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Aktiv men inte rekryterandeFAS3INTERVENTIONELL

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.

Överblick

Status
Aktiv men inte rekryterande
Fas
FAS3
Sponsor
Amgen
Inskrivningsmål
111
Starta
22 Apr 2024
Beräknad slutförande
30 jul 2027
Osteogenesis Imperfecta

Vad handlar denna studie om?

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.

Viktiga punkter

  • 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.

Vem kan vara berättigad?

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.

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 aged between 5 and 17 years?
  2. Do you have a diagnosis of Osteogenesis Imperfecta Type I, III, or IV?
  3. Can you walk, even if you need some help?
  4. Have you had at least 3 broken bones in the last 2 years, or certain spine fractures?
  5. Do you not have other specific bone-related conditions that would prevent participation?
Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

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.

Potentiella risker och fördelar

Taking part in a clinical study can offer potential benefits, such as gaining access to a new treatment that might improve your condition, and receiving close medical monitoring from specialists. However, there are also potential risks, including side effects from the study medicines, which will be carefully explained to you. The new medicine might not be more effective than current treatments, or it could even be less effective for some individuals. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Platser (65)

  • Childrens Hospital of Alabama
    Verifierat postnummer
    Birmingham, USA
  • Phoenix Childrens Hospital
    Verifierat postnummer
    Phoenix, USA
  • University of California, Los Angeles Interventional Clinical Trials
    Verifierat postnummer
    Los Angeles, USA
  • Nemours Hospital for Children
    Verifierat postnummer
    Wilmington, USA
  • Nemours Childrens Hospital
    Verifierat postnummer
    Orlando, USA
  • University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
    Verifierat postnummer
    Tampa, USA
  • Emory Universitet
    Verifierat postnummer
    Atlanta, USA
  • Indiana Universitet
    Verifierat postnummer
    Indianapolis, USA
  • Kennedy Krieger Institutet
    Verifierat postnummer
    Baltimore, USA
  • Boston Childrens Hospital
    Verifierat postnummer
    Boston, USA
  • Mayo Clinic Childrens Center
    Verifierat postnummer
    Rochester, USA
  • Gillette Childrens Hospital and Clinic Saint Paul
    Verifierat postnummer
    Saint Paul, United States

Vanliga frågor

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.

Kommer jag att veta vilken medicin jag får?

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.

Hur länge kommer studien att pågå?

If you join, you would be in the study for about 12 months.

Hur du får reda på mer

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