Ongoing clinical trials

OIFE supports and encourages the advancement into research to find new treatments, improve diagnosis and aid a better understanding of OI.
We strive to keep the OI community informed of what Clinical Trials and Research projects are ongoing. You can see a list below.

Ultragenyx-led ORBIT Study

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.

The clinical trial have sites participating in the following countries: Argentina, Australia, Canada, France, Germany, Italy, The Netherlands, Poland, Portugal, Turkey, UK and USA.

Learn more about the study at ClinicalTrials.gov or at the project webpage.

Ultragenyx-led COSMIC Study

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 the study at ClinicalTrials.gov or at the project webpage. Please reach out to trialrecruitment@ultragenyx.com if you are interested in participating.

The clinical trial have sites participating in the following countries: Brazil, Canada, France, Germany, Italy, The Netherlands, Poland and USA.

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

Poise 1 (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, 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.

AMGEN (Romosozumab vs. Bisphosphonates)

A multicenter phase 3 clinical trial is being conducted to study the efficacy and safety of a clinical trial drug compared to bisphosphonates in children and adolescents who have OI. The trial is open to eligible children from age 5 to less than 18 years of age who have a diagnoses of OI Type I through IV.

The clinical trial will have sites participating in the following countries: Canada, United States, Australia, Japan, Austria, Belgium, Hungary, Poland, Slovakia, Switzerland, France, Germany, Spain, Turkey and United Kingdom. For more information, please visit the clinical trial registry page.

Logo Amgen

The TOPaZ Trial

The purpose of the TOPaZ Trial is to investigate if a a two-year course of teriparatide (TPTD) followed by antiresorptive therapy with a single infusion of zoledronic acid (ZA) in adults with OI reduces the proportion of patients who experience a fracture as compared with standard care.

See more information at the project webpage or at clinicaltrials.gov

The MOI-A Trial (Losartan)

The purpose of the repurposing project called the MOI-A Trial is to investigate if the blood pressure drug Losartan will reduce bone turnover in older adolescents and adults with OI by reducing circulating levels of TGFβ and hence TGFβ pathway signalling. The project aim to identify the dose of Losartan that is effective in reducing circulating levels of CTX, a bone resorption (destruction) marker, without causing undue side effects. The project is one of the demonstrator project in the repurposing consortium Remedi4All.

The clinical trial will have sites participating in UK and Italy. Read more on the project website or on this page.

BOOSTB4

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

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Do you want to learn more about research in OI?

People who are interested in research and development are encouraged to check out OIFE’s resource page about research and development. You can also find an expansive list of OI-related research projects on www.clinicaltrials.gov.

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