SUMMARY

• CAPLYTA tablets are not indicated for the treatment of autistic disorder in adults, children, or adolescents.
• An ongoing, phase 3, multicenter, randomized, double-blind (DB), placebo-controlled study (NCT06690398) is evaluating the efficacy and safety of CAPLYTA in pediatric patients aged 5 to 17 years with a primary diagnosis of irritability associated with autism spectrum disorder (ASD).¹ 
• An ongoing, phase 1b, multicenter, open-label, multiple-dose study (NCT06557902) is evaluating the safety, tolerability, and pharmacokinetics (PK) of CAPLYTA in pediatric patients aged 5 to <13 years with a primary clinical diagnosis of ASD with symptoms of irritability.² 
• A case study of two 18-year-old male patients reported the impact of CAPLYTA in intermittent explosive disorder in ASD.³ 

CLINICAL DATA

Use of CAPLYTA in Pediatric Patients

Phase 3 Study

An ongoing, phase 3, multicenter, randomized, DB, placebo-controlled study (NCT06690398) is evaluating the efficacy and safety of CAPLYTA in pediatric patients aged 5 to 17 years with a primary diagnosis of irritability associated with ASD. The study is planned in the following 3 phases: 14 days of screening; 6 weeks of DB treatment where patients are randomized 1:1:1 to receive CAPLYTA high dose (42 mg), CAPLYTA low dose (21 mg), or placebo orally once daily; and 1 week of safety follow-up at ~1 week after receiving the last dose of the study drug.¹ 

Currently, only patients aged 13 to 17 years meeting the following key criteria are eligible for enrollment¹:

• ASD diagnosed on the basis of the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition Text Revision (DSM-5-TR), and confirmed using the Kiddie Schedule for Affective Disorders and Schizophrenia for School-Age Children-Present and Lifetime Version (K-SADS-PL)
• Aberrant Behavior Checklist - Irritability (ABC-I) subscale score >18 at screening and baseline
• Clinical Global Impression - Severity (CGI-S) score >4 with respect to irritability associated with ASD at screening and baseline

The primary outcome measure is ABC-I subscale score at week 6.¹ 

Overall, 174 patients are estimated to be enrolled. The estimated primary completion is March 2027 and the estimated study completion is April 2027. For additional information, please visit http://clinicaltrials.gov.¹ 

Phase 1 Study

An ongoing, phase 1b, multicenter, open-label, multiple-dose study (NCT06557902) is evaluating the safety, tolerability, and PK of CAPLYTA in pediatric patients aged 5 to <13 years with a primary clinical diagnosis of ASD with symptoms of irritability. The study consists of 2 treatment groups. Group 1 consists of patients aged 10 to <13 years who receive CAPLYTA capsules once daily as follows: 10.5 mg on days 1 and 2, and 10.5 mg or 21 mg on days 3, 4, and 5. Group 2 consists of patients aged 5 to <10 years who receive CAPLYTA orally disintegrating tablets (ODT) once daily as follows: 5 mg on days 1 and 2, and 5 mg or 10.5 mg on days 3, 4, and 5. Dose adjustments in Group 2 are permitted after dosing the first 6 patients; however, doses should not exceed 21 mg in subsequent patients.² 

Patients with the ability to swallow capsules and who meet the following criteria at screening are eligible for enrollment²:

• ABC-I subscale score ≥12
• CGI-S score ≥3
• Body mass index greater than the fifth percentile according to age- and gender-specific Clinical Growth Charts (2000) from the Centers for Disease Control and Prevention (CDC)

Primary outcome measures are the following PK parameters of lumateperone on day 5: maximum plasma concentration (C_max), time of maximum plasma concentration (T_max), and area under the plasma concentration-time curve from time 0 to the end of dosing
(AUC_0-tau).² 

Overall, 26 patients are estimated to be enrolled. The estimated primary completion is July 2026 and the estimated study completion is July 2026. For additional information, please visit http://clinicaltrials.gov.² 

Use of CAPLYTA in Adult Patients

Case Study

A case study of two 18-year-old male patients reported the impact of CAPLYTA in intermittent explosive disorder in ASD.³ 

Psychiatric examination of the patient in case A revealed that the patient was nonverbal and intermittently grunting and screaming; he exhibited darting eyes, no response to verbal commands, posturing of arms in the air, and a hostile stance. After 10 days of CAPLYTA 42 mg administered once nightly, the patient was no longer banging on the furniture or biting. He remained nonverbal but did not scream or exhibit a hostile behavior.³ 

Psychiatric examination of the patient in case B revealed that he was nonverbal, screaming, throwing furniture, hitting walls, and uncooperative to verbal commands. After 1 month of CAPLYTA 42 mg administered once nightly, the patient was not severely violent. After 2 months, the patient’s aggression was much less severe and he could attend school. Behavioral problems with aggression were noticeable 1 hour before the next evening’s CAPLYTA dose.³ 

Literature Search

A literature search of MEDLINE^®, EMBASE^®, BIOSIS Previews^®, and DERWENT^® (and/or other resources, including internal/external databases) was conducted on 08 September 2025.