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

Bioprojet

Status and phase: Active, not recruiting; Phase 2

Conditions: Autism Spectrum Disorder

Treatments: Drug: Placebo; Drug: Pitolisant

Study type: Interventional

Funder types: Other

Identifiers

NCT05953389

P21-01 / BF2.649

Details and patient eligibility

About

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.

Full 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.

Enrollment: 62 estimated patients

Sex: Male

Ages: 6 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

62 participants in 2 patient groups, including a placebo group

Pitolisant

Experimental group

Description:

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.

Treatment:

Drug: Pitolisant

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo