Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A 52-Week Open-Label Extension Study of Pimavanserin in Children and Adolescents with Irritability Associated with Autism Spectrum Disorder (ASD)

This trial is a year-long follow-up study for children and teenagers with autism spectrum disorder (ASD) who experience irritability. Participants will be given a medicine called Pimavanserin. This type of study is 'open-label', which means both the patients and the doctors know they are receiving the study medication. This stage of the research, called Phase II, aims to explore how well the medicine works and, more importantly, to carefully check its safety. The main goal is to monitor for any side effects. Researchers will also be looking to see if the medicine helps reduce irritability. This includes checking vital signs, weight, heart health, and carrying out physical exams and blood tests. It's about understanding the medicine's overall effects and safety over a longer period.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Acadia Pharmaceuticals Inc.

Enrolment target

191

Start

26 Aug 2024

Irritability associated with autistic disorder in children and adolescents with ASD

What is this study about?

This research study is looking into a medication called Pimavanserin for children and teenagers who have autism spectrum disorder (ASD) and experience irritability. Irritability can include things like temper tantrums, aggression, or self-harming behaviours. This study is an 'open-label extension', which means it's a continuation of earlier research, and everyone involved – the patients, their families, and the doctors – will know that the study medication, Pimavanserin, is being given. This is different from studies where some people get the medicine and others get a dummy pill (placebo) without anyone knowing who received what.

The main purpose of this particular study, which is in 'Phase II' of drug development, is to understand more about the safety of Pimavanserin when used over a longer period. While the medicine has been explored before, this phase helps researchers gather more information on its effects and how it might help with irritability. It's a key step in learning if the medicine could be a helpful option in the future.

Over 52 weeks (a full year), the research team will carefully monitor the health of participants. They'll be looking out for any unwanted effects or changes in health. They will also assess if there are any improvements in irritability, using specific tools to measure this. The overall aim is to gather a lot of information to make sure the medicine is safe and to see how effective it might be for young people with ASD-related irritability.

Key takeaways

This study is for children and teenagers with autism who have irritability.
The medicine being tested is called Pimavanserin.
The main aim is to check the medicine's safety over one year.
Participants will have regular health checks, including heart tests and blood tests.
It's an 'open-label' study, meaning everyone knows the study medicine is being given.

Who may be eligible?

This study is designed for children and teenagers who have been diagnosed with autism spectrum disorder (ASD) and are experiencing irritability. When we say 'irritability', we are referring to behaviours like temper tantrums, aggression, or hurting oneself.

Anyone of any age within the child and adolescent range, irrespective of their sex, could potentially be considered for this study. The study looks for participants who have been experiencing these specific challenges associated with their autism.

To be considered for this trial, you would typically need to have been part of an earlier study involving this medication, as this is an 'extension' study. Doctors involved in the trial would carefully check all aspects of a child's health and medical history to make sure the study is a good fit and safe 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.

Does your child have a diagnosis of autism spectrum disorder (ASD)?
Does your child experience irritability associated with their ASD (e.g., tantrums, aggression)?
Is your child a child or teenager?
Has your child previously taken part in a study for this drug?

Answer every question to see your result.

What does participation involve?

Taking part in this study means your child would receive the study medication, Pimavanserin, for a period of 52 weeks (one year). During this time, they would have regular check-ups and assessments. These appointments are designed to closely monitor their health and to see how they are responding to the medication.

These check-ups would include measuring vital signs like blood pressure and heart rate, checking their weight and body mass index (BMI), and performing 12-lead electrocardiograms (ECGs) to look at their heart's electrical activity. There would also be physical examinations, blood tests, urine tests, and hormone assessments. Doctors would also use specific questionnaires and scales to assess irritability, and other potential side effects.

The total duration of participating in this study would be approximately 52 weeks. You and your child would have ongoing contact with the study team throughout this period, with regular visits to the clinic for these assessments. You're always able to ask questions and discuss how your child is feeling.

Potential risks and benefits

Participating in a study like this means your child could potentially benefit from the study medication, Pimavanserin, which might help reduce their irritability. However, as with any medication, there's always a chance of side effects, which the study team will monitor very carefully. These can range from mild to more serious, and the study is specifically designed to understand these better. It's important to remember that 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 (5)

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

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Unverified
Spain
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Unverified
Poland
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Unverified
France
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Unverified
Italy
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Unverified
Hungary

Common questions

What is Pimavanserin?

Pimavanserin is the name of the medicine being studied. It's being investigated to see if it can help children and teenagers with autism who experience irritability.

What does 'open-label' mean?

'Open-label' means that everyone involved – the patients, their families, and the doctors – will know that the study medication is being given. There are no dummy pills in this particular study.

What is 'Phase II'?

Phase II is a stage in testing new medicines. It focuses on exploring how well the medicine works and, very importantly, checking its safety in a larger group of people than in earlier phases.

How long will the study last?

This study will last for 52 weeks, which is about one year. During this time, there will be regular check-ups and tests.

What kind of irritability are you looking at?

The study focuses on irritability that is linked to autism spectrum disorder. This can include challenging behaviours like temper tantrums, aggression, or self-harming behaviours.