Interested in participating? Learn about a clinical study opportunity.
Orbit Clinical Phase 3 Study
You are between the ages of 5 and 26 years
Have a confirmed diagnosis of OI Types I, III, or IV
Had at least one fracture in the past year, two fractures in the past 2 years, or a tibia, humerus, or femur fracture in the past 2 years
Fractures can be self-treated or assessed by your doctor
Agree to not take non-approved therapies for OI during this study, including bisphosphonates and other investigational medications
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.
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.
Screening Period:
Up to 1 month
Study Treatment Period:
1 to 2 years with maximum study enrollment time of 24 months
Treatment Extension Period:
Receive setrusumab until commercial medicine is available
You will receive study medicine or placebo 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.
Support is provided for travel costs to clinic visits.
Monthly infusion of study medicine or placebo into your arm
Monthly physical examinations
Regular X-rays and bone density tests
Heart tests
Blood and urine tests
A smartphone app to track your health and fractures (a smartphone will be provided if you don’t have one)
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.
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.
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.
Cosmic Clinical Phase 3
Consider having your child participate if he or she:
Is at least 2 but less than 7 years of age
Has a clinical diagnosis of OI Types I, III, or IV
Has had at least one fracture in the past year or two fractures in the past 2 years or a femur, tibia, or humerus fracture in the past 2 years
Has received intravenous bisphosphonate therapy for treatment of OI
The purpose of this study is to learn more about how a new medicine called setrusumab can reduce fractures compared to bisphosphonates in children with OI, as well as to study the safety of setrusumab. Setrusumab is called an investigational drug because it is still being researched and can only be obtained through a clinical study. The goal of the study is to learn more about the best use of setrusumab so that the data can be submitted to Health Authorities, like the FDA.
Your bones are a living tissue and the cells are constantly turning over— making new bone (bone formation) and breaking preexisting bone (bone resorption). Certain treatments for OI slow bone resorption. Less bone resorption can increase the density of bones, but the bone remains abnormal and some patients still have fractures. Setrusumab is expected to work in a different way. Setrusumab works by building new bone, as well as slowing bone resorption. In this study, we will see if children who receive setrusumab have fewer fractures over 2 years.
Ultragenyx recognizes that your participation in clinical research has an impact on your 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.
Screening Period:
Up to 1 month
Screening includes assessments to confirm you meet the entry requirements for the study, and will include lab tests, imaging and physical examination.
Study Treatment Period:
2 years
Treatment Extension Period:
Receive setrusumab a minimum of 12-months or until commercial medicine is available
Infusions of setrusumab or bisphosphonates into your child’s arm
Monthly physical examinations
X-rays (every 12 months) and bone density tests (every 6 months)
Heart tests (every 6 months)
Blood and urine tests
A smartphone app to track your child’s health and fractures (a smartphone will be provided if you don’t have one)
Your child will be assigned by chance, like flipping a coin, to receive study treatment with either:
You will know which of these your child receives. If your child receives setrusumab, infusions will be once a month. If your child receives bisphosphonates, your child’s doctor will choose the treatment plan based on local guidelines; the timing of infusions will depend on the plan chosen.
To learn about current and future studies, you only need to fill out the form once.
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By submitting this form, you confirm that a diagnosis of OI has been made by a medical professional.
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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 tri[email protected], if it’s not in your regular inbox.
Email our PEL team about current and future medical research opportunities.
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 Ultragenyx clinical and non-interventional clinical trials and related sites where you could be considered for enrollment. 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.
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