Osteogenesis imperfecta (OI), or brittle bone disease, is an inherited bone disorder that is present at birth. You might be wondering, what are symptoms of osteogenesis imperfecta? Well, they vary based on the type, from mild to severe. Type 1 includes easily broken bones, loose joints, muscle weakness, mild or no bone deformity, curved spine with potential to compress the vertebrae with age, possible hearing loss, and more. Type 2 is much more severe, with most cases causing death at birth or shortly after, but includes multiple broken bones before birth, severe bone deformities, very small stature, underdeveloped lungs, and so on. Type 3 is the most severe among those who survive the neonatal period and they typically suffer the most disabilities—easily broken bones from mild trauma, moderate to severe bone deformity, small stature, loose joints, poor muscle development in arms and legs, curved spine and compression or collapse of vertebrae, and more. There are a total of 8 types with similar symptoms, varying in severity.
Unfortunately, there is no cure, but thanks to clinical trials, certain treatments have been found to help children with this rare bone disease. In fact, there are still clinical trials occurring today, and their success relies on people signing up so the results are meaningful. Here are some new OI studies to consider signing up for today to make a difference in the lives of many children and individuals with OI:
1. Ultragenyx Studies
Ultragenyx is currently recruiting for two studies, ORBIT and COSMIC, to investigate the treatment of OI in pediatric and young adults. These two studies hope to determine whether setrusumab can safely and effectively restore bone production. If this works, this treatment could be life-changing for those with OI—there is potential for bones to become stronger, reduce fractures, and grow new bones. In both studies, patients will receive medical care from a study team that has experience with OI—the top priority is always the health of the patient
2. 7707: Use of clear aligners for treatment of dental malocclusion
This study conducted by the Brittle Bones Disorders Consortium (BBDC) is recruiting participants now. This study is to test the use of clear aligners for treatment of dental malocclusion in those with OI Types III and IV. The BBDC is looking to determine the safety of clear algiers, transparent plastic trays designed to fit over the teeth, to fix teeth misalignment and move teeth to the desired position over time. There is a goal of 57 participants, could this be you
3. Poise 1 (Sanofi)
Sanofi is conducting a study in adults with OI Types 1 and 4 to determine the success of an anti-TGFb antibody called SAR439459. This Phase 1 study will involve SAR439459 with an intravenous (IV) insertion in the arm with a follow-up 6 months later. Sanofi is currently recruiting a limited number of participants, but will increase the number in future studies. Additionally, this study involves digital, non-invasive studies to better understand how those with OI move and be active throughout the day.
It is highly encouraged that you sign up for a clinical trial as they can make a life-changing difference for many individuals suffering from OI. It helps foster clinical trials that are more transparent, prevents selective publication and selective reporting of outcomes, and not to mention it can help find successful treatments to improve the quality of life of those with OI. Consider signing up for one of the above mentioned clinical trials today.
