P21-01: A 12-week, Multicenter, Randomized, double-blind, placebo-controlled, parallel-group, Exploratory Clinical Trial Evaluating the efficacy and safety of pitolisant in children and adolescents with Autism Spectrum Disorders
Overview
HELP US UNDERSTAND AUTISM SPECTRUM DISORDERS
STUDY P21-01
A research study to evaluate the effect of pitolisant on autism spectrum disorders main symptoms
What does the study involve?
- First on-site visit (including a blood draw) to confirm participating may be worthwhile for your child
- Second on-site visit two weeks later – Beginning of the treatment phase
- Three on-site visits 3, 8 and 12 weeks later (end of the treatment period, including one other blood draw)
- Follow-up call 3 weeks later = end of study
Why should your child join?
• It is hoped that pitolisant may increase your child’s awareness and help improve their ability to interact with other people
• Pitolisant is already used in children for another condition and well tolerated
• The main (but rare) side effects are headache and insomnia
Your child may be able to participate if he/she:
• Is aged from 6 to 17 years old
• Doesn’t suffer from intellectual difficulties
Your doctor will tell you which medications are authorized or not during the study.
To learn more about the study
Please contact the doctor in charge of your child’s follow-up (regarding his/her autism spectrum disorder)
Are you interested in taking part in this study?
Find out more by filling out this form
Planned end date
30 Nov 2025 00:00Conditions
Autism Spectrum DisorderInclusion Criteria
1. Participants (if possessing adequate understanding, in the investigator's opinion) and their parent(s)/legal guardian(s) are willing and able to give informed assent and consent for participation in the trial.
2. Male children and adolescents ≥ 6 and < 18 years old for the duration of study participation.
3. Participants have a diagnosis of autism spectrum disorders as per the Diagnostic and Statistical Manual of Mental Disorders fifth edition (DSM-5) criteria confirmed by ADOS-2 or ADI-R historical diagnosis within the last 3 years prior to screening or by ADI-R at screening.
4. Participants have an Intelligence Quotient (IQ) ≥ 70 using Wechsler Intelligence Scale (WAIS-IV or WISC-V) confirmed by historical data within the last 3 years or at screening. If total IQ score is inconclusive due to heterogenicity of sub-scores, participant should have at minimum both sub-scores for verbal comprehension and perceptual reasoning ≥ 70.
5. Social Responsiveness Scale version 2 (SRS-2) total T-score ≥ 66 at screening and baseline.
6. All ongoing medications or interventions (except those listed as prohibited in “previous and concomitant medications/therapy” section) for ASD related symptoms must have been stable for at least 4 weeks prior to screening, and the participant/caregiver must be willing to maintain a stable regimen throughout the trial.
7. Participants’ language, hearing and vision must be compatible with the study assessments as judged by the investigator.
8. Participants must have the ability to swallow the investigational medicinal product (IMP).
9. Participant has a care provider who can reliably bring participant to clinic visits, provide trustworthy ratings, support participant with study compliance requirements, and interacts with the participant on a regular basis (e.g., spends at minimum 3 hours/ day and 4 x /week).
10. Participants should benefit from appropriate health insurance system (for French participant only).
Exclusion Criteria
1. Participants with previous genetic diagnosis of ASD-known "syndromic" ASD (e.g. Fragile X syndrome, Angelman syndrome, Prader-Willi, Rett's syndrome, tuberous sclerosis, Dup15q syndrome).
2. Participants having a diagnosis other than ASD that dominates the clinical presentation (e.g., Attention-Deficit/Hyperactivity Disorder (ADHD), anxiety disorder, depressive disorder) per investigator judgement.
3. Participant with a history of suicidal behaviour or suicidal ideation in the past 12 months, or a positive answer to questions 4 or 5 (current or over the last 6 months) on the Columbia-Suicide-Severity Rating Scale (C-SSRS) at screening or baseline, and/or is a significant risk for suicidal behaviour per investigator judgement.
4. History or current diagnosis of major depressive episode or severe psychiatric disorders, e.g. schizophrenia, bipolar disorder, organic brain syndrome or psychosis, preventing the participant from completing the study assessments per investigator judgement.
5. History or current diagnosis of epilepsy or any seizure occurring after the age of 5.
6. Participants with a clinically significant deviation(s) from normal on 12-lead ECG that results in an active medical problem, per investigator judgement or with a QTcF > 450ms at screening.
7. Participants with severe hepatic impairment (Child Pugh C) or with any other hepatic significant abnormality in the physical examination or ALT or AST ≥ 2 x ULN for age at laboratory results.
8. Participants with clinically relevant abnormal laboratory values suggesting an unknown disease and requiring further clinical investigation per investigator judgement.
9. Any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the trial.
10. Other active clinically significant illness, infection, acid-related gastric disorders, or neoplastic pathology within the last 3 years which could interfere with the study conduct or counter-indicate the study treatments or place the participant at risk during the trial or compromise his study participation.
11. Known or suspected history of alcohol or illicit drug (e.g. cannabis, amphetamines or opioids when not prescribed and used under medical supervision) or any history of cocaine abuse/dependence prior to screening according to investigator judgement.
12. Any positive test of abuse drugs at screening.
13. Prior (within the last 4 weeks prior to screening) or current therapy with strong CYP2D6 inhibitor drugs, strong CYP3A4 inducer drugs, antipsychotics medications, first-generation antihistamines (H1-antagonists) crossing the blood-brain barrier and tricyclic antidepressants as detailed in the “previous and concomitant medications/therapy” section.
14. Known hypersensitivity to the investigational medicinal product including active substance and excipients
15. Any participant presenting congenital galactosemia, glucose-galactose malabsorption or lactase deficiency.
16. Participants taking part in another interventional study and/or the use of any investigational therapy within the 30 days prior to screening.
17. Participant with a family relationship to a person involved in the study at the investigator’s site or at the Sponsor.