Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta

Launched by AMGEN · Jul 24, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called romosozumab to see how well it works compared to a standard treatment, bisphosphonates, in children and adolescents with Osteogenesis Imperfecta (OI), a condition that makes bones fragile and prone to fractures. The main goal is to find out if romosozumab can reduce the number of fractures and improve bone strength over a 12-month period. Participants in this study will be closely monitored to assess their bone health and any fractures that occur during the treatment.

To be eligible for this trial, participants need to be between 5 and 17 years old, have a diagnosis of OI, and have experienced multiple fractures in the last two years. They should also meet specific health criteria, such as having a certain level of bone density. If someone joins the study, they can expect to receive either romosozumab or bisphosphonates and will attend regular visits for health check-ups and assessments. It’s important to note that participants and their guardians will need to provide consent before starting the study, and they should be aware of any other health conditions or medications that might affect their eligibility.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.
• • OR
• • Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
• • Ambulatory male and female children and adolescents, age 5 to \<18 years, including ambulatory with assistance as defined in the pediatric osteogenesis imperfecta (OI) population.
• • Clinical diagnosis of OI, defined as clinical history consistent with type I, III, or IV OI as determined by presence of expected phenotype (examples include: facial shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic features, fracture pattern) and lack of additional features unrelated to type I, III, or IV OI (eg, blindness, mental retardation, neuropathy, and craniosynostosis).
• • o If familial, also must be autosomal dominant.
• * Meets at least one of the following:
• • 3 or more fractures within the previous 2 years, or
• • 1 or more nonvertebral fracture(s) within the previous 2 years and at least 1 prevalent vertebral fracture, or
• • 2 or more prevalent vertebral fractures.
• Exclusion Criteria:
• • Disease Related
• • History of an electrophoresis pattern inconsistent with type I, III or IV OI.
• • History of known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1A1/COL1A2) causing OI or other metabolic bone disease.
• • History of congenital dislocation of the radial head, interosseous membrane calcification, or exuberant callus formation.