Safety, Tolerability, and Pharmacokinetics of Lumateperone in Pediatric Patients With Schizophrenia or Schizoaffective Disorder

An Open-label Multiple Oral Dose Study to Determine the Safety, Tolerability, and Pharmacokinetics of Lumateperone in Patients, Ages 13 to 17 Years, Diagnosed With Schizophrenia or Schizoaffective Disorder

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04779177

Enrollment

Registered

2021-03-03

Start date

2021-03-12

Completion date

2022-07-30

Last updated

2025-11-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pediatric, Schizophrenia

Brief summary

Study ITI-007-020 is a Phase 1b, multicenter, open-label study to evaluate the safety, tolerability, and PK of lumateperone as treatment for adolescent patients with schizophrenia or schizoaffective disorder.

Interventions

DRUGLumateperone 42 mg

Lumateperone 42 mg, oral administration

DRUGLumateperone 28 mg

Lumateperone 28 mg, oral administration

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

13 Years to 17 Years

Healthy volunteers

Inclusion criteria

Main Inclusion Criteria: * Male or female patients between 13 and 17 years of age, inclusive * Clinical diagnosis of schizophrenia or schizoaffective disorder according to Diagnostic and Statistical Manual of Mental Disorders (DSM-5) * Free from acute exacerbation of their psychosis for at least 3 months prior to Screening * Clinical Global Impression - Severity (CGI-S) score ≤ 4 * Body mass index (BMI) within 2 standard deviations of, age- and gender-specific body measurements (based on CDC Clinical Growth Chart, 2000) * Ability to swallow capsules Main

Exclusion criteria

* Has a primary psychiatric diagnosis other than schizophrenia or schizoaffective disorder * Reports having experienced suicidal ideation within 6 months prior to Screening, any suicidal behavior within 2 years prior to Screening based on the Columbia-Suicide Severity Rating Scale (C-SSRS), 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 electrocardiogram (ECG) or a QT interval corrected for heart rate using Fridericia formula \> 450 msec in males or \> 470 msec in females

Design outcomes

Primary

Measure	Time frame	Description
Pharmacokinetics: Cmax	Day 1 and Day 5	Maximum plasma concentration of lumateperone
Pharmacokinetics: Tmax	Day 1 and Day 5	Time of maximum concentration of lumateperone in plasma
Pharmacokinetics: AUC0-t	0 to 24 hours post-dose on Day 1 and Day 5	Area under the plasma concentration time curve from time zero to the last measurable of concentration of lumateperone
Pharmacokinetics: AUC0-tau	0 to 24 hours post-dose on Day 1 and Day 5	Area under the plasma lumateperone concentration time curve from time zero to the end of dosing (tau)
Pharmacokinetics: t1/2	Day 1 and Day 5	Terminal elimination half-life of lumateperone
Pharmacokinetics: CL/F	Day 1 and Day 5	Apparent oral clearance of lumateperone

Secondary

Measure	Time frame	Description
Change From Baseline in Alanine Aminotransferase	Baseline and Day 6	—
Percentage of Subjects With Treatment-emergent Adverse Events	up to 30 days after last dose, up to a total of 35 days	—
Change From Baseline in Abnormal Involuntary Movement Scale (AIMS)	Baseline and Day 6	AIMS is a measure of facial and oral movements, extremity movements and trunk movements. The AIMS total score is reported based on 7 items (items 1 through 7). Each item is rated on a scale from none (0) to severe (4). The AIMS total score ranges from 0 to 28. Higher values of total AIMS score indicate increased severity in abnormal movement.
Change From Baseline in Systolic and Diastolic Blood Pressure	Baseline and Day 6	—
Change From Baseline in ECG QT Interval	Baseline and Day 6	QTcF
Change From Baseline in Hemoglobin	Baseline and Day 6	—
Change From Baseline in White Blood Cell Count	Baseline and Day 6	—
Change From Baseline in Aspartate Aminotransferase	Baseline and Day 6	—

Countries

United States

Participant flow

Pre-assignment details

Lumateperone 42 mg once daily was evaluated in the study. Lumateperone 28 mg once daily was a potential dosage, however it was not evaluated in the study.

