Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents

To join this study, we are looking for boys who are between 6 and 17 years old. They must have been diagnosed with Autism Spectrum Disorder (ASD) by a doctor, using specific recognised assessments like the DSM-5 criteria, and further confirmed by ADOS-2 or ADI-R tests. It's also important that they, along with their parent or guardian, are happy and willing to take part and understand what the study involves.

Participants should also have an Intelligence Quotient (IQ) of 70 or higher, as measured by standard tests, and a specific score on the Social Responsiveness Scale Second Edition (SRS-2), which helps us understand social communication skills. This ensures that the study includes individuals who meet specific criteria for the research.

There are also some reasons why someone might not be able to join. For example, if there's a known genetic cause for their ASD (like Fragile X syndrome), or if they have a history of seizures after the age of 5. The study also cannot include individuals who have had recent thoughts or actions of self-harm, or certain heart or liver problems, as these could make the study medication unsafe for them.

If you or your child decides to take part in this study, it would involve several visits to the study clinic over a period of about 12 weeks. During these visits, the study team will carry out various assessments. These might include detailed discussions about your child's well-being, physical check-ups, and taking blood or urine samples to understand how the body is reacting to the medication. They might also complete questionnaires or specific tasks to measure how their ASD symptoms are progressing.

Your child will be given either the study medication, Pitolisant, or a placebo (a dummy pill) to take regularly as instructed by the study doctor. Neither you nor the study team will know which one your child is receiving. Throughout the study, the team will closely monitor your child's health and any side effects. There will be regular follow-up calls or visits to ensure everything is progressing safely. The total duration of active participation in the study for your child will be approximately 12 weeks.