Autism Spectrum DisorderPhase 2NCT05953389
Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents
A Phase 2 study testing Pitolisant in people with Autism Spectrum Disorder, run by Bioprojet at 15 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 17 years, men only
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- Pitolisant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 15 sitesin 4 countries
- Study dates
- 20 Dec 2023to Nov 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
15 sites in 4 countries.
15 sites in 4 countries
Italy9 sites
IRCCS-Istituto delle Scienze Neurologiche di Bologna
Bologna, 40139
Istituto Scientifico IRCCS E. Medea
Bosisio Parini, 23842
Clinica Di Neuropsichiatria Infantile
Cagliari, 09121
IRCCS Fondazione Stella Maris
Calambrone, 56128
A.O.U Ospedali Riuniti di Foggia- Cardiologia (U.O.C)
Foggia, 71122
IRCCS Centro Neurolesi Bonino Pulejo
Messina, 98124
Universita degli Studi di Napoli Federico II
Naples, 80131
IRCCS Instituto Neurologico Casimiro Mondino
Pavia, 27100
Azienda ospedaliero-universitaria Senese
Siena, 53100
France3 sites
Centre Hospitalier Charles Perrens
Bordeaux, 33076
Nantes University Hospital
Nantes, 44093
CH le Rouvray
Sotteville-lès-Rouen, 76300
Spain2 sites
Hospital Universitari Vall d'Hebron
Barcelona, 08030
Hospital Universitario de Burgos
Burgos, 09006
About this study
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
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.
Read the 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.
- Conditions
- Autism Spectrum Disorder
- Being tested
- Pitolisant, Placebo
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PitolisantDrug
Also called BF2.649
histamine H3 receptor antagonist/inverse agonist
PlaceboDrug
Matching placebo of pitolisant tablets
| Study group | Role | What they receive |
|---|---|---|
| Pitolisant | Experimental | 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. Receives: Drug: Pitolisant |
| Placebo | Placebo comparator | One or two tablets of matching placebo per day for 12 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Social Responsiveness Scale Second Edition (SRS-2) total score
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.
Measured: 12 weeks
Secondary outcomes (2)
Vineland Adaptive Behavior Scale III (VABS III) total score
Instrument that measures socialization, communication, daily living skills, motor skills and maladaptive behavior of individuals with intellectual disabilities;
Measured: 12 weeks
Incidence of Treatment-Emergent adverse events (AEs) as assessed by AEs collection
Safety assessment of pitolisant based on adverse events (AEs) reporting during the treatment pe
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2023
- Study start20 Dec 2023
- Primary completionOct 2025 (estimated)
- Study completionNov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 62 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bioprojet
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Olivier Bonnot, MD, Prof., Principal investigator, Etablissement Public de Santé Barthélemy Durand, Sainte-Geneviève-des-Bois, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05953389
- Sponsor's protocol ID
- P21-01 / BF2.649
- Status last verified
- Oct 2025
- First submitted
- 12 Jul 2023
- Last updated
- 2 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05953389
- Acronym / sponsor ID
- P21-01 / BF2.649
- Conditions
- Autism Spectrum Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 62 participants (estimated)
- Lead sponsor
- BioprojetUniversity, hospital or other
- Study start
- 20 Dec 2023 (actual)
- Primary completion
- Oct 2025 (estimated)
- Study completion
- Nov 2025 (estimated)
- First posted
- 20 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pitolisant | Experimental | Drug: PitolisantOne 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. |
| Placebo | Placebo comparator | Drug: PlaceboOne or two tablets of matching placebo per day for 12 weeks. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PitolisantAlso: BF2.649histamine H3 receptor antagonist/inverse agonist | Drug | Pitolisant |
| PlaceboMatching placebo of pitolisant tablets | Drug | Placebo |
Primary outcome
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.
2 secondary outcomes
Vineland Adaptive Behavior Scale III (VABS III) total score
Time frame: 12 weeks
Incidence of Treatment-Emergent adverse events (AEs) as assessed by AEs collection
Time frame: 12 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Olivier Bonnot, MD, Prof. principal investigator
Etablissement Public de Santé Barthélemy Durand, Sainte-Geneviève-des-Bois, France
15 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pitolisant in people with Autism Spectrum Disorder, run by Bioprojet at 15 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 2 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Italy, France, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Social Responsiveness Scale Second Edition (SRS-2) total score (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2025; AzTrials last refreshed its copy on 1 Nov 2026.