Ultragenyx, in partnership with Mereo, are leading the Orbit clinical study, which is for individuals living with osteogenesis imperfecta (OI) Types I, III and IV. The purpose of this study is to investigate the efficacy and safety of setrusumab, a monoclonal antibody, for the potential treatment of OI in pediatric and young adult patients. The study aims to understand the potential reduction in fractures as well as other impacts of OI. Study participants are at least 5 but not yet 26 years of age, have a confirmed diagnosis of OI Types I, III, or IV, and are willing not to receive bisphosphonate therapy during the study.

Learn more about this study here.

The purpose of this study is to evaluate the effect of setrusumab, a monoclonal antibody, against intravenous bisphosphonates (IV-BP) in children living with types I, III or IV The study is focusing on reduction in fracture rate, including morphometric vertebral fractures, in younger pediatric participants as well as other parameters.

Currently enrolling patients aged 2 but less than 7 years old with OITypes I, III, and IV. Learn more about this study here. Please reach out to trialrecruitment@ultragenyx.com if you are interested in participating.

For more information on both of these trials, please reach out to trialrecruitment@ultragenyx.com or OIStudyInfo@ultragenyx.com

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, where the researchers evaluate the treatment’s safety and determine a safe dosage range.

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 will consider expanding enrollment criteria in future studies.

TGFb is a signaling molecule, which is a way cells communicate and coordinate with each other. Specifically, it is an important part of the bone remodeling environment, playing a role in the balance of forces which remove and build new bone. It even has roles in pain. In OI, signaling related to TGFb is dysregulated, so controlling that signaling with SAR439459 may be a way to influence symptoms caused by OI.

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 assessments in this study include digital, non-invasive strategies to better understand how OI patients move and are active throughout the day as well as direct patient feedback on daily activity and pain.

Study participants will be compensated for their travel and accommodation associated with visits to the study site. Such travel and accommodation can be arranged directly by Sanofi or a third-party service provider appointed by Sanofi.

For this early study, our two sites in Europe are located in France. The other sites are in the US, Canada and Australia. Additional information on participating study sites and how to contact Sanofi, if interested, is available under ClinicalTrials.gov NCT05231668.

Boost Brittle Bones Before Birth (BOOSTB4) is a research study investigating the possibility of using stem cell therapy as treatment for severe OI. The treatment involves transplanting stem cells into the baby affected with OI during pregnancy or soon after birth. The main aim of the BOOSTB4 clinical trial is to test whether the treatment is safe and effective. See more information at the project webpage or at clinicaltrials.gov