Investigating the effects of pitolisant on autism in boys aged 6 to 17
An Exploratory, Multicenter, Randomized, Double-blind, Placebo-controlled Study Evaluating the Effect and Safety of Pitolisant in Children and Adolescents with Autism Spectrum Disorders
This study is testing if the medication pitolisant can help boys aged 6 to 17 with autism feel better over 12 weeks.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 62 (estimated) |
| Ages | 6 Years to 17 Years |
| Sex | Male |
| Sponsor | Bioprojet Academic / other |
| Locations | 15 sites (Bordeaux and 14 other locations) |
| Trial ID | NCT05953389 on ClinicalTrials.gov |
What this trial studies
This is a multicenter, randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, and pharmacokinetics of pitolisant in male children and adolescents diagnosed with Autism Spectrum Disorders (ASD). The study will last for 12 weeks and aims to assess the drug's effectiveness based on established diagnostic criteria. Participants will be closely monitored for changes in their condition and any side effects during the trial.
Who should consider this trial
Good fit: Ideal candidates are male children and adolescents aged 6 to 17 with a confirmed diagnosis of Autism Spectrum Disorder.
Not a fit: Patients with syndromic forms of Autism Spectrum Disorder or a history of suicidal behavior may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for children and adolescents with Autism Spectrum Disorders.
How similar studies have performed: While this approach is novel in the context of ASD, previous studies on pitolisant for other conditions have shown promising results.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Bordeaux and 14 other locations
- Centre Hospitalier Charles Perrens — Bordeaux, France (Recruiting)
- Nantes University Hospital — Nantes, France (Active_not_recruiting)
- CH le Rouvray — Sotteville-lès-Rouen, France (Recruiting)
- IRCCS-Istituto delle Scienze Neurologiche di Bologna — Bologna, Italy (Recruiting)
- Istituto Scientifico IRCCS E. Medea — Bosisio Parini, Italy (Active_not_recruiting)
- Clinica Di Neuropsichiatria Infantile — Cagliari, Italy (Active_not_recruiting)
- IRCCS Fondazione Stella Maris — Calambrone, Italy (Active_not_recruiting)
- A.O.U Ospedali Riuniti di Foggia- Cardiologia (U.O.C) — Foggia, Italy (Recruiting)
- IRCCS Centro Neurolesi Bonino Pulejo — Messina, Italy (Recruiting)
- Universita degli Studi di Napoli Federico II — Napoli, Italy (Recruiting)
- IRCCS Instituto Neurologico Casimiro Mondino — Pavia, Italy (Active_not_recruiting)
- Azienda ospedaliero-universitaria Senese — Siena, Italy (Active_not_recruiting)
- Hospital Universitari Vall d'Hebron — Barcelona, Spain (Active_not_recruiting)
- Hospital Universitario de Burgos — Burgos, Spain (Recruiting)
- Cognition Health — London, United Kingdom (Recruiting)
Study contacts
- Principal investigator: Olivier Bonnot, MD, Prof. — Etablissement Public de Santé Barthélemy Durand, Sainte-Geneviève-des-Bois, France
- Study coordinator: Stéphanie Renaux
- Email: s.renaux@bioprojet.com
- Phone: +33147036633
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.