At a glance
ClinicalIndex Comparison Record- ✓Age 18 years or older
- ✓Diagnosed with moderate-to-severe osteogenesis imperfecta based on clinical features
- ✓Genetic confirmation: glycine substitution in COL1A1 or COL1A2, or pathogenic variants in CRTAP, PPIB, or LEPRE1 (or clinical assessment if genetic data unavailable at screening)
- ✓Females of childbearing potential must have negative urine pregnancy test and agree to acceptable birth control for entire study duration
- ✕Fracture within 3 months prior to screening
- ✕Known unhealed fracture involving a long bone
- ✕Personal history of basal cell carcinoma, squamous cell carcinoma, keratoacanthomas, or any other cancer (recent or remote)
- ✕Current clinically significant infection
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Multicenter Study to Evaluate Safety of Fresolimumab in Adults With Moderate-to-severe Osteogenesis Imperfecta
In Brief
A Phase 1 clinical trial evaluating Fresolimumab for Osteogenesis Imperfecta. Completed, enrolled 11 participants across 2 sites.
Detailed 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.
Study Details
Timeline
Interventions
The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.