Study Comparing Setrusumab and Bisphosphonates for Children with Osteogenesis Imperfecta

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What is this study about?

This study is being conducted to evaluate the effects of a new medication compared to existing treatments for children with Osteogenesis Imperfecta. Osteogenesis Imperfecta is a rare genetic condition that affects the development of bones, often making them fragile and more prone to breaking. The research compares the test drug setrusumab against a group of medications known as bisphosphonates, which are a class of drugs used to strengthen bone. The bisphosphonates used in this study include pamidronate disodium, zoledronic acid, and neridronate sodium.

The purpose of the study is to determine if setrusumab can more effectively reduce the frequency of bone fractures, including specific types of fractures in the spine, compared to the standard bisphosphonates. Participants will receive their assigned medication through an infusion, which is a method of delivering fluids or medicine directly into a vein. The study will monitor various factors, such as bone strength measured by dual-energy X-ray absorptiometry, which is a specialized type of X-ray used to check bone density, and the frequency of new bone breaks over time.

1 joining the study

Upon joining the study, you will be assigned to receive either setrusumab or a type of medication known as bisphosphonates. These medications are used to help manage a condition called osteogenesis imperfecta, which affects bone strength.

2 medication administration

If you are assigned to receive setrusumab, it will be administered either through a vein (intravenous) or under the skin (subcutaneous).

If you are assigned to receive bisphosphonates, such as zoledronic acid or pamidronate disodium, these will be given through a vein (infusion).

The specific dosage and frequency of the medication will be determined by the study team based on your individual needs.

3 monitoring and assessments

Throughout the study, you will have regular check-ups to monitor your health and the effects of the medication. This includes checking for any fractures and assessing your bone health using imaging techniques.

You will also be asked to provide blood samples at scheduled times to measure the concentration of setrusumab in your body, if applicable.

4 reporting side effects

It is important to report any side effects or unusual symptoms you experience during the study. This helps the study team ensure your safety and adjust your treatment if necessary.

5 completion of the study

The study is expected to continue until June 2026. Upon completion, you will have a final assessment to evaluate the overall effects of the treatment on your condition.

Who Can Join the Study?

Must be a boy or girl between the ages of 2 and less than 7 years old at the time of giving consent to participate.
Must have a clinical diagnosis of osteogenesis imperfecta (OI) Types I, III, or IV, confirmed by finding a genetic mutation in the COL1A1 or COL1A2 genes.
Must have a history of at least 1 fracture in the past 12 months, at least 2 fractures in the past 24 months, or at least 1 fracture in the femur, tibia, or humerus in the past 24 months.
Must have had any prior exposure to, or currently be receiving, intravenous bisphosphonate therapy for the treatment of OI. Intravenous bisphosphonate therapy is a treatment given through a vein to help strengthen bones.
Must have a serum 25-hydroxyvitamin D level of at least 20 ng/mL at the screening visit. If the vitamin D level is below 20 ng/mL, the participant may be re-evaluated after taking vitamin D supplements for at least 14 days as directed by the study doctor.

Who Cannot Join the Study?

Patients who have a different condition than osteogenesis imperfecta cannot participate. This is a genetic disorder that affects bones.
Patients who are not within the specified age range for the study cannot participate.
Patients who are not willing or able to follow the study procedures cannot participate.
Patients who have other medical conditions that might interfere with the study cannot participate.
Patients who are currently participating in another clinical trial cannot participate.
Patients who have had recent major surgery or are planning to have surgery during the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have allergies to the study medication or similar drugs cannot participate.
Patients who have a history of drug or alcohol abuse cannot participate.
Patients who have certain abnormal laboratory test results cannot participate.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Assistance Publique Hopitaux De Paris	Paris	France
Uwufabcgyh Hdkamqot Cxhabek	Cologne	Germany
Uobuirodtt Dvbdz Synac Df Rakn Lh Sctfcdic	Rome	Italy

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.08.2023
Germany	Not recruiting	01.08.2023
Italy	Not recruiting	01.08.2023
Poland	Not recruiting	01.08.2023
The Netherlands	Not recruiting	01.08.2023

Trial locations

Investigated drugs:

Setrusumab is a medication being studied for its potential to help people with a condition called osteogenesis imperfecta, which makes bones very fragile and easy to break. This medication is designed to strengthen bones and reduce the number of fractures, including those in the spine. It works by targeting a specific pathway in the body that affects bone strength, aiming to make bones more resilient.

Bisphosphonates are a group of medications that are commonly used to treat bone conditions by slowing down the process that breaks down bone tissue. In this study, they are used as a comparison to see how well they work against setrusumab in reducing fractures in children with osteogenesis imperfecta. These medications are usually given through an intravenous (IV) infusion, which means they are delivered directly into the bloodstream to help strengthen bones and prevent fractures.

Investigated diseases:

Osteogenesis imperfecta

Osteogenesis Imperfecta – Osteogenesis imperfecta is a genetic disorder characterized by fragile bones that break easily, often with little or no apparent cause. It is caused by a defect in the production of collagen, a protein that is crucial for bone strength. The condition can vary in severity, with some individuals experiencing frequent fractures and others having only a few throughout their lifetime. In addition to bone fragility, individuals may have other symptoms such as blue sclerae, hearing loss, and dental issues. The progression of the disease can lead to bone deformities and reduced mobility over time. The severity and specific symptoms can differ widely among affected individuals.

Trial ID:

2023-504196-24-00

Protocol code:

UX143-CL314

NCT ID:

NCT05768854

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study Comparing Setrusumab and Bisphosphonates for Children with Osteogenesis Imperfecta

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?