Active not recruitingPHASE2INTERVENTIONAL

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

This research study is investigating a new medication called Pitolisant for children and teenagers, specifically boys aged 6 to 17, who have Autism Spectrum Disorder (ASD). The main goal is to understand if Pitolisant can help improve some of the symptoms associated with ASD and if it is safe and well-tolerated. Participants will receive either Pitolisant or a placebo (a dummy pill with no active medicine) without knowing which one they are getting. We also want to learn how the body processes Pitolisant in this age group. This is the first time this medication has been studied in children and adolescents with ASD, and it will last for about 12 weeks for each participant.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Bioprojet

Enrolment target

Start

20 Dec 2023

Estimated completion

01 Nov 2025

Autism Spectrum Disorder

What is this study about?

This study, called a 'Proof of Concept' study, is designed to see if a new medication named Pitolisant could be helpful for boys aged 6 to 17 who have Autism Spectrum Disorder (ASD). It's a small-scale study, meaning it's an early step to gather information about whether the treatment works and is safe before larger studies are considered. The researchers want to find out if Pitolisant can improve some of the challenges associated with ASD and how well young people tolerate it.

Participants will be placed into one of two groups at random: one group will receive Pitolisant, and the other will receive a placebo, which looks like the study drug but contains no active medicine. Neither the participants, their families, nor the study team will know who is getting which – this is known as a 'double-blind' study and helps make the results fairer and more reliable. The study also aims to understand how the body absorbs, uses, and gets rid of Pitolisant, which is important for determining the right dose.

This is the very first time Pitolisant has been studied in children and teenagers with ASD. The study will last for 12 weeks and is taking place in several different locations to gather a wide range of information. The insights gained from this research are crucial for potentially developing new treatment options for young people with ASD in the future.

Key takeaways

This study evaluates an experimental medication, Pitolisant, for boys with ASD.
Participation involves receiving either Pitolisant or a placebo for 12 weeks.
The study aims to check if Pitolisant helps ASD symptoms and is safe.
It's a 'double-blind' study, meaning no one knows who gets the active medicine.
Close medical supervision and free study-related care are provided.
You can withdraw from the study at any time.

Who may be eligible?

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.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child a boy aged between 6 and 17?
Does your child have a confirmed diagnosis of Autism Spectrum Disorder?
Does your child have an IQ of 70 or higher?
Does your child NOT have a known genetic cause for their ASD (e.g., Fragile X Syndrome)?
Has your child NOT had any seizures after the age of 5?
Has your child NOT had serious thoughts or actions of self-harm in the last year?

Answer every question to see your result.

What does participation involve?

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.

Potential risks and benefits

Taking part in this study might offer some potential benefits, as your child would receive a new medication that could possibly help with their ASD symptoms under close medical supervision. However, there's no guarantee that Pitolisant will be effective for everyone, or that your child will receive the active medication, as some will get a placebo. As with any medication, there are potential risks and side effects, which will be fully explained by the study team. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (15)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Centre Hospitalier Charles Perrens
Verified postcode
Bordeaux, France
Nantes University Hospital
Verified postcode
Nantes, France
CH le Rouvray
Verified postcode
Sotteville-lès-Rouen, France
IRCCS-Istituto delle Scienze Neurologiche di Bologna
Verified postcode
Bologna, Italy
Istituto Scientifico IRCCS E. Medea
Verified postcode
Bosisio Parini, Italy
Clinica Di Neuropsichiatria Infantile
Verified postcode
Cagliari, Italy
IRCCS Fondazione Stella Maris
Verified postcode
Calambrone, Italy
A.O.U Ospedali Riuniti di Foggia- Cardiologia (U.O.C)
Verified postcode
Foggia, Italy
IRCCS Centro Neurolesi Bonino Pulejo
Verified postcode
Messina, Italy
Universita degli Studi di Napoli Federico II
Verified postcode
Naples, Italy
IRCCS Instituto Neurologico Casimiro Mondino
Verified postcode
Pavia, Italy
Azienda ospedaliero-universitaria Senese
Verified postcode
Siena, Italy

Common questions

What is Pitolisant?

Pitolisant is an investigational medication being studied for its potential effects on symptoms related to Autism Spectrum Disorder.

Will my child automatically get Pitolisant if we join?

No, participants will be randomly assigned to receive either Pitolisant or a placebo (a dummy pill), and neither you nor the doctors will know which one they are getting.

How long does the study last for each child?

Each child will participate in the study for approximately 12 weeks.

Are there any costs involved if my child joins?

No, participation in the clinical study, including all study-related treatments and assessments, will be free of charge.

Can we stop participating at any time?

Yes, you are free to withdraw your child from the study at any point, for any reason, without it affecting their regular treatment.