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

Phase 2

Not yet recruiting

• Conditions: Autism Spectrum Disorder
• Interventions: Drug: Placebo; Drug: Pitolisant

• Registration Number: NCT05953389
• Lead Sponsor: Bioprojet

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.

Study Timeline

• Status Verified: 2023-07
• Study First Submitted: 2023-07-12
• Study First Submitted QC: 2023-07-12
• Last Update Submitted: 2023-07-12
• Study First Posted: 2023-07-20
• Last Update Posted: 2023-07-20
• Study Start: 2023-10
• Primary Completion: 2025-01
• Study Completion: 2025-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: Male
• Target Recruitment: 62

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	One or two tablets of matching placebo per day for 12 weeks.
Pitolisant	Pitolisant	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.

Primary Outcome Measures

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

Name	Time	Method
Vineland Adaptive Behavior Scale III (VABS III) total score	12 weeks	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	12 weeks	Safety assessment of pitolisant based on adverse events (AEs) reporting during the treatment pe

Trial Locations

Locations (15)

Centre Hospitalier Charles Perrens; 🇫🇷 Bordeaux, France

Nantes University Hospital; 🇫🇷 Nantes, France

CH le Rouvray; 🇫🇷 Sotteville-lès-Rouen, France

IRCCS-Istituto delle Scienze Neurologiche di Bologna; 🇮🇹 Bologna, Italy

Istituto Scientifico IRCCS E. Medea; 🇮🇹 Bosisio Parini, Italy

Clinica Di Neuropsichiatria Infantile; 🇮🇹 Cagliari, Italy

IRCCS Fondazione Stella Maris; 🇮🇹 Calambrone, Italy

A.O.U Ospedali Riuniti di Foggia- Cardiologia (U.O.C); 🇮🇹 Foggia, Italy

IRCCS Centro Neurolesi Bonino Pulejo; 🇮🇹 Messina, Italy

Universita degli Studi di Napoli Federico II; 🇮🇹 Napoli, Italy

IRCCS Instituto Neurologico Casimiro Mondino; 🇮🇹 Pavia, Italy

Azienda ospedaliero-universitaria Senese; 🇮🇹 Siena, Italy

Hospital Universitari Vall d'Hebron; 🇪🇸 Barcelona, Spain

Hospital Universitario de Burgos; 🇪🇸 Burgos, Spain

Cognition Health; 🇬🇧 London, United Kingdom