- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05953389
Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents
July 12, 2023 updated by: Bioprojet
An Exploratory, Multicenter, Randomized, Double-blind, Placebo-controlled Study Evaluating the Effect and Safety of Pitolisant in Children and Adolescents With Autism Spectrum Disorders
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.
Study Overview
Status
Not yet recruiting
Conditions
Intervention / Treatment
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 Type
Interventional
Enrollment (Estimated)
62
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Stéphanie Renaux
- Phone Number: +33147036633
- Email: s.renaux@bioprojet.com
Study Contact Backup
- Name: Teresa Gidaro, MD
- Phone Number: +33147036633
- Email: t.gidaro@bioprojet.com
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / 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.
|
histamine H3 receptor antagonist/inverse agonist
Other Names:
|
Placebo Comparator: Placebo
One or two tablets of matching placebo per day for 12 weeks.
|
Matching placebo of pitolisant tablets
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Social Responsiveness Scale Second Edition (SRS-2) total score
Time Frame: 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.
|
12 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Vineland Adaptive Behavior Scale III (VABS III) total score
Time Frame: 12 weeks
|
Instrument that measures socialization, communication, daily living skills, motor skills and maladaptive behavior of individuals with intellectual disabilities
|
12 weeks
|
Incidence of Treatment-Emergent adverse events (AEs) as assessed by AEs collection
Time Frame: 12 weeks
|
Safety assessment of pitolisant based on adverse events (AEs) reporting during the treatment pe
|
12 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Olivier Bonnot, MD, Prof., Nantes hospital, Department of Child & adolescent psychiatry, Nantes, France
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Estimated)
October 1, 2023
Primary Completion (Estimated)
January 1, 2025
Study Completion (Estimated)
January 1, 2025
Study Registration Dates
First Submitted
July 12, 2023
First Submitted That Met QC Criteria
July 12, 2023
First Posted (Actual)
July 20, 2023
Study Record Updates
Last Update Posted (Actual)
July 20, 2023
Last Update Submitted That Met QC Criteria
July 12, 2023
Last Verified
July 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- P21-01 / BF2.649
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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-
Centre Hospitalier Intercommunal Robert BallangerRecruitingAutism Spectrum Disorder (ASD)France
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BioprojetCompletedNarcolepsy | CataplexyFrance
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