Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta
Status:
Recruiting
Trial end date:
2023-08-01
Target enrollment:
Participant gender:
Summary
Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People
with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and,
in adult years, hearing loss. OI can range from very severe to very mild. The current
standard-of-care for severe types of OI involves the use of IV medications (bisphosphonates)
and surgery to put rods in bones to strengthen them. These therapies, although often
life-saving, are new and very little is known about their long-term effects on bone and other
body systems.
Transforming growth factor beta (TGF-β) is a protein important in bone formation.
Fresolimumab is an antibody that can silence TGF-β . In studies with mice with OI, it has
been shown that silencing TGF-β can lead to higher bone mass, quality and strength. The
purpose of this study is to determine if fresolimumab is safe in the treatment of OI.
Phase:
Phase 1
Details
Lead Sponsor:
Baylor College of Medicine
Collaborators:
Genzyme, a Sanofi Company Hospital for Special Surgery, New York Hugo W. Moser Research Institute at Kennedy Krieger, Inc. Oregon Health and Science University Shriners Hospitals for Children University of California, Los Angeles University of Nebraska University of South Florida