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

NCT05953389 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: P21-01 / BF2.649
• Study Type: interventional
• Target: 62
• Locations: 4 countries
• Sites: 15

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.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Vineland Adaptive Behavior Scale III (VABS III) total score

  12 weeks

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

  12 weeks

Study Arms (2)

Pitolisant

EXPERIMENTAL

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.

Drug: Pitolisant

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Pitolisant DRUG

histamine H3 receptor antagonist/inverse agonist

Also known as: BF2.649

Pitolisant

Placebo DRUG

Matching placebo of pitolisant tablets

Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bioprojet: lead

Study Sites (15)

Centre Hospitalier Charles Perrens

Bordeaux, 33076, France

Location

Nantes University Hospital

Nantes, 44093, France

Location

CH le Rouvray

Sotteville-lès-Rouen, 76300, France

Location

IRCCS-Istituto delle Scienze Neurologiche di Bologna

Bologna, 40139, Italy

Location

Istituto Scientifico IRCCS E. Medea

Bosisio Parini, 23842, Italy

Location

Clinica Di Neuropsichiatria Infantile

Cagliari, 09121, Italy

Location

IRCCS Fondazione Stella Maris

Calambrone, 56128, Italy

Location

A.O.U Ospedali Riuniti di Foggia- Cardiologia (U.O.C)

Foggia, 71122, Italy

Location

IRCCS Centro Neurolesi Bonino Pulejo

Messina, 98124, Italy

Location

Universita degli Studi di Napoli Federico II

Napoli, 80131, Italy

Location

IRCCS Instituto Neurologico Casimiro Mondino

Pavia, 27100, Italy

Location

Azienda ospedaliero-universitaria Senese

Siena, 53100, Italy

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08030, Spain

Location

Hospital Universitario de Burgos

Burgos, 09006, Spain

Location

Cognition Health

London, W1G 9JF, United Kingdom

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

pitolisant

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Olivier Bonnot, MD, Prof.

  Etablissement Public de Santé Barthélemy Durand, Sainte-Geneviève-des-Bois, France

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 12, 2023
• First Posted: July 20, 2023
• Study Start: December 20, 2023
• Primary Completion: October 1, 2025
• Study Completion: November 1, 2025
• Last Updated: October 2, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

IT (9); GB (1); FR (3); ES (2)