Autism Spectrum Disorder
Conditions
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.
Interventions
DRUGPitolisant
histamine H3 receptor antagonist/inverse agonist
DRUGPlacebo
Matching placebo of pitolisant tablets
Sponsors
Bioprojet
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
MALE
Age
6 Years to 17 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
France, Italy, Spain, United Kingdom
Outcome results
None listed