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 on Medication for Irritability in Children with Autism

L
Leslie Hulvershorn, MD, MSc

Primary Investigator

Enrolling By Invitation
13 years - 17 years
All
Phase N/A
2 participants needed
1 Location

Brief description of study

This is a multicenter study in pediatric patients aged 13 to 17 years with a primary diagnosis of Autism Spectrum Disorder associated with irritability to evaluate the efficacy of high and low doses of lumateperone compared to placebo for the treatment of irritability associated with Autism Spectrum Disorder and to determine the safety and tolerability of high and low doses of lumateperone compared to placebo.

THIS STUDY IS ENROLLING BY INVITATION ONLY

Detailed description of study

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 of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Irritability Associated With Autism Spectrum Disorder, ASD, Riley
  • Age: 13 years - 17 years
  • Gender: All

Inclusion Criteria:

  1. The Child has a legally authorized representative (eg, parent or legal guardian) who is willing and able to be responsible for the safety and well-being of the child, provide information about the patient's condition, and accompany the patient to study visits.
  2. Able to provide consent as follows:
    1. The child's legally authorized representative must provide written, informed consent.
    2. The patient should provide written assent when developmentally appropriate.
  3. The child is a male or female and is 13 to 17 years of age
  4. The child has an official primary diagnosis of Autism Spectrum Disorder
  5. The child is able to swallow capsules

Exclusion Criteria:

  1. The child has a primary psychiatric diagnosis other than Autism Spectrum Disorder, Except Attention Deficit Hyperactivity Disorder (ADHD) and Mild and moderate intellectual disability
  2. The child has a history or current diagnosis of Rett syndrome or Fragile X syndrome
  3. The child has a history of suicidal intent, suicidal plan or suicidal attempt in the past 2 years
  4. The child is considered to be an imminent danger to themselves or others.

Other inclusion and exclusion criteria may apply

Updated on 30 Jun 2025. Study ID: PSYC-ICT-ITI-007-602, 25410

This study investigates the effects of an investigational medication on irritability in children and adolescents with Autism Spectrum Disorder (ASD). Autism Spectrum Disorder is a developmental condition that affects communication, behavior, and social interaction. The study involves pediatric participants aged 5 to 17 years diagnosed with irritability associated with ASD, confirmed by specific diagnostic criteria.

Participants will undergo a three-phase process: a screening period to assess eligibility, a double-blind treatment period where they will receive either a high dose, low dose of the investigational medication, or a placebo, and a safety follow-up period. A placebo is an inactive substance that looks like the investigational medicine but does not contain any medicine.

  • Who can participate: Children aged 13 to 17 years with ASD and irritability can join. They need a representative to give consent and must meet specific diagnostic criteria.
  • Study details: Participants will be randomly assigned to receive either the investigational medication at different doses or a placebo. They will take the treatment once daily and attend follow-up visits for monitoring.
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only