Author: OI Foundation

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.

A number of recent surveys have shown that approximately 60% of individuals with OI report that they suffer from chronic pain. To increase research around pain management, the OI Foundation and Osteogenesis Imperfecta Federation Europe (OIFE) have come together to form the Pain and OI Project, an international resource group made up of a variety of professionals and representatives of OI.  The project goal is to make recommendations on how to assess and measure pain, to promote educational activities about OI to target groups, and to construct a toolbox for children and adults to help manage chronic pain. Learn more about this project at https://oife.org/2022/10/28/oife-pain-oi-project/

Michael Geisman Fellowship Grant

The Michael Geisman Fellowship Grant program awards funding to post-doctoral trainees who are currently working on projects with clear relevance to osteogenesis imperfecta (OI), or who have projects that will enable them to develop expertise in OI research.

Applicant Requirements:

• Applicant must hold an MD, DDS, DO, or PhD, and be appointed at the level of a post-doctoral trainee, or equivalent, within an academic institution.
• Applicant should have completed their Ph.D. or clinical training within the past five (5) years.

Fellowship Guidelines:

• Michael Geisman Fellowship awards provide up to $50,000 per year. It is the intention of the OI Foundation that grant monies be used to fund actual costs related to the research being performed including Fellow salary, fringe benefits, and supplies.
• Fellowship awards are for one year; a second year of funding may be approved based upon satisfactory performance during the first year of funding.
• Research must be done under the supervision of mentor with training and experience in osteogenesis imperfecta research or research in a related field.

How to Apply:

1. Complete application (pg. 2-8)
2. Mentor of applicant must submit a copy of his/her NIH biosketch and a letter of recommendation on behalf of the trainee, which also confirms that the mentor will supervise the trainee’s research.
3. Applications require two additional letters of recommendation from scientists or clinicians who can comment upon the applicant’s training, ability, and potential to develop expertise in OI research.
4. Submit application, reference letters, and mentor biosketch as PDF documents to sconnors@oif.org NO LATER THAN November 30, 2022.

If you have any questions, please contact Stacie Connors at sconnors@oif.org.

Click here for the 2022 Michael Geisman Fellowship application.