Clinical trial NCT06690398: Multicenter Study of Lumateperone for the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Irritability Associated With Autism Spectrum Disorder
Lead sponsor: Intra-Cellular Therapies, Inc.
Sponsor class: INDUSTRY
First posted: 2024-11-15
Enrollment: 174
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).

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.

Eligibility criteria: 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 (C-SSRS) 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.

Primary outcomes:
measure: Aberrant Behavior Checklist - Irritability (ABC-I)
timeFrame: Week 6
Secondary outcomes:
measure: Clinical Global Impression-Severity (CGI-S)
timeFrame: Week 6
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Clinical Site
state: Alabama
city: Peoria
country: United States
facility: Clinical Site
state: Arizona
city: Phoenix
country: United States
facility: Clinical Site
state: Arizona
city: Buena Park
country: United States
facility: Clinical Site
state: California
city: Corona
country: United States
facility: Clinical Site
state: California
city: Oceanside
country: United States
facility: Clinical Site
state: California
city: Upland
country: United States
facility: Clinical Site
state: California
city: West Covina
country: United States
facility: Clinical Site
state: California
city: Colorado Springs
country: United States
facility: Clinical Site
state: Colorado
city: Gainesville
country: United States
facility: Clinical Site
state: Florida
city: Miami Lakes
country: United States
facility: Clinical Site
state: Florida
city: Miami Springs
country: United States
facility: Clinical Site
state: Florida
city: Miami
country: United States
facility: Clinical Site
state: Florida
city: Miami
country: United States
facility: Clinical Site
state: Florida
city: Miami
country: United States
facility: Clinical Site
state: Florida
city: Miami
country: United States
facility: Clinical Site
state: Florida
city: West Palm Beach
country: United States
facility: Clinical Site
state: Florida
city: Savannah
country: United States
facility: Clinical Site
state: Georgia
city: Arlington Heights
country: United States
facility: Clinical Site
state: Illinois
city: Springfield
country: United States
facility: Clinical Site
state: Illinois
city: Boston
country: United States
facility: Clinical Site
state: Massachusetts
city: Saint Charles
country: United States
facility: Clinical Site
state: Missouri
city: Las Vegas
country: United States
facility: Clinical Site
state: Nevada
city: Cincinnati
country: United States
facility: Clinical Site
state: Ohio
city: Garfield
country: United States
facility: Clinical Site
state: Ohio
city: Westlake
country: United States
facility: Clinical Site
state: Ohio
city: Nashville
country: United States
facility: Clinical Site
state: Tennessee
city: Dallas
country: United States
facility: Clinical Site
state: Texas
city: Frisco
country: United States
facility: Clinical Site
state: Texas
city: Houston
country: United States
facility: Clinical Site
state: Texas
city: Plano
country: United States
facility: Clinical Site
state: Texas
city: Petersburg
country: United States
facility: Clinical Site
state: Virginia
city: Bellevue
country: United States
facility: Clinical Site
state: Washington
Study officials:
References: