Multicenter Study of Lumateperone for the Treatment of Irritability Associated With Autism Spectrum Disorder (ASD) in Pediatric Patients

A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of Age

This is a multicenter, randomized, double-blind, placebo-controlled study in pediatric patients aged 5 to 17 years with a primary diagnosis of irritability associated with Autism Spectrum Disorder (ASD) based on Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition Text Revision (DSM-5-TR) and confirmed by the Kiddie Schedule for Affective Disorders and Schizophrenia Present and Lifetime Version (K-SADS-PL).

Study Overview

• Status: Recruiting
• Conditions: Irritability Associated With Autism Spectrum Disorder
• Intervention / Treatment: Drug: Lumateperone high dose; Drug: Lumateperone low dose; Drug: Placebo

Detailed Description

The study will be conducted in 3 phases:

• Screening Period (up to 14 days) during which patient eligibility will be assessed.
• Double-blind Treatment Period (DBTP) (6 weeks) during which all patients will be randomized in a 1:1:1 ratio to receive either lumateperone high dose, lumateperone low dose, or placebo as a once daily dose.
• Safety Follow-up Period (1 week) during which all patients will return to the clinic for a safety follow-up (SFU) visit approximately 1 week after the last dose of study drug.

• Study Type: Interventional
• Enrollment (Estimated): 174
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: ITI Clinical Trials; Phone Number: 646 440-9333; Email: ITCIClinicalTrials@itci-inc.com

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72204
      • Recruiting
      • Clinical Site
  • California
    • Imperial, California, United States, 92251
      • Not yet recruiting
      • Clinical Site
    • Sacramento, California, United States, 95817
      • Not yet recruiting
      • Clinical Site
    • Sherman Oaks, California, United States, 91403
      • Recruiting
      • Clinical Site
  • Connecticut
    • New Haven, Connecticut, United States, 06511
      • Not yet recruiting
      • Clinical Site
  • Florida
    • Hialeah, Florida, United States, 33012
      • Recruiting
      • Clinical Site
    • Miami, Florida, United States, 33122
      • Recruiting
      • Clinical Site
    • Miami, Florida, United States, 33144
      • Recruiting
      • Clinical Site
    • Miami, Florida, United States, 33130
      • Recruiting
      • Clinical Site
    • Miami, Florida, United States, 33165
      • Recruiting
      • Clinical Site
    • Miami, Florida, United States, 33173
      • Recruiting
      • Clinical Site
    • Miami Gardens, Florida, United States, 33056
      • Recruiting
      • Clinical Site
    • Miami Lakes, Florida, United States, 33014
      • Recruiting
      • Clinical Site
    • Orlando, Florida, United States, 32803
      • Recruiting
      • Clinical Site
    • Orlando, Florida, United States, 32809
      • Recruiting
      • Clinical Site
    • Pompano Beach, Florida, United States, 33060
      • Recruiting
      • Clinical Site
  • Georgia
    • Atlanta, Georgia, United States, 30318
      • Recruiting
      • Clinical Site
    • Lawrenceville, Georgia, United States, 30046
      • Recruiting
      • Clinical Site
  • Illinois
    • Naperville, Illinois, United States, 60563
      • Recruiting
      • Clinical Site
  • Indiana
    • Evansville, Indiana, United States, 47713
      • Not yet recruiting
      • Clinical Site
    • Indianapolis, Indiana, United States, 46202
      • Recruiting
      • Clinical Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Not yet recruiting
      • Clinical Site
  • Michigan
    • Bloomfield Hills, Michigan, United States, 48302
      • Recruiting
      • Clinical Site
  • Missouri
    • Saint Louis, Missouri, United States, 63108
      • Not yet recruiting
      • Clinical Site
  • Nebraska
    • Lincoln, Nebraska, United States, 68526
      • Recruiting
      • Clinical Site
  • New York
    • Bronx, New York, United States, 10467
      • Recruiting
      • Clinical Site
    • Orangeburg, New York, United States, 10962
      • Recruiting
      • Clinical Site
    • Staten Island, New York, United States, 10314
      • Recruiting
      • Clinical Site
  • North Carolina
    • Kinston, North Carolina, United States, 28504
      • Not yet recruiting
      • Clinical Site
  • Ohio
    • Avon Lake, Ohio, United States, 44012
      • Recruiting
      • Clinical Site
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73116
      • Recruiting
      • Clinical Site
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Not yet recruiting
      • Clinical Site
  • Texas
    • Austin, Texas, United States, 78759
      • Recruiting
      • Clinical Site
    • Coppell, Texas, United States, 75019
      • Not yet recruiting
      • Clinical Site
    • Dallas, Texas, United States, 75251
      • Recruiting
      • Clinical Site
    • Fort Worth, Texas, United States, 76104
      • Recruiting
      • Clinical Site
    • Houston, Texas, United States, 77090
      • Recruiting
      • Clinical Site
  • Virginia
    • Richmond, Virginia, United States, 23220
      • Recruiting
      • Clinical Site
  • Washington
    • Everett, Washington, United States, 98201
      • Recruiting
      • Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. All patients must have a legally authorized representative LAR (eg, parent or legal guardian) who is willing and able to be responsible for the safety and well-being of the patient, provide information about the patient's condition, and accompany the patient to study visits.

