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NCT Number: NCT05953389

Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents

Proof of concept, multicenter, randomized, double-blind, placebo-controlled, parallel-group, study to investigate the effect, safety, tolerability and pharmacokinetics of pitolisant in male children and adolescents with Autism Spectrum Disorders.

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Key information

Conditions

Autism Spectrum Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

6 year–17 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Centre Hospitalier Charles Perrens, Bordeaux, France

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About this study

First clinical study to assess the effect of BF2.649 in male children and adolescent with a diagnosis of Autism spectrum disorders according to DSM-5 criteria and confirmed by the Autism Diagnostic Observation Schedule (ADOS-2) or Autism Diagnostic Interview-Revised (ADI-R) over a 12-weeks period.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants and their parent(s)/legal guardian(s) are willing and able to give informed assent and consent for participation in the study.
  • Male children and adolescents aged from 6 to 17 inclusive for the duration of study participation.
  • Diagnosis of autism spectrum disorders (ASD) as per the Diagnostic and Statistical Manual of Mental Disorders (DSM-5) criteria confirmed by the Autism Diagnostic Observation Schedule (ADOS-2) or the Autism Diagnostic Interview-Revised (ADI-R).
  • Intelligence Quotient (IQ) ≥ 70 using Wechsler Intelligence Scale.
  • Social Responsiveness Scale Second Edition (SRS-2) total T-score ≥ 66 at screening and baseline.

Exclusion criteria

  • Previous genetic diagnosis of ASD-known "syndromic" ASD (e.g., Fragile X syndrome, Angelman syndrome, Prader-Willi, Rett's syndrome, tuberous sclerosis, Dup15q syndrome).
  • History of suicidal behavior or suicidal ideation in the past 12 months, or a positive answer to questions 4 or 5 on the Columbia-Suicide-Severity Rating Scale (C-SSRS) at screening and/or baseline, and/or is a significant risk for suicidal behavior per investigator judgement.
  • History or current diagnosis of epilepsy or any seizure occurring after the age of 5.
  • Clinically significant deviation from normal on 12-lead ECG that results in an active medical problem, per investigator judgement or, with a corrected QT interval by Fridericia (QTcF) > 450ms at screening.
  • Severe hepatic impairment (Child Pugh C) or with any other hepatic significant abnormality in the physical examination or alanine aminotransferase (ALAT) or aspartate aminotransferase (ASAT) ≥ 2x Upper Limit of Normal (ULN) for age at laboratory results.

Treatment and study plan

Pitolisant

Drug

histamine H3 receptor antagonist/inverse agonist

Other names: BF2.649

Placebo

Drug

Matching placebo of pitolisant tablets

Primary outcomes

  1. Social Responsiveness Scale Second Edition (SRS-2) total score

    Time frame: 12 weeks

    65-item informant-based rating scale designed specifically for use in Autism Spectrum Disorders to quantitatively measure an individual's ability to engage in emotionally appropriate reciprocal social behavior with higher scores indicating greater impairment.

Secondary outcomes

  1. Vineland Adaptive Behavior Scale III (VABS III) total score

    Time frame: 12 weeks

    Instrument that measures socialization, communication, daily living skills, motor skills and maladaptive behavior of individuals with intellectual disabilities;

  2. Incidence of Treatment-Emergent adverse events (AEs) as assessed by AEs collection

    Time frame: 12 weeks

    Safety assessment of pitolisant based on adverse events (AEs) reporting during the treatment pe

Sponsors and collaborators

Lead sponsor

Bioprojet

Other

Registry information

Official study title

An Exploratory, Multicenter, Randomized, Double-blind, Placebo-controlled Study Evaluating the Effect and Safety of Pitolisant in Children and Adolescents With Autism Spectrum Disorders

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Jul 20, 2023
Registry last updated
Oct 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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