Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta
This research study is investigating a new treatment, romosozumab, for children and teenagers between 5 and 17 years old who have a condition called Osteogenesis Imperfecta (OI), also known as brittle bone disease. The main goal is to find out if romosozumab is more effective than the commonly used bisphosphonate medicines at reducing the number of broken bones over a 12-month period. Researchers will also look at whether romosozumab helps to make bones stronger. People taking part will be carefully monitored for any side effects. This study aims to find better ways to help young people with OI live healthier lives with fewer fractures.
एक नजर में
यह अध्ययन किस बारे में है?
This study is for children and teenagers with a condition called Osteogenesis Imperfecta, often known as brittle bone disease. This condition makes bones break more easily. Scientists are always trying to find new and better ways to help people with OI, and this study is looking at a new medicine called romosozumab.
Currently, many people with OI are treated with medicines called bisphosphonates, which help strengthen bones. This study wants to compare romosozumab with bisphosphonates to see which treatment works better. Researchers will specifically look at how many broken bones occur over 12 months and how much bone density (strength) improves in the participants' spines.
The information gathered from this study will help doctors understand if romosozumab could be a good new option for treating young people with OI, potentially leading to fewer fractures and better overall bone health. Making sure new treatments are both safe and effective is very important.
मुख्य निष्कर्ष
- This study is for children and teenagers (5-17 years old) with specific types of brittle bone disease (Osteogenesis Imperfecta).
- It compares a new medicine (romosozumab) to existing bone-strengthening medicines (bisphosphonates).
- The main aim is to see if the new medicine can prevent more broken bones and improve bone strength.
- Participation involves regular clinic visits, blood tests, and bone scans over 12 months.
- You won't know which medicine you're receiving to keep the study fair.
- All study treatments and assessments are provided at no cost, and you can leave at any time.
कौन पात्र हो सकता है?
To join this study, children and teenagers need to be between 5 and 17 years old. They must have a diagnosis of certain types of Osteogenesis Imperfecta (OI), specifically Type I, III, or IV. This means their brittle bone disease should fit a certain pattern, and they should not have other health conditions that are not usually part of these types of OI.
Participants should be able to walk, even if they need some help, and they must have experienced a certain number of broken bones recently. This could mean having three or more broken bones in the last two years, or at least one broken bone in the last two years along with a broken bone in their spine that happened earlier, or two or more broken bones in their spine at any time.
There are also some reasons why someone might not be able to join. For example, if tests show they have a different type of OI than what the study is looking for, or if their OI is caused by a different gene mutation. Also, certain other bone problems would prevent them from taking part.
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 aged between 5 and 17 years?
- Do you have a diagnosis of Osteogenesis Imperfecta Type I, III, or IV?
- Can you walk, even if you need some help?
- Have you had at least 3 broken bones in the last 2 years, or certain spine fractures?
- Do you not have other specific bone-related conditions that would prevent participation?
भागीदारी में क्या शामिल है?
If you decide to take part in this study, the total length of your participation will be 12 months. During this time, you would receive either the new medicine, romosozumab, or a bisphosphonate medicine, which is a standard treatment for OI. You and your doctors would not know which treatment you are receiving.
You would have regular visits to the clinic to receive your medication, have blood tests, and undergo bone scans (like X-rays or DEXA scans) to check your bone density. Doctors would also ask you about any new broken bones you might have and monitor your general health very closely throughout the study. All these tests and visits are designed to see how well the treatment is working and make sure you are safe.
संभावित जोखिम और लाभ
स्थान (65)
- Childrens Hospital of Alabamaसत्यापित पोस्टकोडबर्मिंघम, संयुक्त राज्य अमेरिका
- Phoenix Childrens Hospitalसत्यापित पोस्टकोडफीनिक्स, संयुक्त राज्य अमेरिका
- University of California, Los Angeles Interventional Clinical Trialsसत्यापित पोस्टकोडलॉस एंजेलेस, संयुक्त राज्य अमेरिका
- Nemours Hospital for Childrenसत्यापित पोस्टकोडविलमिंगटन, संयुक्त राज्य अमेरिका
- Nemours Childrens Hospitalसत्यापित पोस्टकोडऑरलैंडो, संयुक्त राज्य अमेरिका
- University of South Florida - Carol and Frank Morsani Center for Advanced Health Careसत्यापित पोस्टकोडटाम्पा, संयुक्त राज्य अमेरिका
- एमोरी विश्वविद्यालयसत्यापित पोस्टकोडअटलांटा, संयुक्त राज्य अमेरिका
- इंडियाना यूनिवर्सिटीसत्यापित पोस्टकोडइंडियानापोलिस, संयुक्त राज्य अमेरिका
- केनेडी क्रिगर संस्थानसत्यापित पोस्टकोडबाल्टीमोर, संयुक्त राज्य अमेरिका
- Boston Childrens Hospitalसत्यापित पोस्टकोडबोस्टन, संयुक्त राज्य अमेरिका
- Mayo Clinic Childrens Centerसत्यापित पोस्टकोडरोचेस्टर, संयुक्त राज्य अमेरिका
- Gillette Childrens Hospital and Clinic Saint Paulसत्यापित पोस्टकोडसेंट पॉल, संयुक्त राज्य अमेरिका
सामान्य प्रश्न
What is Osteogenesis Imperfecta (OI)?
OI is a condition that makes bones fragile and prone to breaking easily. It's often called 'brittle bone disease'.
What is romosozumab?
Romosozumab is a new medicine being studied to see if it can help make bones stronger and reduce fractures in people with OI.
What are bisphosphonates?
Bisphosphonates are a type of medicine commonly used to treat OI by helping to strengthen bones.
क्या मुझे पता चलेगा कि मुझे कौन सी दवा मिल रही है?
No, this is a 'blinded' study, meaning neither you nor your doctor will know if you are receiving romosozumab or a bisphosphonate. This helps ensure fair results.
अध्ययन कितने समय तक चलेगा?
If you join, you would be in the study for about 12 months.
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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