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

Autism Spectrum Disorder Clinical Trial

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05953389
• Other study ID #: P21-01 / BF2.649
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: October 2023
• Est. completion date: January 2025

Study information

• Verified date: July 2023
• Source: Bioprojet
• Contact: Stéphanie Renaux
• Phone: +33147036633
• Email: s.renaux[@]bioprojet.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 62
• Est. completion date: January 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder
• Child Development Disorders, Pervasive

Intervention

Drug:
• Pitolisant
histamine H3 receptor antagonist/inverse agonist
• Placebo
Matching placebo of pitolisant tablets

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bioprojet

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	12 weeks
Secondary	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	12 weeks
Secondary	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	12 weeks