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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.

Överblick

Status
Registrering genom inbjudan
Fas
FAS3
Sponsor
ACADIA Pharmaceuticals Inc.
Inskrivningsmål
160
Starta
11 Mar 2024
Beräknad slutförande
01 jun 2029

Vad handlar denna studie om?

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.

Viktiga punkter

  • 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.

Vem kan vara berättigad?

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.

Kan denna studie passa dig?

Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.

  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?
Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

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.

Potentiella risker och fördelar

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.

Platser (30)

  • Children's of Alabama
    Verifierat postnummer
    Birmingham, USA
  • Phoenix Barnsjukhus
    Verifierat postnummer
    Phoenix, USA
  • University of California Irvine
    Verifierat postnummer
    Orange, USA
  • Stanford University School of Medicine
    Verifierat postnummer
    Palo Alto, USA
  • Rady Barnsjukhus San Diego
    Verifierat postnummer
    San Diego, USA
  • Barnsjukhuset Colorado
    Verifierat postnummer
    Aurora, USA
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Verifierat postnummer
    Chicago, USA
  • University of Iowa
    Verifierat postnummer
    Iowa City, USA
  • Boston Barnsjukhus
    Verifierat postnummer
    Boston, USA
  • Children's Mercy Kansas City
    Verifierat postnummer
    Kansas City, USA
  • SSM Health/Saint Louis University
    Verifierat postnummer
    St Louis, USA
  • Maimonides medicinska centrum
    Verifierat postnummer
    Brooklyn, USA

Vanliga frågor

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.

Vem kan delta i denna studie?

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

Kommer jag att veta om jag får den aktiva medicinen?

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

Vad är huvudsyftet med denna studie?

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

Hur du får reda på mer

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