Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

This medical study is looking into a new nasal spray called Carbetocin for adults who have Prader-Willi Syndrome. One of the main challenges for people with this condition is a constant feeling of hunger, called hyperphagia, which can be very difficult to manage. The aim of this study is to see if the Carbetocin nasal spray can help reduce this feeling of hunger and improve related behaviours. Participants will be randomly given either the active Carbetocin spray or a placebo, which looks and feels the same but contains no medicine. Researchers will then compare how both groups feel over a 12-week period. This is a Phase 3 study, which means it's a key step to confirm if the treatment is safe and effective.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Acadia Pharmaceuticals Inc.

Enrolment target

Start

28 May 2024

What is this study about?

This study is a clinical trial designed to test a new treatment for adults living with Prader-Willi Syndrome (PWS). PWS is a complex genetic condition, and one of its most challenging symptoms is a constant, overwhelming hunger, known as hyperphagia. This hunger can lead to significant difficulties, including unhealthy weight gain and challenging behaviours around food.

The treatment being studied is called Carbetocin, and it's given as a nasal spray. The researchers want to find out if this nasal spray can help reduce the intense feeling of hunger and improve how people manage their eating. To make sure the results are fair and accurate, some participants will receive the actual Carbetocin spray, while others will receive a 'placebo' spray. A placebo looks exactly like the real medicine but contains no active drug. Neither the participants nor the doctors will know who is getting which spray until the study is over.

The main goal is to see how much the hunger symptoms change after 12 weeks of treatment, using a special questionnaire designed to measure hyperphagia. Researchers will also look at overall improvements in the condition and how participants feel their symptoms have changed. This type of study, called a Phase 3 trial, is a very important step in determining if a new medicine is safe and effective enough to eventually be made available to more people.

Key takeaways

This study tests a new nasal spray (Carbetocin) for constant hunger (hyperphagia) in adults with Prader-Willi Syndrome.
It's a Phase 3 trial, a key step to see if the treatment works and is safe.
Participants will receive either the active spray or a dummy spray (placebo) for 12 weeks.
The main aim is to see if the spray reduces hunger symptoms.
Both men and women aged 18 and over with PWS and hyperphagia can join.
Participation involves clinic visits, questionnaires, and using the nasal spray.

Who may be eligible?

This study is looking for adult volunteers, aged 18 years and older, who have been diagnosed with Prader-Willi Syndrome. Both men and women are welcome to take part.

To ensure the study is safe and reliable, the research team will check other health information to make sure participants meet specific criteria. For example, they'll need to confirm that your hyperphagia is active and that you don't have other health conditions or are taking certain medications that might interfere with the study results or make the treatment unsafe for you.

It's important to discuss all your health conditions and current medications with the study team. They will carefully review everything to determine if this study is the right fit for you.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with Prader-Willi Syndrome?
Do you experience significant problems with constant hunger (hyperphagia)?
Are you able to attend regular clinic appointments for 12 weeks?
Are you willing to use a nasal spray as part of the study?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will first go through a screening process to make sure you meet all the requirements. Once confirmed, you will be randomly assigned to either receive the Carbetocin nasal spray or a placebo nasal spray (which looks identical but contains no medicine). You will use the nasal spray as instructed by the study team.

Over the course of 12 weeks, you will have several visits to the study clinic. During these visits, the research team will ask you questions about your hunger and eating behaviours, and you will complete questionnaires. They will also carry out assessments to see how your symptoms are changing and check your general health and safety. The total duration of your active participation in the study, including treatment and assessments, will be 12 weeks. There may also be some follow-up planned after this period.

Potential risks and benefits

Participating in this study might offer potential benefits, such as gaining access to a new treatment for hyperphagia that isn't yet widely available. You'll also receive careful medical monitoring throughout the trial. However, there are potential risks, as any medication can have side effects, and the study drug might not be effective for everyone. You will be fully informed of all known potential side effects before deciding to participate. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (5)

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Spain
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Germany
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Italy
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Belgium
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France

Common questions

What is hyperphagia?

Hyperphagia is a medical term for an abnormally excessive or insatiable appetite and consumption of food. For people with Prader-Willi Syndrome, it means a constant, overwhelming feeling of hunger.

What does 'placebo' mean?

A placebo is a substance or treatment that looks exactly like the real medicine but doesn't contain any active drug. It helps researchers understand if the new medicine is truly effective or if improvements are due to other factors.

Why is it important that neither I nor the doctors know if I'm getting the real medicine?

This is called a 'double-blind' study. It's done to ensure the results are unbiased. If people knew what treatment they were getting, it could accidentally influence how they report their symptoms or how doctors assess them.

What is a Phase 3 study?

A Phase 3 study is a crucial stage in drug development, where a new treatment is tested on a larger group of people to confirm its effectiveness, monitor side effects, and compare it to existing treatments or a placebo.

Will I have to pay to be in the study?

No, you will not have to pay to participate in the study. All study-related treatments and assessments are typically provided at no cost to you.