Study Announcement: Poise 1

The OI Foundation would like to bring your attention to a phase 1 study of a new medical treatment being conducted by Sanofi. Sanofi is seeking participants from the OI community.

Please take a moment to read the following message from Sanofi:

Sanofi is conducting an early phase study in adults with OI Types I and IV with an anti-TGFb antibody called SAR439459. This study is called Poise 1 and is a Phase 1 study*. This study involves a single administration of SAR439459 given intravenously (IV) into the arm, with a 6-month follow period.

At this early stage in development, Sanofi is recruiting a limited range of study participants but we do anticipate expanding enrollment criteria in future studies. Participants in the Poise 1 study are not likely to experience benefits from SAR439459, and 25% will receive a placebo, but all participants will help with the scientific understanding of OI and SAR439459 as we prepare for future long-term studies. The study also includes digital, non-invasive strategies to better understand how OI patients move and are active throughout the day.

TGFb signaling is an important part of the bone remodeling environment, playing a role in the balance of forces which remove and build new bone. In OI, signaling related to TGFb is dysregulated and Sanofi is currently the only company pursuing this specific approach to treat OI.  A molecule similar to SAR439459, called fresolimumab, has been shown to increase bone mineral density in some Type IV OI patients after a single dose.

All participants will be reimbursed for travel and accommodations involved in visiting study sites during the study duration, including airfare for those who are not within driving distance of a study site.  Please see the study brochure for more information about where the study sites are located and for references to the information given above.

WHAT IS A PHASE 1 STUDY?

Interested in learning more about participating in Clinical Trials? Take a look at the OI Foundation’s What You Need To Know About Clinical Trials Factsheet.

• Male or female between 18 and 65 years old, with the exception of postmenopausal women
• Confirmed diagnosis of Osteogenesis Imperfecta Types I or IV, including documented genetic mutation in the COL1A1 or COL1A2 genes. Sanofi will provide OI-specific genotyping if it is not already in the patient’s medical history.
• Have experienced ≥ 2 bone fractures since the age of 18 OR at least 1 bone fracture in the last 10 years.