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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.

Auf einen Blick

Status
Aktiv, aber nicht rekrutierend
Phase
PHASE3
Sponsor
Amgen
Einschreibeziel
111
Starten
22. Apr 2024
Geschätzte Fertigstellung
30 Jul 2027
Osteogenesis Imperfecta

Worum geht es in dieser Studie?

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.

Wichtige Erkenntnisse

  • 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.

Wer könnte berechtigt sein?

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.

Könnte diese Studie für Sie geeignet sein?

Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.

  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?
Beantworten Sie jede Frage, um Ihr Ergebnis zu sehen.

Was beinhaltet die Teilnahme?

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.

Potenzielle Risiken und Vorteile

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.

Locations (65)

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

Häufige Fragen

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.

Werde ich wissen, welches Medikament ich bekomme?

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.

Wie lange wird die Studie dauern?

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

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