Cariprazine for Autism Spectrum Disorder

This trial tests the safety and effectiveness of cariprazine, an antipsychotic medication, for reducing irritability in children and teens with autism spectrum disorder (ASD). ASD often involves communication and behavior challenges, with many experiencing high irritability, including aggression and self-injury. Participants will receive either cariprazine or a placebo (a non-active treatment) for eight weeks, and neither they nor their doctors will know which one they receive. Ideal candidates for this trial have an ASD diagnosis and frequently experience irritability that disrupts daily life. Participants will attend regular clinic visits for checkups and to monitor any side effects. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potential new treatment for ASD.

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study doctors to understand how your current medications might interact with the trial.

Earlier studies found that children generally tolerated cariprazine well. Researchers used these results to determine appropriate doses for future pediatric studies. Most children did not experience serious side effects, indicating no major safety concerns.

Cariprazine is unique because it targets the dopamine D3 receptor, offering a novel mechanism of action compared to traditional autism spectrum disorder treatments, which often focus on serotonin or other neurotransmitter pathways. This specificity might help alleviate some of the core symptoms of autism with potentially fewer side effects. Researchers are excited because cariprazine could provide a new option for individuals who don't respond well to existing medications, offering hope for improved symptom management.

Research has shown that cariprazine, which participants in this trial may receive, may help reduce irritability in young people with autism spectrum disorder (ASD). In earlier studies, about 69% of those taking cariprazine were rated as "much improved" or "very much improved" in their symptoms, compared to only 38% improvement in those not taking the drug. Children and teens generally tolerated cariprazine well, with most side effects being mild or moderate. A common side effect was weight gain. These findings suggest that cariprazine could be a useful option for managing irritability in ASD.¹²³⁴⁶