Active not recruitingPHASE3INTERVENTIONAL

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.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Amgen

Enrolment target

111

Start

22 Apr 2024

Estimated completion

30 Jul 2027

Osteogenesis Imperfecta

What is this study about?

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.

Key takeaways

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.

Who may be eligible?

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.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

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?

Answer every question to see your result.

What does participation involve?

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.

Potential risks and benefits

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)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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

Common questions

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.

Will I know which medicine I'm getting?

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.

How long will the study last?

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

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.