PARTICIPATING IN OI STUDIES

Join the OI Registry
Individuals with OI (18 and older) and parents of children with OI are encouraged to join the OI Registry. The OI Registry is a database of individuals with OI who are interested in participating in OI research. Once you are enrolled, you will begin to receive information on upcoming studies and online surveys about OI. Your participation in these studies helps move OI research forward. If you have any questions, please contact the OI Foundation at bonelink@oif.org or (844)889-7579.

Click here to join the OI Registry


CURRENT OI STUDIES

Supporting research is an important part of the OI Foundation’s mission. Often, the success of clinical studies of a rare disorder like osteogenesis imperfecta (OI) depends on getting enough people to participate in the study so the results are meaningful. People who are interested in participating in a clinical trial are encouraged to review the fact sheet What You Need to Know about Clinical Trials. Additional information about research studies can be found on www.clinicaltrials.gov.

The following studies are currently enrolling participants.
Click on the study title for detailed information about who is eligible to participate and other study information.

Disclaimer: The OI Foundation is not involved in the design or management of this research, and as such, is neither endorsing nor supporting these studies. 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.

The Linked Clinical Research Centers program (LCRC), established by the OI Foundation and the Children’s Brittle Bone Foundation started a major longitudinal or natural history study of OI. The Longitudinal Study of Osteogenesis Imperfecta has been extended and expanded through the Brittle Bone Disorders Consortium. The purpose of this study is to collect information about the experiences of people with all types of OI across the entire life-span. Five centers in the United States and one in Canada are enrolling children and adults into this study. People of all ages and all types of OI are needed. Participants in the original study are encouraged to re-enroll at the site they had been using or at one of the newly added sites. If you were in the first study and have not been contacted by the study coordinator, please call them or call the OI Foundation. Study participants will receive an annual physical and various diagnostic studies.

Click here for more information about this study.

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

The purpose of this study is to determine if it is safe to use Invisalign clear aligners in correcting the misalignment of teeth in people with OI. Clear aligners are transparent plastic trays that are designed to fit over your teeth. With each new tray, teeth are moved a little at a time until they reach the desired position. We plan to have approximately 57 people take part in this study.

For more information, please contact Dianne Nguyen (BBDC Project Manager) at (713)798-6694 or diannen@bcm.edu.

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

The purpose of this study is to better understand the mental health and pain experiences of people living with Osteogenesis Imperfecta (OI). Study investigators will conduct virtual interviews and administer self-report questionnaires to individuals living with OI (ages 8-89), as well as caregivers and clinicians who treat people with OI. For completion of the hour-long virtual interview and questionnaire, participants will be compensated $50.

The study aims to recruit 50 participants, though larger studies will follow

Inclusion criteria:

  • OI diagnosis (or clinician or caregiver of individual with OI)
  • Ages 8-89
  • USA Resident
  • Able to communicate in English

How to participate:

If you or your child are interested in participating in the study, please complete the interest survey.

Additional questions may be directed to OIWellbeing@bcm.edu or (713) 798-8563 for more information

The osteogenesis imperfecta program at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) enrolls children with types III and IV OI. Under the direction of Dr. Joan Marini, this program has over 25 years’ experience providing comprehensive multidisciplinary evaluations and care for children with OI. Pediatric participants are offered follow-up into young adulthood. Four protocols are available.

Screening Protocol: Children are first admitted under this protocol for diagnostic and screening evaluations. Testing may include: skin biopsy, blood draw, X-rays or bone density scan (DEXA).

Natural History Protocol: Provides comprehensive evaluation and monitoring. Includes genetics, rehabilitation, physical therapy, developmental, bone and spine X-rays, bone density scans (DEXA), dental, hearing, pulmonary, cardiac and neurologic examination.

Growth Protocol: This protocol evaluates the effectiveness of growth hormone on improving growth and bone quality in children with OI. All children complete a year of pretreatment measurements and testing before beginning one year of growth hormone injections. After the first year of treatment, children who respond to growth hormone may continue treatment for additional years.

Participation in NIH studies is free of charge. To learn more contact Dr. Joan Marini. Phone Number: 301-496-0741; Email: oiprogram@mail.nih.gov

 

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 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*. 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).