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.
Em resumo
Sobre o que é este estudo?
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.
Pontos principais
- 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.
Quem pode ser elegível?
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.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- 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?
O que envolve a participação?
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.
Riscos e benefícios potenciais
Locations (65)
- Childrens Hospital of AlabamaCódigo postal verificadoBirmingham, Estados Unidos
- Phoenix Childrens HospitalCódigo postal verificadoPhoenix, Estados Unidos
- University of California, Los Angeles Interventional Clinical TrialsCódigo postal verificadoLos Angeles, Estados Unidos
- Nemours Hospital for ChildrenCódigo postal verificadoWilmington, Estados Unidos
- Nemours Childrens HospitalCódigo postal verificadoOrlando, Estados Unidos
- University of South Florida - Carol and Frank Morsani Center for Advanced Health CareCódigo postal verificadoTampa, Estados Unidos
- Universidade EmoryCódigo postal verificadoAtlanta, Estados Unidos
- Indiana UniversityCódigo postal verificadoIndianápolis, Estados Unidos
- Kennedy Krieger InstituteCódigo postal verificadoBaltimore, Estados Unidos
- Boston Childrens HospitalCódigo postal verificadoBoston, Estados Unidos
- Mayo Clinic Childrens CenterCódigo postal verificadoRochester, Estados Unidos
- Gillette Childrens Hospital and Clinic Saint PaulCódigo postal verificadoSaint Paul, United States
Perguntas frequentes
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.
Saberei qual medicamento estou recebendo?
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.
Quanto tempo durará o estudo?
If you join, you would be in the study for about 12 months.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.