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Enrolling by invitationPHASE3INTERVENTIONAL

OLE Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

This study is investigating a nasal spray called carbetocin for people aged 5 to 30 with Prader-Willi Syndrome (PWS). People with PWS often experience excessive hunger, called hyperphagia. This study is an extension of a previous one, focusing on the long-term safety and whether participants can tolerate the spray over an extended period. Researchers want to understand if daily use of the carbetocin nasal spray (3.2 mg, three times a day) helps manage this challenging symptom. Participants will be those who have already completed the initial study. The aim is to gather more information on its effects when used for a longer time.

At a glance

Status
Enrolling by invitation
Phase
PHASE3
Sponsor
ACADIA Pharmaceuticals Inc.
Enrolment target
160
Start
11 Mar 2024
Estimated completion
01 Jun 2029

What is this study about?

This study is known as an Open-Label Extension (OLE) study. This means that everyone taking part will receive the carbetocin nasal spray, and both they (and their caregivers) and the study doctors will know what treatment they are receiving. It's designed to follow on from an earlier study where some participants might have received a placebo (a dummy spray with no active medicine) instead of carbetocin. This open-label format allows researchers to look closely at the medication's longer-term effects.

The main goal of this particular study is to see how safe the carbetocin nasal spray is when used over a longer period, and how well people can tolerate it. 'Tolerability' refers to how comfortable people are with the treatment and whether they experience any side effects. Researchers will be monitoring participants closely for any unexpected reactions or difficulties with using the spray.

By gathering information on long-term safety and tolerability, this study helps us understand more about carbetocin as a potential treatment for the intense, constant hunger (hyperphagia) experienced by individuals with Prader-Willi Syndrome. This condition can significantly impact daily life, and finding safe and effective ways to manage it is very important.

Key takeaways

  • Focuses on long-term safety and tolerability of carbetocin nasal spray.
  • For individuals aged 5-30 with Prader-Willi Syndrome.
  • Participants must have completed a previous study (ACP-101-302).
  • Aims to help manage excessive hunger (hyperphagia).
  • Requires active involvement of a caregiver.
  • All participants will receive the active carbetocin treatment.

Who may be eligible?

This study is for individuals aged between 5 and 30 years old who have Prader-Willi Syndrome. To be considered, you must have already successfully completed a previous study involving carbetocin (Study ACP-101-302) and met all the entry rules for that initial study. Your doctor would also need to believe that you would benefit from continuing to receive the carbetocin treatment for a longer time.

You would also need to live with a caregiver who is willing and able to understand and follow all the study procedures and attend all scheduled appointments. This is important to ensure the study runs smoothly and safely for everyone involved.

There are certain health conditions that would prevent someone from joining, such as a history of stroke, serious brain injury, epilepsy, or frequent severe migraines. If you have active severe mental health symptoms, or a history of significant mental health issues like psychosis or suicide attempts, you would not be able to participate. Also, if you have certain heart conditions or have had problems tolerating the study medication or procedures in the previous study, you might not be suitable.

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.

  1. Are you between 5 and 30 years old?
  2. Do you have Prader-Willi Syndrome?
  3. Have you completed the previous ACP-101-302 study?
  4. Does your doctor think you might benefit from continuing this treatment?
  5. Do you live with a caregiver who can help you with the study?
Answer every question to see your result.

What does participation involve?

If you join this study, you will continue to use the carbetocin nasal spray three times a day. You and your caregiver will need to attend regular appointments at the study clinic. These visits will involve health checks, such as measuring your vital signs (like blood pressure and heart rate), and ECGs (heart rhythm tests) to monitor how your heart is doing. The study team will also be asking about any side effects you might experience and how well you are managing with the nasal spray. The exact total duration of your participation will depend on the study design, but it is an open-label extension, meaning it's designed for long-term follow-up for those who completed the initial study.

Potential risks and benefits

Participating in this study might offer the potential benefit of continued access to a treatment that could help manage excessive hunger associated with Prader-Willi Syndrome, and contribute to scientific understanding of its long-term effects. However, as with any medication, there's a risk of side effects, which will be carefully monitored by the study team. You will be kept fully informed of any known risks. Remember, participation is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (30)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Children's of Alabama
    Verified postcode
    Birmingham, United States
  • Phoenix Children's Hospital
    Verified postcode
    Phoenix, United States
  • University of California Irvine
    Verified postcode
    Orange, United States
  • Stanford University School of Medicine
    Verified postcode
    Palo Alto, United States
  • Rady Children's Hospital San Diego
    Verified postcode
    San Diego, United States
  • Children's Hospital Colorado
    Verified postcode
    Aurora, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Verified postcode
    Chicago, United States
  • University of Iowa
    Verified postcode
    Iowa City, United States
  • Boston Children's Hospital
    Verified postcode
    Boston, United States
  • Children's Mercy Kansas City
    Verified postcode
    Kansas City, United States
  • SSM Health/Saint Louis University
    Verified postcode
    St Louis, United States
  • Maimonides Medical Center
    Verified postcode
    Brooklyn, United States

Common questions

What is Carbetocin?

Carbetocin is a medication being studied in a nasal spray form for people with Prader-Willi Syndrome.

What is 'Hyperphagia'?

Hyperphagia is a medical term for excessive, insatiable hunger, which is a common and challenging symptom for people with Prader-Willi Syndrome.

Who can join this study?

This study is for individuals aged 5-30 with Prader-Willi Syndrome who have already finished a specific previous study (ACP-101-302).

Will I know if I'm getting the active medicine?

Yes, this is an 'open-label' study, meaning everyone, including you and the study team, will know you are receiving the carbetocin nasal spray.

What is the main purpose of this study?

The main purpose is to evaluate the long-term safety and how well people can tolerate the carbetocin nasal spray for Prader-Willi Syndrome.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "OLE Study of Carbetocin Nasal Spray for the Treatment of Hyp…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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