Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
This research study is looking into a new nasal spray called carbetocin as a possible treatment for a common problem in people with Prader-Willi syndrome (PWS) called hyperphagia. Hyperphagia means having an extreme, constant feeling of hunger. The study is for people aged 5 to 30 years old with PWS who experience this strong urge to eat. For 12 weeks, some participants will receive the active carbetocin spray, while others will get a placebo (a spray with no medicine) to compare the effects. This is a "blinded" study, meaning neither the participants nor their doctors will know who is getting which spray. The goal is to find out if carbetocin can safely and effectively reduce the feeling of hunger in PWS.
במבט חטוף
על מה המחקר הזה?
You might be reading this because you, or someone you care for, is living with Prader-Willi syndrome (PWS) and the challenge of feeling constantly hungry, also known as hyperphagia. This can be really tough, and researchers are always looking for better ways to help. This particular study is trying out a new nasal spray medication called carbetocin.
Carbetocin is being tested to see if it can help reduce the intense hunger that people with PWS often experience. The study will last for 12 weeks, and it's set up in a way that allows us to fairly compare the new spray against a 'dummy' spray (a placebo). This means some participants will get the actual medicine, and others will get an identical-looking spray that doesn't contain any active ingredient. Neither you nor your study doctor will know which one you're receiving until the study is over. This is a common and important way to test if a new medicine truly works.
By taking part, you could contribute to important scientific understanding that might lead to new treatments for hyperphagia in PWS. The main goal is to find out if carbetocin is a safe and effective way to manage this challenging symptom. Hopefully, the results from this study will provide valuable information for the PWS community.
נקודות עיקריות
- This study is testing a nasal spray (carbetocin) for extreme hunger in Prader-Willi syndrome.
- It's for people with PWS aged 5 to 30 years old.
- Participants will use a nasal spray three times a day for 12 weeks.
- Some will get the real medicine, others a dummy spray (placebo), and neither you nor your doctor will know which.
- Close monitoring of your health and hunger levels will happen during study visits.
- Your participation could help find new ways to treat hyperphagia in PWS.
מי עשוי להיות זכאי?
This study is looking for people aged between 5 and 30 years old who have been diagnosed with Prader-Willi syndrome (PWS) through genetic testing. A key part of the study is that participants must have significant hyperphagia, meaning a strong, constant feeling of hunger that leads to seeking out food.
To join, you'll need to live with a caregiver who understands the study and can help you follow the study's instructions and attend appointments. There are also some health conditions or situations that would prevent someone from joining, such as certain heart, kidney, or liver problems, or having had certain recent surgeries. You also can't have other specific genetic conditions or be using certain medications that might interfere with the study, like some other nasal sprays.
Before you can officially join, the study doctors will do a thorough check to make sure it's safe and appropriate for you.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- Are you, or the person you care for, aged between 5 and 30 years old?
- Do you have a confirmed genetic diagnosis of Prader-Willi syndrome?
- Do you experience a strong, ongoing feeling of hunger (hyperphagia) that leads to food-seeking?
- Do you live with a caregiver who can help you follow study instructions and attend appointments?
- Have you avoided other nasal sprays or certain medications listed by the study team?
- Do you have good general health, without severe heart, kidney, or other major health issues?
מה כרוך בהשתתפות?
If you decide to take part in this study, you would be involved for about 12 weeks, plus some screening appointments beforehand. During the study, you'd be asked to use a nasal spray three times a day. You wouldn't know if it's the active medicine or a dummy spray, and neither would your doctor. You'd also need to attend several study visits where the team would do health checks, ask questions about your hunger and overall well-being, and collect information. These visits are important for monitoring your health and seeing how the spray might be affecting you. You'd also need to avoid using other nasal sprays or certain medications for the duration of the study.
סיכונים ותועלות פוטנציאליים
Locations (30)
- בית החולים לילדים של אלבמהמיקוד מאומתבירמינגהם, ארצות הברית
- בית החולים לילדים פיניקסמיקוד מאומתפניקס, ארצות הברית
- אוניברסיטת קליפורניה אירוויןמיקוד מאומתאורנג', ארצות הברית
- Stanford University School of Medicineמיקוד מאומתפאלו אלטו, ארצות הברית
- Rady Children's Hospital San Diegoמיקוד מאומתסן דייגו, ארצות הברית
- בית החולים לילדים קולורדומיקוד מאומתאורורה, ארצות הברית
- בית החולים לילדים אן ורוברט ה. לורי בשיקגומיקוד מאומתשיקגו, ארצות הברית
- University of Iowaמיקוד מאומתאיווה סיטי, ארצות הברית
- Boston Children's Hospitalמיקוד מאומתבוסטון, ארצות הברית
- Children's Mercy Hospitalמיקוד מאומתקנזס סיטי, ארצות הברית
- SSM Health/Saint Louis Universityמיקוד מאומתסנט לואיס, ארצות הברית
- Maimonides Medical Centerמיקוד מאומתBrooklyn, United States
שאלות נפוצות
What is hyperphagia?
Hyperphagia is an extreme, constant feeling of hunger that is common in people with Prader-Willi syndrome, leading to a strong urge to find and eat food.
מהו פלצבו?
A placebo is a 'dummy' treatment that looks exactly like the real medicine but contains no active ingredients. It's used in studies to fairly compare the effects of the new medicine.
האם אדע אם אני מקבל את התרופה האמיתית?
No, this is a 'blinded' study, meaning neither you nor your study doctor will know if you're receiving the carbetocin spray or the placebo until the study is over.
כמה זמן אהיה במחקר?
The main treatment part of the study lasts 12 weeks, plus some initial screening appointments to see if you're eligible.
האם אני יכול להפסיק להשתתף אם אני רוצה?
Yes, you are free to leave the study at any time, for any reason, without affecting your regular medical care.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
דיון
דיון קהילתי
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