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.
एक नजर में
यह अध्ययन किस बारे में है?
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.
मुख्य निष्कर्ष
- 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.
कौन पात्र हो सकता है?
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.
क्या यह अध्ययन आपके लिए उपयुक्त हो सकता है?
यह देखने के लिए इन त्वरित प्रश्नों का उत्तर दें कि क्या आप पात्र हो सकते हैं। यह केवल एक मार्गदर्शिका है — अंतिम निर्णय शोध टीम द्वारा लिया जाता है।.
- 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?
भागीदारी में क्या शामिल है?
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.
संभावित जोखिम और लाभ
स्थान (30)
- Children's of Alabamaसत्यापित पोस्टकोडबर्मिंघम, संयुक्त राज्य अमेरिका
- Phoenix Children's Hospitalसत्यापित पोस्टकोडफीनिक्स, संयुक्त राज्य अमेरिका
- University of California Irvineसत्यापित पोस्टकोडऑरेंज, संयुक्त राज्य अमेरिका
- Stanford University School of Medicineसत्यापित पोस्टकोडपैलो आल्टो, संयुक्त राज्य अमेरिका
- Rady Children's Hospital San Diegoसत्यापित पोस्टकोडसैन डिएगो, संयुक्त राज्य अमेरिका
- चिल्ड्रन हॉस्पिटल कोलोराडोसत्यापित पोस्टकोडऑरोरा, संयुक्त राज्य अमेरिका
- एन एंड रॉबर्ट एच. लुरी चिल्ड्रन हॉस्पिटल ऑफ शिकागोसत्यापित पोस्टकोडशिकागो, संयुक्त राज्य अमेरिका
- आयोवा विश्वविद्यालयसत्यापित पोस्टकोडआयोवा सिटी, संयुक्त राज्य अमेरिका
- बोस्टन चिल्ड्रन हॉस्पिटलसत्यापित पोस्टकोडबोस्टन, संयुक्त राज्य अमेरिका
- Children's Mercy Hospitalसत्यापित पोस्टकोडकैनसस सिटी, संयुक्त राज्य अमेरिका
- SSM Health/Saint Louis Universityसत्यापित पोस्टकोडसेंट लुइस, संयुक्त राज्य अमेरिका
- Maimonides Medical Centerसत्यापित पोस्टकोडBrooklyn, United States
सामान्य प्रश्न
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.
प्लेसबो क्या है?
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.
क्या मुझे पता चलेगा कि मुझे असली दवा मिल रही है?
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.
मैं अध्ययन में कितने समय तक रहूँगा?
The main treatment part of the study lasts 12 weeks, plus some initial screening appointments to see if you're eligible.
क्या मैं भाग लेना बंद कर सकता हूँ अगर मैं चाहूँ?
Yes, you are free to leave the study at any time, for any reason, without affecting your regular medical care.
और अधिक जानने के लिए कैसे पता करें
किसी अध्ययन में भाग लेने का निर्णय लेने से पहले हमेशा अपने जीपी या विशेषज्ञ से बात करें।.
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