Genetics - Pediatric, Phase III

Setrusumab vs bisphosphonates in children from 2 to < 7 years old with osteogenesis imperfecta (OI)

The study is sponsored by Ultragenyx.
The purpose of the study is to evaluate the new medication, setrusumab, an anti-sclerostin monoclonal antibody, by comparing its effects to IV-Bisphosphonate (BP) therapy in children with OI Types I, III, or IV who have a recent history of fractures.
After a screening visit to determine eligibility, subjects are randomized to either receive setrusumab at their Baseline Visit or to resume their IV BP therapy, for up to 24 months. At the end of this controlled comparison period, subjects assigned to the IV-BP arm will be allowed to transition to setrusumab. During this Extension Period, all subjects will be offered setrusumab for a minimum of 12 months.

The following procedures are scheduled to occur at various visits during the course of the study – physical examination, skeletal assessments including X-rays and bone density, questionnaires, audiometry and assessment of blood and urinary tests. Setrusumab will be administered monthly to those children randomized to that treatment.

Contact Information

For more information about this study, including how to volunteer, contact Thomas Carpenter, MD

You can help our team find trials you might be eligible for by creating a volunteer profile in MyChart. To get started, create a volunteer profile, or contact helpusdiscover@yale.edu, or call 877.978.8343 for more information.

Principal Investigator