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Study on Setrusumab for Patients with Osteogenesis Imperfecta (Brittle Bone Disease)

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Osteogenesis Imperfecta, often referred to as brittle bone syndrome. This is a genetic disorder that causes bones to break easily, sometimes with little or no apparent cause. The study is testing a treatment called Setrusumab, which is a type of medication known as a monoclonal antibody. Monoclonal antibodies are proteins made in a lab that can bind to substances in the body, and in this case, Setrusumab is designed to target and neutralize a protein involved in bone breakdown.

The purpose of the study is to evaluate the safety and effectiveness of Setrusumab in reducing the rate of bone fractures in individuals with Osteogenesis Imperfecta. The study is divided into two phases. In the first phase, different doses of Setrusumab will be tested to find the most effective dose. In the second phase, participants will receive either Setrusumab or a placebo to compare the effects. The study will monitor participants over a period of time to see how the treatment affects their bone health and fracture rates.

Participants in the study will receive Setrusumab through an intravenous infusion, which means the medication is given directly into a vein. The study will involve regular visits to the clinic for these infusions and for monitoring the participants' health and response to the treatment. The goal is to determine if Setrusumab can help reduce the number of fractures and improve the quality of life for those living with Osteogenesis Imperfecta.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, eligibility is confirmed based on age, diagnosis of osteogenesis imperfecta (OI), fracture history, and vitamin D levels.
Participants must agree to avoid certain therapies and use effective contraception if applicable.
Step 2
Phase II: dose evaluation
This phase involves receiving setrusumab through an intravenous infusion. The dosage is evaluated to determine the most effective strategy.
The primary goal is to observe changes in specific blood markers related to bone health after one month.
Step 3
Phase III: double-blind treatment
Participants are randomly assigned to receive either setrusumab or a placebo. Neither the participant nor the study team knows which treatment is given.
The focus is on reducing the rate of fractures over the treatment period, excluding certain types of fractures.
Step 4
Monitoring and assessments
Throughout the study, regular assessments are conducted to monitor bone density, fracture rates, and any side effects.
Blood samples are taken at scheduled times to measure the concentration of setrusumab and check for any antibodies against the medication.
Step 5
Completion of the study
The study is expected to conclude by May 2026. Participants will have a final assessment to evaluate the overall impact of the treatment on their condition.

Who can join the trial?

9 criteria

Must be a male or female between 5 and 25 years old at the time of giving consent to participate.
Must have a diagnosis of **Osteogenesis Imperfecta (OI)** Type I, III, or IV. This is confirmed by finding specific changes in genes called **COL1A1** or **COL1A2**. If the genetic test is unclear, the doctor can confirm the diagnosis based on symptoms.
Must have had at least one broken bone in the past year, at least two broken bones in the past two years, or at least one break in the tibia, femur, or humerus in the past two years. The tibia, femur, and humerus are bones in the legs and arms.
Must have a blood level of **25-hydroxyvitamin D** of at least 20 ng/mL at the screening visit. If the level is lower, it can be checked again after taking vitamin D supplements for at least 14 days as advised by the doctor.
Must agree not to receive **bisphosphonate therapy** during the study. This is a type of treatment for bone conditions.
Females who can have children and males who are fertile must agree to use highly effective birth control from the time of consent until 60 days after the last dose of the study drug. Females must agree not to become pregnant, and males must agree not to father a child or donate sperm during this time.
Must be willing and able to give informed consent if 18 years or older, or provide assent (if possible) and have a legally authorized representative give consent if younger, after understanding the study details.
Must agree to allow access to medical records for collecting information about X-rays, broken bones, growth, and disease history.
Must be willing and able, in the opinion of the study doctor, to complete all parts of the study, attend all study visits, and follow the study procedures.

Who cannot join the trial?

5 criteria

Patients who have a different condition than Osteogenesis imperfecta (OI), which is a genetic disorder that affects bones.
Patients who are not within the specified age range for the study.
Patients who are not part of the specified clinical trial group.
Patients who are not male or female, as both genders are included in the study.
Patients who are considered part of a vulnerable population, which means they might need special protection or care.

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Verified sites

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Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
Responsive
Lodz, Poland
Universita Degli Studi Di Roma La Sapienza
Responsive
Rome, Italy
Universita' Degli Studi Di Verona
Responsive
Verona, Italy

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Investigated drugs

Setrusumab is a medication being studied for its potential to help people with Osteogenesis Imperfecta, a condition that makes bones fragile and more likely to break. The trial aims to find the best way to use this medication to reduce the number of fractures in patients, excluding certain types of fractures like those in the fingers, toes, face, and skull.

Setrusumab

What is already known about the treatment

Setrusumab – Setrusumab is administered through an intravenous infusion, allowing the medication to be delivered directly into the bloodstream. It is currently being studied in clinical trials for its effectiveness and safety in treating osteogenesis imperfecta, a condition characterized by fragile bones. The main therapeutic indication for setrusumab is to reduce the fracture rate in individuals with this condition. At the molecular level, setrusumab works by inhibiting a protein that affects bone formation and strength, thereby potentially increasing bone density and reducing fractures. It is classified as a monoclonal antibody, which is a type of medication designed to target specific proteins in the body.

Investigated diseases

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. Individuals with this condition may experience frequent fractures, bone deformities, and short stature. The severity of symptoms can vary widely, with some people having only a few fractures in a lifetime and others having hundreds. In addition to bone issues, people with Osteogenesis Imperfecta may have other symptoms such as hearing loss, dental problems, and blue sclerae. The condition is present from birth and affects individuals throughout their lives.