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Study on the Effects of Pitolisant for Children and Adolescents with Autism Spectrum Disorder

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects and safety of a medication called pitolisant in children and adolescents who have Autism Spectrum Disorders (ASD). The study involves using Wakix tablets, which contain the active substance pitolisant, and are available in two different strengths: 4.5 mg and 18 mg. The purpose of the study is to explore how pitolisant might help with the social communication and interaction challenges that are common in individuals with ASD.

Participants in the study will be randomly assigned to receive either the pitolisant tablets or a placebo, which looks like the real medication but does not contain the active substance. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are unbiased. The study will last for a period of 12 weeks, during which the participants will take the tablets orally.

Throughout the study, the effects of pitolisant on social communication and interaction will be assessed using a tool called the Social Responsiveness Scale Second Edition (SRS-2). The study will also monitor any changes in behavior, sleep patterns, and overall well-being. The goal is to determine if pitolisant can improve the core symptoms of ASD and to ensure that it is safe for use in children and adolescents. Participants will be closely monitored for any side effects or adverse reactions during the study period.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, an initial visit is scheduled. During this visit, the participant undergoes a series of assessments to confirm eligibility. This includes verifying the diagnosis of autism spectrum disorders and ensuring all criteria are met.

    The participant's ability to swallow the investigational medicinal product is also assessed. The participant and their caregiver are informed about the study procedures and requirements.

  2. Step 2

    Baseline assessments

    Baseline assessments are conducted to gather initial data. This includes measuring the Social Responsiveness Scale Second Edition (SRS-2) total score, which evaluates social communication and interaction skills.

    Other assessments may include the Vineland Adaptive Behavior Scale III and the Child’s Sleep Habits Questionnaire to establish a starting point for the study.

  3. Step 3

    Medication administration

    Participants are randomly assigned to receive either the study medication, pitolisant, or a placebo. The medication is provided in the form of film-coated tablets, taken orally.

    The dosage and frequency of administration are determined by the study protocol. Participants are required to take the medication as instructed for the duration of the study.

  4. Step 4

    Regular follow-up visits

    Regular follow-up visits are scheduled throughout the study period. During these visits, participants undergo various assessments to monitor progress and any changes in symptoms.

    The Social Responsiveness Scale Second Edition (SRS-2) and other relevant scales are used to evaluate the effects of the medication.

  5. Step 5

    End of treatment assessments

    At the end of the treatment period, final assessments are conducted. These assessments measure any changes from the baseline in social communication and interaction skills.

    The results are used to determine the effectiveness and safety of the medication.

  6. Step 6

    Study completion

    Upon completion of the study, participants may receive information about the overall findings. Any necessary follow-up care or recommendations are provided to ensure the participant's well-being.

Who can join the trial?

10 criteria

  • Participants and their parent(s) or legal guardian(s) must be willing and able to give informed consent to join the study. This means they understand what the study involves and agree to take part.
  • Participants must have a diagnosis of autism spectrum disorders (ASD) confirmed by specific tests or a historical diagnosis within the last 3 years.
  • Participants must have a care provider who can bring them to clinic visits, provide reliable feedback, support them with study requirements, and interact with them regularly (at least 3 hours a day, 4 days a week).
  • Participants must agree to follow specific contraceptive guidelines during the study.
  • Participants must be children or adolescents aged 6 to 17 years old.
  • Participants must have an Intelligence Quotient (IQ) of 70 or higher, confirmed by recent tests or historical data. If the total IQ score is unclear, they should have specific sub-scores of 70 or higher in verbal comprehension and perceptual reasoning.
  • Participants must have a Social Responsiveness Scale Second Edition (SRS-2) total T-score of 66 or higher at the start of the study. This score measures social communication and interaction skills.
  • All current medications or treatments for ASD symptoms must have been stable for at least 4 weeks before starting the study, and participants must be willing to keep them stable during the study.
  • Participants must have language, hearing, and vision abilities that allow them to complete the study assessments.
  • Participants must be able to swallow the study medication.

Who cannot join the trial?

3 criteria

  • Patients who are not diagnosed with autism spectrum disorders cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.
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Investigated drugs

Pitolisant is a medication being studied for its potential benefits in children and adolescents with Autism Spectrum Disorders (ASD). It is thought to help improve social communication and interaction, which are often challenging for individuals with ASD. The study aims to see if pitolisant can make a positive difference in these areas by evaluating changes in a specific score that measures social responsiveness. This medication works by affecting certain chemicals in the brain that are involved in wakefulness and alertness, which might also influence social behaviors.

What is already known about the treatment

Pitolisant – Pitolisant is administered orally in the form of tablets and is currently being studied for its potential benefits in treating autism spectrum disorders (ASD) in children and adolescents. This medication is still under investigation, and its effects and safety are being evaluated in clinical trials. The main therapeutic indication for pitolisant in this context is to improve social communication and interaction deficits, which are core symptoms of ASD. At the molecular level, pitolisant works by increasing the activity of histamine in the brain, which is thought to help improve wakefulness and cognitive function. It is classified pharmacologically as a histamine H3 receptor antagonist/inverse agonist.

Investigated diseases

Autism Spectrum Disorder – Autism Spectrum Disorder is a developmental condition that affects communication and behavior. It is characterized by challenges with social skills, repetitive behaviors, and speech and nonverbal communication. The symptoms typically appear in early childhood and can vary widely in severity. Individuals may have difficulty understanding social cues and forming relationships. They might also engage in repetitive activities or have specific routines. The progression of symptoms can change over time, with some individuals experiencing improvements in social skills and communication.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2023-503678-18-00Protocol codeP21-01Estimated enrolment70 patientsSponsorBioprojet Pharma

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