percentage of Participants with responder status of "Very Much Improved" or "Much Improved" on the Clinical Global Impression- Change Irritability (CGI-C Irritability) Scale

  Week 8

• Change from Baseline in Parent-Rated Anxiety Scale for Youth with Autism Spectrum Disorder (PRAS-ASD) Total Score

  Baseline (Week 0) to Week 8

• Change from Baseline in Caregiver Strain Questionnaire Short Form 7-Item (CGSQ SF-7) total score

  Baseline (Week 0) to Week 8

Study Arms (2)

Cariprazine

EXPERIMENTAL

Participants will receive age-and weight dependent flexible doses of cariprazine once daily for 8-weeks.

Drug: Cariprazine

Placebo

PLACEBO COMPARATOR

Participants will receive placebo once daily for 8-weeks.

Drug: Placebo

Interventions

Cariprazine DRUG

Oral Capsules or Oral Solution

Also known as: VRAYLAR, AGN-241780

Cariprazine

Placebo DRUG

Oral capsules or Oral Solution

Placebo

Eligibility Criteria

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

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• AbbVie: lead

Study Sites (36)

Advanced Research Center /ID# 245049

Anaheim, California, 92805, United States

Location

Sun Valley Research Center /ID# 243133

Imperial, California, 92251-9401, United States

Location

CHOC Children's Hospital /ID# 245577

Orange, California, 92868-4203, United States

Location

Pacific Clinical Research Management Group /ID# 243019

Upland, California, 91786, United States

Location

D&H Doral Research Center-Doral /ID# 255457

Doral, Florida, 33122, United States

Location

Sarkis Clinical Trials /ID# 242868

Gainesville, Florida, 32607, United States

Location

Advanced Research Institute of Miami /ID# 243179

Homestead, Florida, 33030-4613, United States

Location

Sandhill Research LLC /ID# 245079

Lake Mary, Florida, 32746, United States

Location

Axcess Medical Research /ID# 244952

Loxahatchee Groves, Florida, 33470, United States

Location

G+C Research Group, LLC /ID# 252016

Miami, Florida, 33126-2018, United States

Location

Medical Research Group of Central Florida /ID# 243170

Orange City, Florida, 32763, United States

Location

K2 Medical Research - Orlando - South Orlando Avenue /ID# 248694

Orlando, Florida, 32751, United States

Location

APG Research, LLC /ID# 243130

Orlando, Florida, 32803, United States

Location

Nona Pediatric Center /ID# 245078

Orlando, Florida, 32829-8070, United States

Location

Asclepes Research Centers - Spring Hill /ID# 248682

Spring Hill, Florida, 34609-5692, United States

Location

D&H Tamarac Research Center /ID# 250436

Tamarac, Florida, 33321-2979, United States

Location

Atlanta Center for Medical Research /ID# 243124

Atlanta, Georgia, 30331, United States

Location

Atlanta Behavioral Research, LLC /ID# 243082

Dunwoody, Georgia, 30338, United States

Location

iResearch Savannah /ID# 254582

Savannah, Georgia, 31405, United States

Location

Alivation Research /ID# 242915

Lincoln, Nebraska, 68526-9474, United States

Location

Dr. Hosneara, M.D. LLC /ID# 252129

Jackson Heights, New York, 11372-3922, United States

Location

Richmond Behavioral Associates An Evolution Research Group Portfolio Company /ID# 259578

Staten Island, New York, 10314-1607, United States

Location

New Dawn Psychiatric Services PLLC /ID# 243697

Kinston, North Carolina, 28501-1603, United States

Location

Quest Therapeutics of Avon Lake /ID# 252013

Avon Lake, Ohio, 44012, United States

Location

Cincinnati Children's Hospital /ID# 243968

Cincinnati, Ohio, 45229, United States

Location

CincyScience /ID# 242993

West Chester, Ohio, 45069, United States

Location

BioBehavioral Research of Austin /ID# 255460

Austin, Texas, 78759, United States

Location

Relaro Medical Trials /ID# 243126

Dallas, Texas, 75243, United States

Location

Cedar Health Research /ID# 248683

Dallas, Texas, 75251-2202, United States

Location

AIM Trials /ID# 243120

Plano, Texas, 75093, United States

Location

Family Psychiatry of The Woodlands /ID# 242867

The Woodlands, Texas, 77381, United States

Location

Advanced Research Institute /ID# 243098

Ogden, Utah, 84405, United States

Location

Northwest Clinical Research Center /ID# 243083

Bellevue, Washington, 98007, United States

Location

Core Clinical Research /ID# 243084

Everett, Washington, 98201, United States

Location

Dr. Samuel Sanchez PSC /ID# 245739

Caguas, 00727, Puerto Rico

Location

GCM Medical Group PSC /ID# 245735

San Juan, 00917-3104, Puerto Rico

Location

Related Links

• Related Info

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

cariprazine

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• ABBVIE INC.

  AbbVie

  STUDY DIRECTOR

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: June 28, 2022
• First Posted: June 30, 2022
• Study Start: July 7, 2022
• Primary Completion: October 4, 2024
• Study Completion: October 4, 2024
• Last Updated: April 4, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
• Access Criteria: To learn more about the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/

Locations

US (34); PR (2)