Safety, Tolerability and Pharmacokinetics of Lumateperone in Pediatric Patients With Autism Spectrum Disorder

An Open-label, Multiple-dose Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Lumateperone in Pediatric Patients, Ages 5 to Less Than 13 Years, Diagnosed With Autism Spectrum Disorder

Study ITI-007-035 is a Phase 1b, multicenter, open-label study to evaluate the safety, tolerability, and PK of lumateperone for pediatric patients with Autism Spectrum Disorder.

Study Overview

• Status: Recruiting
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: Lumateperone 10.5 mg capsule; Drug: Lumateperone 21 mg capsule; Drug: Lumateperone 5 mg ODT; Drug: Lumateperone 10.5 mg ODT; Drug: Lumateperone 15.5 mg ODT; Drug: Lumateperone 21 mg ODT

• Study Type: Interventional
• Enrollment (Estimated): 26
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: ITI Clinical Trials; Phone Number: 6464409333; Email: ITCIClinicalTrials@itci-inc.com

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33133
      • Not yet recruiting
      • Clinical Site 6
    • Orlando, Florida, United States, 32803
      • Not yet recruiting
      • Clinical Site 7
  • Georgia
    • Atlanta, Georgia, United States, 30331
      • Recruiting
      • Clinical Site 1
    • Decatur, Georgia, United States, 30030
      • Recruiting
      • Clinical Site 2
    • Savannah, Georgia, United States, 31405
      • Recruiting
      • Clinical Site 3
  • Missouri
    • Saint Charles, Missouri, United States, 63304
      • Recruiting
      • Clinical Site 4
  • Nebraska
    • Lincoln, Nebraska, United States, 68526
      • Recruiting
      • Clinical Site 5
  • Washington
    • Everett, Washington, United States, 98201
      • Recruiting
      • Clinical Site 8

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Male or female patients between 5 to less than 13 years of age
• Primary clinical diagnosis of ASD with symptoms of irritability
• ABC-I subscale score of ≥12 at Screening
• CGI-S score of ≥3 at Screening
• Body mass index (BMI) greater than the 5th percentile according to age- and gender-specific CDC Clinical Growth Charts (2000) at Screening
• Ability to swallow capsules

Exclusion Criteria:

• Has a primary psychiatric diagnosis other than ASD
• Reports suicidal ideation (Type 3, 4 or 5 on the Baseline/Screening version of the C-SSRS) within 6 months prior to Screening, or any suicidal behavior within 2 years prior to Screening, and/or the Investigator assesses the patient to be a safety risk to him/herself or others
• Clinically significant abnormality within 2 years of Screening that in the Investigator's opinion may place the patient at risk or interfere with study outcome variables
• History of a clinically significant cardiac disorder and/or abnormal screening ECG or a QT interval corrected for heart rate using Fridericia formula (QTcF) > 460 msec at Screening
• Patients with a history of orthostatic hypotension or who have orthostatic hypotension at Screening
• Has a history of uncontrolled/disruptive behavior in the past 30 days that, in the Investigator's opinion, would preclude the ability to participate in study procedures

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Group 1 (10 to less than 13 years); Lumateperone capsule once daily: 10.5 mg on Days 1 and 2; 10.5 mg or 21 mg on Day 3; 10.5 mg or 21 mg on Days 4 and 5	Drug: Lumateperone 10.5 mg capsule; Lumateperone 10.5 mg capsule, oral administration; Drug: Lumateperone 21 mg capsule; Lumateperone 21 mg capsule, oral administration
Experimental: Group 2 (5 to less than 10 years); Lumateperone ODT once daily: 5 mg on Days 1 and 2; 5 mg or 10.5 mg on Day 3; 5 mg or 10.5 mg on Days 4 and 5; dosing regimen may be adjusted after the first 6 patients dosed and will not exceed 21 mg in subsequent patients	Drug: Lumateperone 5 mg ODT; Lumateperone 5 mg ODT, oral administration; Drug: Lumateperone 10.5 mg ODT; Lumateperone 10.5 mg ODT, oral administration; Drug: Lumateperone 15.5 mg ODT; Lumateperone 5 mg ODT + 10.5 mg ODT, oral administration; Drug: Lumateperone 21 mg ODT; Lumateperone 21 mg ODT, oral administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics: Cmax	Maximum plasma concentration of lumateperone	Day 5
Pharmacokinetics: Tmax	Time of maximum plasma concentration of lumateperone	Day 5
Pharmacokinetics: AUC0-tau	Area under the plasma lumateperone concentration time curve from time zero to the end of dosing (tau)	Day 5

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in ECG QT interval		Day 6
Change from baseline in hemoglobin		Day 6
Change from baseline in white blood cell count		Day 6
Change from baseline in aspartate aminotransferase		Day 6
Change from baseline in alanine aminotransferase		Day 6
Percentage of patients with treatment-emergent adverse events		Up to 30 days after last dose
Change from baseline in systolic and diastolic blood presssure		Day 6
Change from baseline in Abnormal Involuntary Movement Scale (AIMS)	AIMS is a measure of facial and oral movements, extremity movements and trunk movements. Items are rated on a scale from none (0) to severe (4).	Day 6

Collaborators and Investigators

• Sponsor: Intra-Cellular Therapies, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 10, 2024
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): July 31, 2026

Study Registration Dates

• First Submitted: August 14, 2024
• First Submitted That Met QC Criteria: August 14, 2024
• First Posted (Actual): August 16, 2024

Study Record Updates

• Last Update Posted (Actual): August 16, 2024
• Last Update Submitted That Met QC Criteria: August 14, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: ITI-007-035

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