Exploratory study of pitolisant in children and adolescents with Autism Spectrum Disorders
Research type
Research Study
Full 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.
IRAS ID
1007904
Contact name
Pascale Vernade
Contact email
Sponsor organisation
Bioprojet Pharma
Eudract number
2023-503678-18
Research summary
Rationale: Autism refers to a range of conditions that are characterised by challenges with social skills, speech and nonverbal communication, and repetitive and restrictive behaviours. Children and adolescent living with autism experience a quality-of-life decline, especially regarding their social and emotional functioning.
Despite autism being considered a common neurological condition, there has been few pharmacological advancements in the field. Pitolisant represents a potential option for the treatment of autism social features of autistic subjects as observed in autistic animal model experiments.
The aim of the present clinical study is to evaluate the effect of pitolisant on the social and communication impairment of autistic male children and adolescents.
The main objective of this study is to evaluate the impact of pitolisant on the autistic social communication and interaction features as measured by the Social Responsiveness Scale Second Edition (SRS-2) total score.
Trial design
This is a Phase II exploratory study. Treatment (either pitolisant or placebo) will be assigned like flipping a coin by a computer 1 to 1, and neither the patient nor the trial doctor will be aware of the treatment assigned to the participant (blinding). Subjects are participating for 15 weeks including 2 weeks of screening period, 12 weeks of treatment period and 1 week of follow-up after the end of treatment. A total of 5 visits and 2 phone calls are planned for each participant during the study. Unscheduled visit by phone call or at site may be performed at any time during the study for safety reason, in case of undesirable event for doctor decision on the treatment.
Trial population:
Autistic male children and adolescent aged between 6 and 17, with a moderate social impairment, no learning disability, normal cardiac and hepatic function and no features of severe depression or epilepsy are eligible to this clinical study.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
23/WM/0149
Date of REC Opinion
12 Sep 2023
REC opinion
Further Information Favourable Opinion