Interested in participating? Learn about a clinical study opportunity.

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STUDY| Clinical Interventional

Orbit Clinical Phase 2/3 Study


5-26 years of age icon

You are between the ages of 5 and 26

Osteogenesis Imperfecta genetic icon

Have a confirmed diagnosis of OI Types I, III, or IV

Osteogenesis Imperfecta bone icon

Had at least one fracture in the past year, two fractures in the past 2 years, or one tibia, humerus, or femur fracture in the past 2 years

Fractures can be self-treated or assessed by your doctor

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Agree to not take non-approved therapies for OI during this study, including bisphosphonates and other investigational medications

Why are we doing this study?

The purpose of this clinical trial is to investigate the efficacy and safety of a new investigational medicine called setrusumab in pediatric and young adult patients with OI Types I, III, or IV.

“Investigational” means setrusumab is not approved by Health Authorities and is still being tested in research studies. Current treatments for OI focus on preventing bone from breaking down (bone resorption). However, bone remains abnormal, and some patients still experience fractures.

Setrusumab is an investigational therapy that works differently than bisphosphonates. We are performing this study to see if treatment with setrusumab will reduce the total number of fractures a patient with OI experiences over 1-2 years.

Osteogenesis Imperfecta clinical research logo
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Additional travel support may be available

Ultragenyx recognizes that your participation in clinical research has an impact on your (and your loved ones’) time. We may provide compensation and travel support for participation in our clinical trials—the details may vary program to program. You will learn more during the consent process.

What is involved?

Study duration
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Screening Period:

Up to 1 month

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Study Treatment Period:

1 to 2 years (depending on when you enroll)

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Treatment Extension Period:

Receive setrusumab until commercial medicine is available


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You will receive study medicine (or placebo if in Phase 3) and have lab tests and assessments at monthly study visits.

The number of visits you will have will depend on when you enroll in the study.

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Support is provided for travel costs to clinic visits.

What you can expect?

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Monthly infusion of study medicine (or placebo if in Phase 3) into your arm

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Monthly physical examinations

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Regular X-rays and bone density tests

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Heart tests

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Blood and urine tests

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A smartphone app to track your health and fractures (a smartphone will be provided if you don’t have one)

Study treatment

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There will be two groups for each phase in this study. For Phase 2, you will receive one of two study doses.

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For Phase 3, you will be assigned to receive either the study medicine or placebo (a pretend medicine). Two people will receive study medicine for every one person who receives placebo. After the study treatment period, everyone (100%) will receive the study medicine.

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  • setrusumab dose X
  • setrusumab dose Y
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  • setrusumab dose X/Y (dose selected from Phase 2)
  • placebo
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Why is it important to be willing to not receive bisphosphonate therapy and other study medications during the study?

In order to evaluate the effects of setrusumab, we need to assess patients who are not on other medications that also impact the bone during the time of the study. This will allow us to best assess the efficacy and safety of setrusumab.

Interested in participating or have questions? Get started

Get Started

To learn about current and future studies, you only need to fill out the form once.
*All fields are required.


By submitting this form, you confirm that a diagnosis of OI has been made by a medical professional.

If you would like to know more about your privacy and the data you submit and data we collect while you are on this website, please see below or visit our privacy policy.

What happens after you submit your information?

Our Patient Enrollment Liaison (PEL) team will contact you by email or phone within one business day to discuss your study eligibility and answer questions.

If you choose to be contacted by email, be sure to check your spam folder for an email from [email protected], if it’s not in your regular inbox.

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Got Questions?

Email our PEL team about current and future medical research opportunities.

Osteogenesis Imperfecta patient representation Osteogenesis Imperfecta patient representation

Patient portrayal.

Ol is a group of genetic disorders that impact bone metabolism. Approximately 85% to 90% of OI cases are caused by mutations in the COL1A1 or COL1A2 genes, leading to bone resorption and inadequate production of new bone. Patients with OI can experience brittle bones, fractures, bone deformities, pain, short stature, and decreased mobility.

If you are thinking about joining a study, our Patient Enrollment Liaison (PEL) team is here to help. Every member of our PEL team has a robust professional background and clinical expertise and experience to help answer your questions. Your PEL team member will assist in exploring clinical and nonclinical studies and can determine if you or someone you care for may be eligible to take part. Your PEL is here to make the process as easy as possible.

To speak with a PEL about current and future medical research opportunities, please email our PEL team at [email protected].

When you voluntarily submit your personal data, Ultragenyx stores and processes this information in an electronic database consistent with our Privacy Policy. Ultragenyx will use this information to contact you to fulfill your request regarding Ultragenyx research opportunities.

Clinical and nonclinical studies provide invaluable information to treat, diagnose, or discover new breakthroughs in treating rare and ultra-rare diseases. By choosing to participate in research, you may help advance treatments, provide crucial insights, and ultimately improve the quality of life for others living with a rare or ultra-rare disease. You might also benefit from learning more about your condition, gain access to specific care, or achieve faster access to treatment. All efforts of participation, big and small, can make a difference.

Ultragenyx is leading the future of rare disease medicine. We leverage our experience, insight, and commitment to help move the rare disease community forward. Our primary goal is to provide medicines to those with limited options, and to help patients face rare disease head on with courage and confidence.