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

Primary Purpose

Autism Spectrum Disorder

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Pitolisant
Placebo
Sponsored by
Bioprojet
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder

Eligibility Criteria

6 Years - 17 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    Placebo Comparator

    Arm Label

    Pitolisant

    Placebo

    Arm Description

    One tablet of pitolisant 5 mg, two tablets of pitolisant 5 mg, one tablet of pitolisant 20 mg or two tablets of pitolisant 20 mg per day for 12 weeks.

    One or two tablets of matching placebo per day for 12 weeks.

    Outcomes

    Primary Outcome Measures

    Social Responsiveness Scale Second Edition (SRS-2) total score
    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 Outcome Measures

    Vineland Adaptive Behavior Scale III (VABS III) total score
    Instrument that measures socialization, communication, daily living skills, motor skills and maladaptive behavior of individuals with intellectual disabilities
    Incidence of Treatment-Emergent adverse events (AEs) as assessed by AEs collection
    Safety assessment of pitolisant based on adverse events (AEs) reporting during the treatment pe

    Full Information

    First Posted
    July 12, 2023
    Last Updated
    July 12, 2023
    Sponsor
    Bioprojet
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05953389
    Brief Title
    Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents
    Official 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
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    July 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    October 2023 (Anticipated)
    Primary Completion Date
    January 2025 (Anticipated)
    Study Completion Date
    January 2025 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Bioprojet

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    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.
    Detailed Description
    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Autism Spectrum Disorder

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigatorOutcomes Assessor
    Allocation
    Randomized
    Enrollment
    62 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Pitolisant
    Arm Type
    Experimental
    Arm Description
    One tablet of pitolisant 5 mg, two tablets of pitolisant 5 mg, one tablet of pitolisant 20 mg or two tablets of pitolisant 20 mg per day for 12 weeks.
    Arm Title
    Placebo
    Arm Type
    Placebo Comparator
    Arm Description
    One or two tablets of matching placebo per day for 12 weeks.
    Intervention Type
    Drug
    Intervention Name(s)
    Pitolisant
    Other Intervention Name(s)
    BF2.649
    Intervention Description
    histamine H3 receptor antagonist/inverse agonist
    Intervention Type
    Drug
    Intervention Name(s)
    Placebo
    Intervention Description
    Matching placebo of pitolisant tablets
    Primary Outcome Measure Information:
    Title
    Social Responsiveness Scale Second Edition (SRS-2) total score
    Description
    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.
    Time Frame
    12 weeks
    Secondary Outcome Measure Information:
    Title
    Vineland Adaptive Behavior Scale III (VABS III) total score
    Description
    Instrument that measures socialization, communication, daily living skills, motor skills and maladaptive behavior of individuals with intellectual disabilities
    Time Frame
    12 weeks
    Title
    Incidence of Treatment-Emergent adverse events (AEs) as assessed by AEs collection
    Description
    Safety assessment of pitolisant based on adverse events (AEs) reporting during the treatment pe
    Time Frame
    12 weeks

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    6 Years
    Maximum Age & Unit of Time
    17 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Stéphanie Renaux
    Phone
    +33147036633
    Email
    s.renaux@bioprojet.com
    First Name & Middle Initial & Last Name or Official Title & Degree
    Teresa Gidaro, MD
    Phone
    +33147036633
    Email
    t.gidaro@bioprojet.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Olivier Bonnot, MD, Prof.
    Organizational Affiliation
    Nantes hospital, Department of Child & adolescent psychiatry, Nantes, France
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No

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

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