2. Able to provide consent as follows:

   1. The patient's LAR must provide written, informed consent.
   2. When developmentally appropriate based on Investigator judgment, the patient should provide written assent.
3. Male or female patients 5 to 17 years of age. Currently, only patients aged 13 to 17 years will be eligible for enrollment.
4. Meet Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition Text Revision (DSM-5-TR) primary diagnosis of ASD as confirmed by the Kiddie Schedule for Affective Disorders and Schizophrenia for School-Age Children-Present and Lifetime Version (K-SADS-PL);
5. ABC-I subscale score of >18 at Screening and Baseline;
6. CGI-S score > 4 with respect to irritability associated with ASD at Screening and Baseline.

Exclusion Criteria:

1. Has a primary psychiatric diagnosis other than ASD. Exceptions include:

   1. Attention Deficit Hyperactivity Disorder (ADHD). If a patient is taking medication(s) for ADHD, they must be on a stable treatment regimen of these medication(s) for 30 days prior to screening and the treatment regimen is expected to remain stable throughout the study. This must be confirmed by the Investigator and noted in the source records.
   2. Mild and moderate intellectual disability based on Investigator judgment and DSM-5 criteria (severe and profound intellectual disability are excluded).
2. History or current diagnosis of Rett syndrome or Fragile X syndrome;

3. In the opinion of the Investigator, the patient has a significant risk for suicidal behavior during their participation in the study or

   1. At Screening, the patient scores "yes" on Suicidal Ideation Items 3, 4, or 5 of the Columbia-Suicide Severity Rating Scale (CSSRS) within 6 months prior to Screening or, at Baseline, the patient scores "yes" on Suicidal Ideation Items 3, 4, or 5 since the Screening Visit;
   2. At Screening, the patient has had 1 or more suicidal attempts within the 2 years prior to Screening; or
   3. The patient is considered to be an imminent danger to themselves or others.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Matching Placebo administered orally once daily
Experimental: Lumateperone high dose; Lumateperone 42 mg for patients ages 13-17 years old	Drug: Lumateperone high dose; Lumateperone administered orally once daily
Experimental: Lumateperone low dose; Lumateperone 21 mg for patients ages 13-17 years old	Drug: Lumateperone low dose; Lumateperone administered orally once daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Aberrant Behavior Checklist - Irritability (ABC-I)	The ABC-I subscale contains 15 items which rate symptoms on a scale ranging from 0 (not at all a problem) to 3 (severe).	Week 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Global Impression-Severity (CGI-S)	The CGI-S is a clinician-rated scale to assess a patient's overall mental health. The scale ranges from 1 (normal, not at all ill) to 7 (among the most extremely ill patients).	Week 6

Collaborators and Investigators

• Sponsor: Intra-Cellular Therapies, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 6, 2024
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): April 1, 2027

Study Registration Dates

• First Submitted: November 22, 2024
• First Submitted That Met QC Criteria: November 22, 2024
• First Posted (Actual): November 26, 2024

Study Record Updates

• Last Update Posted (Estimated): July 8, 2025
• Last Update Submitted That Met QC Criteria: July 3, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autism Spectrum Disorder; Autistic Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: ITI-007-602

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No