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

Sponsor

Bioprojet (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05953389

Collaborator

(none)

Enrollment

Arms

Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Autism Spectrum Disorder	Drug: Pitolisant Drug: Placebo	Phase 2

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

62 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Oct 1, 2023

Anticipated Primary Completion Date :

Jan 1, 2025

Anticipated Study Completion Date :

Jan 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Pitolisant 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 histamine H3 receptor antagonist/inverse agonist Other Names: BF2.649
Placebo Comparator: Placebo One or two tablets of matching placebo per day for 12 weeks.	Drug: Placebo Matching placebo of pitolisant tablets

Arm

Intervention/Treatment

Experimental: Pitolisant

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

histamine H3 receptor antagonist/inverse agonist

Other Names:

BF2.649

Placebo Comparator: Placebo

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

Drug: Placebo

Matching placebo of pitolisant tablets

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 17 Years

Sexes Eligible for Study:

Male

Accepts 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.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

Bioprojet

Investigators

Principal Investigator: Olivier Bonnot, MD, Prof., Nantes hospital, Department of Child & adolescent psychiatry, Nantes, France

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Bioprojet

ClinicalTrials.gov Identifier:

NCT05953389

Other Study ID Numbers:

P21-01 / BF2.649

First Posted:

Jul 20, 2023

Last Update Posted:

Jul 20, 2023

Last Verified:

Jul 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Autistic Disorder

Autism Spectrum Disorder

Child Development Disorders, Pervasive

Study Results

No Results Posted as of Jul 20, 2023