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.

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.