Cariprazine in the Treatment of Pediatric Patients With Irritability Associated With Autism Spectrum Disorder: A Randomized Controlled Trial
In brief
Cariprazine improved irritability in 69% of autistic youth, versus 38% on placebo
In an 8-week randomized trial of 155 youth ages 5 to 17, 68.9% taking cariprazine were rated much or very much improved in irritability, compared with 38.0% taking placebo; irritability scores also improved more with the drug. About half had treatment-emergent side effects, most often sleepiness or headache, and longer-term safety and benefit remain unknown.
- Journal
- Journal of the American Academy of Child and Adolescent Psychiatry (Q1)
- Published
- 22 October 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Robert L Findling, Durim Bozhdaraj, Walt Duffy, Jeffrey R Strawn, Manpreet K Singh, Marc S Weinberg, et al.
- PMID
- 42914007
- DOI
- 10.1016/j.jaac.2026.10.017
Why clinicians should know about it
- Picked for Pediatrics and Child Health (paper of the day, 24 October 2026): Paediatric autism treatment trial with clinical efficacy and safety outcomes
Abstract
OBJECTIVE: To evaluate the efficacy and safety of cariprazine in youth with irritability associated with autism spectrum disorder (ASD). METHOD: This 8-week, multicenter, randomized, double-blind, controlled trial compared age- and weight-dependent flexibly-dosed cariprazine (0.75-3 mg/day) versus placebo in youth (5-17y) with ASD and irritability. The primary efficacy endpoint was week 8 change from baseline in caregiver-rated Aberrant Behavior Checklist-Irritability subscale (ABC-I) score. The first key secondary endpoint was the week 8 proportion of participants who were responders for clinician-rated Clinical Global Impressions-Change (CGI-C) in irritability ("much improved" or "very much improved"). RESULTS: 155 participants (mean age 10.6y, 70% male participants) received ≥1 study dose; 152 had baseline and ≥1 postbaseline ABC-I or CGI-C scores (n=74 cariprazine, n=78 placebo), with 81% completing the treatment period and 3.9% discontinuing due to treatment-emergent adverse events (TEAEs). The cariprazine group had significantly greater week 8 ABC-I improvement than placebo (least square mean difference [95% CI] = -3.7 [-6.75, -0.57], Cohen's d effect size = 0.42, p = 0.0205). A significantly greater percentage of cariprazine-treated participants were CGI-C Irritability responders at week 8 than placebo (68.9% vs. 38.0%, odds ratio [95% CI] = 3.6 [1.7, 7.8], p = 0.0012). Overall, 51% of participants had TEAEs (1.3% had severe TEAEs); most commonly, somnolence and headache. TEAEs, laboratory values, and vital signs were consistent with cariprazine's known adult safety profile. CONCLUSION: Pediatric patients with ASD treated with cariprazine demonstrated significantly greater improvement in irritability than placebo.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.