Participants by arm

Arm	Count
Lumateperone 42 mg Once Daily for 5 Days Lumateperone 42 mg: Lumateperone 42 mg, oral administration	26
Total	26

Baseline characteristics

Characteristic	Lumateperone 42 mg Once Daily for 5 Days
Age, Continuous	15.2 years STANDARD_DEVIATION 1.41
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	26 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	0 Participants
Race (NIH/OMB) Black or African American	26 Participants
Race (NIH/OMB) More than one race	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	0 Participants
Sex: Female, Male Female	11 Participants
Sex: Female, Male Male	15 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 26
other Total, other adverse events	16 / 26
serious Total, serious adverse events	0 / 26

Outcome results

Primary

Pharmacokinetics: AUC0-t

Area under the plasma concentration time curve from time zero to the last measurable of concentration of lumateperone

Time frame: 0 to 24 hours post-dose on Day 1 and Day 5

Population: The PK Analysis Population included all patients who received at least one dose of study drug, had no major protocol deviation that may have impacted PK analyses, and had measurable plasma concentrations to provide an estimate of at least Cmax or AUC.

Arm	Measure	Group	Value (MEAN)	Dispersion
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: AUC0-t	Day 1	43.612 h*ng/mL	Standard Deviation 21.404
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: AUC0-t	Day 5	54.975 h*ng/mL	Standard Deviation 26.992

Primary

Pharmacokinetics: AUC0-tau

Area under the plasma lumateperone concentration time curve from time zero to the end of dosing (tau)

Time frame: 0 to 24 hours post-dose on Day 1 and Day 5

Arm	Measure	Group	Value (MEAN)	Dispersion
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: AUC0-tau	Day 1	48.158 h*ng/mL	Standard Deviation 20.899
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: AUC0-tau	Day 5	58.437 h*ng/mL	Standard Deviation 27.792

Primary

Pharmacokinetics: CL/F

Apparent oral clearance of lumateperone

Time frame: Day 1 and Day 5

Arm	Measure	Group	Value (MEAN)	Dispersion
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: CL/F	Day 1	3062.0 L/h	Standard Deviation 1292
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: CL/F	Day 5	947.14 L/h	Standard Deviation 595.34

Primary

Pharmacokinetics: Cmax

Maximum plasma concentration of lumateperone

Time frame: Day 1 and Day 5

Arm	Measure	Group	Value (MEAN)	Dispersion
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: Cmax	Day 1	19.12 ng/mL	Standard Deviation 11.21
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: Cmax	Day 5	23.22 ng/mL	Standard Deviation 12.6

Primary

Pharmacokinetics: t1/2

Terminal elimination half-life of lumateperone

Time frame: Day 1 and Day 5

Arm	Measure	Group	Value (MEAN)	Dispersion
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: t1/2	Day 1	2.208 h	Standard Deviation 0.6162
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: t1/2	Day 5	2.681 h	Standard Deviation 1.092

Primary

Pharmacokinetics: Tmax

Time of maximum concentration of lumateperone in plasma

Time frame: Day 1 and Day 5

Arm	Measure	Group	Value (MEDIAN)
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: Tmax	Day 1	1.000 h
Lumateperone 42 mg Once Daily for 5 Days	Pharmacokinetics: Tmax	Day 5	1.000 h

Secondary

Change From Baseline in Abnormal Involuntary Movement Scale (AIMS)

AIMS is a measure of facial and oral movements, extremity movements and trunk movements. The AIMS total score is reported based on 7 items (items 1 through 7). Each item is rated on a scale from none (0) to severe (4). The AIMS total score ranges from 0 to 28. Higher values of total AIMS score indicate increased severity in abnormal movement.

Time frame: Baseline and Day 6