SAR439459 for Osteogenesis Imperfecta
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called SAR439459 for individuals with osteogenesis imperfecta (OI), a condition that causes brittle bones. The goal is to determine if a single dose of SAR439459 is safe and how it affects the body, particularly bone strength. Participants will receive either the actual treatment or a placebo (a harmless pill resembling the real treatment) and will be monitored for several months. Suitable candidates have either Type I or IV OI and a history of bone fractures. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications. However, if you are on certain treatments like denosumab, anti-sclerostin antibody, parathyroid hormone, bisphosphonates, or any experimental therapy for osteogenesis imperfecta, you must not have used them within 6 months before the study. Also, you should not be on therapeutic doses of anticoagulants or antiplatelet agents within 7 days before the study.
Is there any evidence suggesting that SAR439459 is likely to be safe for humans?
Research on SAR439459, a type of human-made antibody, remains in the early stages. This treatment has not yet been proven safe or effective for treating osteogenesis imperfecta (OI), a condition affecting bones. The current study examines how well patients tolerate a single dose of SAR439459. As a phase 1 study, the primary goal is to identify any side effects and understand how the body processes the drug.
Solid information on human reactions to SAR439459 is not yet available, as this is one of the first tests in humans. The study will monitor participants closely for 24 weeks after receiving the dose to check for any safety issues. Prospective participants would be among the first to help scientists understand how this treatment works in people.12345Why do researchers think this study treatment might be promising for Osteogenesis Imperfecta?
Most treatments for Osteogenesis Imperfecta, like bisphosphonates, focus on increasing bone density to prevent fractures. But SAR439459 works differently, targeting the disease at a molecular level by blocking the TGF-β pathway, which is involved in bone formation and repair. This novel approach could potentially lead to stronger, more resilient bones without the side effects associated with current therapies. Researchers are excited because this could mean more effective management of the condition with fewer complications.
What evidence suggests that SAR439459 might be an effective treatment for osteogenesis imperfecta?
Research has shown that SAR439459 is a human-made antibody targeting a protein called TGFβ, which influences bone formation. This is crucial for treating osteogenesis imperfecta (OI), a condition that causes fragile bones. In this trial, some participants will receive a single dose of SAR439459, while others will receive a placebo. Although no direct evidence yet supports SAR439459's effectiveness for OI, the treatment aims to strengthen bones by blocking TGFβ's role in weakening them. Early studies in other conditions have shown mixed results, and some concerns about risks exist. More research is needed to determine its effectiveness for OI.12367
Who Is on the Research Team?
Clinical Sciences & Operations
Principal Investigator
Sanofi
Are You a Good Fit for This Trial?
Adults with Osteogenesis Imperfecta (OI) types I or IV, who've had at least one bone fracture in the past decade or two since turning 18. They must weigh over 30 kg and not be pregnant, breastfeeding, or donating sperm. Participants need a documented genetic variant in COL1A1/COL1A2 and must consent to the study.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single IV dose of SAR439459 or placebo
Follow-up
Participants are monitored for safety, pharmacokinetics, pharmacodynamics, and bone mineral density over 24 weeks
What Are the Treatments Tested in This Trial?
Interventions
- SAR439459
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sanofi
Lead Sponsor
Paul Hudson
Sanofi
Chief Executive Officer since 2019
Degree in Economics from Manchester Metropolitan University
Christopher Corsico
Sanofi
Chief Medical Officer
MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University