Clinical trial NCT05953389: 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
Overall status: RECRUITING
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Lead sponsor: Bioprojet
Sponsor class: OTHER
First posted: 2023-07-20
Enrollment: 62
Sex: MALE
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Social Responsiveness Scale Second Edition (SRS-2) total score
timeFrame: 12 weeks
Secondary outcomes:
measure: Vineland Adaptive Behavior Scale III (VABS III) total score
timeFrame: 12 weeks
measure: Incidence of Treatment-Emergent adverse events (AEs) as assessed by AEs collection
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Bordeaux
country: France
facility: Centre Hospitalier Charles Perrens
city: Nantes
country: France
facility: Nantes University Hospital
city: Sotteville-lès-Rouen
country: France
facility: CH le Rouvray
city: Bologna
country: Italy
facility: IRCCS-Istituto delle Scienze Neurologiche di Bologna
city: Bosisio Parini
country: Italy
facility: Istituto Scientifico IRCCS E. Medea
city: Cagliari
country: Italy
facility: Clinica Di Neuropsichiatria Infantile
city: Calambrone
country: Italy
facility: IRCCS Fondazione Stella Maris
city: Foggia
country: Italy
facility: A.O.U Ospedali Riuniti di Foggia- Cardiologia (U.O.C)
city: Messina
country: Italy
facility: IRCCS Centro Neurolesi Bonino Pulejo
city: Napoli
country: Italy
facility: Universita degli Studi di Napoli Federico II
city: Pavia
country: Italy
facility: IRCCS Instituto Neurologico Casimiro Mondino
city: Siena
country: Italy
facility: Azienda ospedaliero-universitaria Senese
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Burgos
country: Spain
facility: Hospital Universitario de Burgos
city: London
country: United Kingdom
facility: Cognition Health
Study officials:
affiliation: Etablissement Public de Santé Barthélemy Durand, Sainte-Geneviève-des-Bois, France
name: Olivier Bonnot, MD, Prof.
role: PRINCIPAL_INVESTIGATOR
References: