Interested in participating? Learn about a clinical study opportunity.

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STUDY| Clinical Interventional

Orbit Clinical Phase 3 Study

Key ELIGIBILITY

5-26 years of age icon

You are between the ages of 5 and 26 years

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 a 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 study is to evaluate how a new investigational medicine called setrusumab can safely and effectively reduce fractures in pediatric and young adult patients with OI. Setrusumab is called an investigational medicine because it is not approved by Health Authorities and is still being tested in research studies. 

Certain treatments for OI slow bone resorption. Less bone resorption can increase the density of bones, but the bone remains abnormal and some OI patients still have fractures. Setrusumab is expected to work in a different way by building new bone, as well as slowing bone resorption. In this study, we will see if patients who receive setrusumab have fewer fractures over the study period. 

For more information, please visit clinicaltrials.gov

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 with maximum study enrollment time of 24 months

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Treatment Extension Period:

Receive setrusumab until commercial medicine is available

STUDY VISITS

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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.

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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 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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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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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.

Universal no symbol

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
ACTIVE, NOT ENROLLING ACTIVE, NOT ENROLLING
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STUDY| Clinical Interventional

Cosmic Clinical Phase 3

Key ELIGIBILITY

Consider having your child participate if he or she:


2-5-age_icon

Is at least 2 but less than 7 years of age

OI_Bones_Icon

Has a clinical diagnosis of OI Types I, III, or IV

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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

intravenous _icon

Has received intravenous bisphosphonate therapy for treatment of OI

Why are we doing this study?

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.

For more information, please visit clinicaltrials.gov

Additional travel support may be available

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.

What is involved?

Study duration

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

STUDY VISITS

Your child will participate in the study for up to 24 months, with an option to extend in the open label extension.
Your child’s visits will include lab tests and assessments. If your child receives setrusumab, infusions will be given at each visit.
If your child receives bisphosphonates, the study doctor will tell you which visits will include infusions.
Where allowed, support is provided for travel costs to clinic visits.

What you can expect?

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Infusions of setrusumab or bisphosphonates into your child’s arm

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Monthly physical examinations

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X-rays (every 12 months) and bone density tests (every 6 months)

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Heart tests (every 6 months)

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Blood and urine tests

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

Study treatment

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Your child will be assigned by chance, like flipping a coin, to receive study treatment with either:

  • setrusumab infusions (50% chance)
  • OR
  • bisphosphonate infusions (50% chance)


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.

Interested in participating or have questions? Get started

Get Started

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Why are we asking this?

As with most clinical trials, certain criteria such as age must be met to determine who can participate.

The date of birth entered should be that of the person who would be enrolled in the trial.
If you are under the age of 18, a parent, care partner, or guardian must fill out this form for you.
Why are we asking this?

Letting us know the Patient's zip code can help us locate clinical trials that are closest to you or your loved one. Many clinical trials are held in hospitals and clinics and may require you to visit a facility in person.

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Please Enter Only US Number

Please Enter Only US Phone Number

To ensure that a human is submitting this information, please answer the question below. Feel free to use a calculator! This question will not impact your ability to get trial information. Once you hit SUBMIT, you will receive a Thank You page that confirms your submission.

By filling out the above form and clicking the SUBMIT button, you consent to providing this information to Ultragenyx Pharmaceuticals Inc. (Ultragenyx) and its employees, affiliates, and service providers. You understand that your information will be used for prescreening for the research program(s) and internal business purposes.

Additionally, with this consent, Ultragenyx will retain such information to provide you with program updates, newsletters, and other communications.

Ultragenyx will retain your personal data in accordance with its regulatory record-keeping requirements or for as long as we have a legitimate legal or business need to do so. You understand that you can withdraw your consent at any time by contacting [email protected]. For more on our privacy practices and your privacy rights, including the right to withdraw your consent, please refer to our Privacy Policy at https://www.ultragenyx.com/privacy-policy/

 

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 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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