Complete the new OI and Pain Survey!

The OI Foundation and OI Federation of Europe (OIFE) have partnered on a new project investigating the experience of pain for people with OI. The following survey investigates the experience of pain for people with OI. This survey is for anyone 18 years old or older and was written with input from OI experts from North America and Europe.

Through this survey, the international Pain and OI Project hopes to learn more about the different types of pain people experience, severity of pain and frequency, and how it impacts people’s lives. Findings from this survey will be published online and presented at OIFE’s Topical Meeting, “Balancing Life with OI” in Stockholm, Sweden from June 9-10, 2023.

Click here to take the survey


 

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?

*Phase 1: a small group of human participants are examined; looking for safety, proper dosage amounts and to confirm it has some response in humans. Phase 1 may be done in healthy volunteers or in patients with the disease, or at-risk of the disease, under 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.

INCLUSION CRITERIA:
  • 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.
LEARN MORE
If you have any questions about this study, you may email Contact-US@sanofi.com or call 800-633-1610, ext option 6 (Toll free for US & Canada).

Disclaimer: The OI Foundation is not involved in the design or management of this research, and as such, is neither endorsing nor supporting this study. The mission of the OIF is to keep the OI community informed of all relevant studies. This information is made available as a service to the OI community.


Pain and OI Project

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/


 

OI Research Announcement: Use of clear aligners…

The OI Foundation would like to bring your attention to a research study being conducted by the Brittle Bone Disorders Consortium:
Please call/email to see if you are eligible to participate or have any questions.

Bethesda, MD area:  National Institutes of Health
Danielle Elangue danielle.elangue@nih.gov 301-771-1378

Los Angeles, CA area: UCLA
Hannah Ebbers HEbbers@mednet.ucla.edu 310.794.6420

For more information about current studies through the Brittle Bones Disorders Consortium (BBDC), visit the BBDC webpage.


 

Michael Geisman Fellowship Grant – Applications Now Available

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.

OI Research Update: Longitudinal Study

BBDC Research Update: Longitudinal Study

On September 22, 2022, the OI Foundation was joined by Dr. Reid Sutton (Geneticist at Baylor College of Medicine, Administrative Director and Principal Investigator of the Brittle Bone Disorders Consortium, and member of the OIF Medical Advisory Council and Board of Directors) to discuss the Longitudinal Study of Osteogenesis Imperfecta. This study, also known as the Natural History Study, is the largest research project ever conducted on people with OI. Dr. Sutton discussed the design of the study, its goals, and what researchers have learned so far. Watch this session recording below.


Participate in the Longitudinal Study of OI!
The purpose of this natural history study is to perform a long-term follow-up of a large group of people with osteogenesis imperfecta (OI). Information collected will include medical history, number of broken bones, surgeries done, medications taken, ability to walk, pain, lung function and breathing, hearing, and bone mineral density. The overall goal is to improve the health and quality of life of people with OI. Click here to learn more and determine if you are eligible to participate.

COVID-19 Survey 3 Results

Thank you to all of the 577 OI community members who took the time to fill out the OI Foundation’s third COVID-19 survey! Our goal in gathering the community’s input is to provide feedback during the changing landscape of COVID-19 and direct the OIF’s resources where most needed. CLICK HERE to view the full survey results and important findings.

 

To complete future surveys about OI, we highly encourage you to sign up for the OI Registry at www.oif.org/OIregistry to be the first to hear about these opportunities.

OI Research Update: New Medical Treatments

On March 15, 2022, the OI Foundation spoke with Dr. Brendan Lee, Principal Investigator for the Brittle Bone Disease Consortium (BBDC). In this interview, they discussed new drugs being studied to treat osteogenesis imperfecta, and the drug development process. Watch the session recording below, or click here to view the recording on YouTube.

 

This session is the fifth in a series meant to demystify and contextualize medical research from the Brittle Bone Disorders Consortium (BBDC). The BBDC is a multicenter initiative that focuses on understanding and providing better treatment options for osteogenesis imperfecta. The previous BBDC research updates can be viewed here

As a partner of the OIF, the BBDC has 3 major goals:

  1. Gain a better understanding of all genetic forms of OI
  2. Expand treatment options for those with OI
  3. Help guide the next generation of physicians and scientists.

This series is intended for anyone who wants to learn more about OI medical research. We hope you will attend a future session!


New study results on drug treatment for OI published in Journal of Clinical Investigation

The study, “Targeting transforming growth factor- β (TGF-β) for treatment of osteogenesis imperfecta” was led by OIF MAC Member Dr. Brendan Lee of Baylor College of Medicine. Click here to learn more about this study.

Research Announcement: ORBIT Study

The OI Foundation would like to bring your attention to a research study being conducted by Ultragenyx Pharmaceutical Inc:

The Ultragenyx Orbit clinical study is for individuals living with osteogenesis imperfecta (OI). The purpose of this study is to investigate the efficacy and safety of setrusumab in pediatric and young adult patients with OI Types I, III, or IV.  Setrusumab is a monoclonal antibody being developed for the treatment of osteogenesis imperfecta (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, had at least one fracture in the past year or at least two fractures in the past 2 years, and are willing to not receive bisphosphonate therapy during the study. For more information, please click here, or reach out to trialrecruitment@ultragenyx.com.

 

If you are interested in learning more about this trial and to see if you can participate you can reach out to trialrecruitment@ultragenyx.org.