An Exploratory, Multicenter, Randomized, double-blind, placebo-controlled study evaluating the effect and safety of pitolisant in children and adolescents with Autism Spectrum Disorders.

Trial details

Who runs it

Bioprojet Pharma

Trial code

EU 2023-503678-18-00

Record updated

2025. szeptember 30.

Original description

The primary objective of this study is to evaluate at the end of treatment period the effect of pitolisant treatment on the ASD social communication and interaction deficit core symptom as assessed by the SRS-2 Total scoreRead full original descriptionShow less

The primary objective of this study is to evaluate at the end of treatment period the effect of pitolisant treatment on the ASD social communication and interaction deficit core symptom as assessed by the SRS-2 Total score

Inclusion: 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 study; Participants have a diagnosis of autism spectrum disorders as per the 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; Participants should benefit from appropriate health insurance system (for French participant only); 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); The participant agrees to follow the contraceptive requirements detailed in the protocol; Male and female children and adolescents aged from 6 to 17 inclusive for the duration of study participation; Participants have an Intelligence Quotient (IQ) ≥ 70 using Wechsler Intelligence Scale (WAIS-IV, WISC-IV or WISC-V) confirmed by historical data within the last 3 years or at screening (WAIS-IV or WISC-V). If total IQ score is unconclusive due to heterogenicity of sub-scores, participant should have at minimum both sub-scores for verbal comprehension and perceptual reasoning ≥ 70; Social Responsiveness Scale Second Edition (SRS-2) total T-score ≥ 66 at screening and baseline; 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 study; Participants’ language, hearing and vision must be compatible with the study assessments as judged by the investigator; Participants must have the ability to swallow the investigational medicinal product (IMP).

Exclusion: 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; Any positive test of abuse drugs at screening; Participants taking part in another interventional study and/or the use of any investigational therapy within the 30 days prior to screening; Known hypersensitivity to the investigational medicinal product including active substance and excipients; 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; Other active clinically significant illness, infection, active acid-related gastric disorders, neoplastic pathology within the last 3 years, or any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator could interfere with the study conduct or counter-indicate the study treatments or place the participant at risk during the study or compromise his study participation; 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; Participant with a 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; Participants with clinically relevant abnormal laboratory values suggesting an unknown disease and requiring further clinical investigation per investigator judgement; Prior (within the last 4 weeks prior to screening) or current therapy with strong CYP2D6 inhibitor drugs, strong CYP3A4 inducer drugs, CYP3A4 substrates having a narrow therapeutic margin, QT-prolonging medicinal products or medicinal products known to increase the risk of repolarization, 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; Participants with impaired renal function (stages 2 to 4 according to the international classification of chronic kidney disease, i.e. creatinine clearance between 15 and 89 ml/min according to the CKD-EPI formula).); Participants with moderate hepatic impairment (Child Pugh B) or with any other hepatic significant abnormality in the physical examination or ALAT or ASAT ≥ 2x Upper Limit of Normal (ULN) for age at laboratory results; Participants with cardiac disorders including clinically significant deviation from normal on 12-lead ECG that results as per investigator judgement in an active medical problem, or with a QTcF > 450ms at screening, where they are co-medicated or not with QT-prolonging medicinal products or medicinal products known to increase the risk of repolarization disorders; Any participant presenting congenital galactosemia, glucose-galactose malabsorption or lactase deficiency due to the presence of lactose in the investigational medical product (IMP); History or current diagnosis of epilepsy or any seizure occurring after the age of 5; Participant with a severe obesity or severe anorexia at screening as calculated by a Body Mass Index (BMI) at or above the 97th percentile and at or below the 3rd percentile, respectively for the same age; Participant with a family relationship to a person involved in the study at the investigator’s site, at the Clinical Research Organisation (CRO) or at the Sponsor; Pregnant or lactating woman.

EU CTIS 2023-503678-18-00