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Study on the Effects of Romosozumab and Bisphosphonates in Children and Adolescents with Osteogenesis Imperfecta

Verified siteRegistered drugNo placebo
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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, which is a genetic disorder characterized by fragile bones that break easily. The study aims to evaluate the effectiveness and safety of a medication called Romosozumab, which is administered as a solution for injection. Romosozumab is being compared to another type of medication known as Bisphosphonates, which are commonly used to treat bone-related conditions. The purpose of the study is to assess how well Romosozumab works in reducing the number of bone fractures in children and adolescents with Osteogenesis Imperfecta over a 12-month period.

Participants in the study will receive either Romosozumab or Bisphosphonates. Romosozumab is given through a subcutaneous injection, which means it is injected under the skin, while Bisphosphonates are typically given as a solution for infusion, meaning they are administered directly into the bloodstream. The study will also involve the use of Calcium and Ergocalciferol (a form of vitamin D) in tablet form to support bone health. The trial will last for a total of 12 months, during which the number of fractures and changes in bone density will be monitored.

Throughout the study, participants will have regular check-ups to monitor their bone health and overall well-being. The study will also measure changes in bone density using a method called DXA (Dual-energy X-ray Absorptiometry), which is a type of imaging test that helps assess bone strength. The goal is to determine if Romosozumab is more effective than Bisphosphonates in reducing fractures and improving bone density in young patients 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.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing the diagnosis of osteogenesis imperfecta and ensuring the presence of specific clinical features.

    Eligibility criteria include being between 5 and 18 years old, having a lumbar spine Z-score of ≤ -1.0, and a history of fractures as specified.

  2. Step 2

    Treatment initiation

    The treatment phase begins with the administration of romosozumab via subcutaneous injection. The dosage is 105 mg, delivered through a pre-filled syringe or pen.

    This medication is administered once a month for a duration of 12 months.

  3. Step 3

    Supplementation

    In addition to the main treatment, ergocalciferol and calcium supplements are taken orally to support bone health.

    The specific dosage and frequency of these supplements are determined based on individual needs and medical guidance.

  4. Step 4

    Monitoring and assessments

    Throughout the study, regular monitoring is conducted to assess the treatment's effectiveness and safety.

    Key assessments include measuring changes in bone density at 6 and 12 months, and tracking the occurrence of any fractures.

  5. Step 5

    Final evaluation

    At the end of the 12-month treatment period, a comprehensive evaluation is performed.

    This includes a final assessment of bone density, fracture incidence, and overall health status.

Who can join the trial?

9 criteria

  • The study is for both males and females.
  • Participants should be between 5 and 18 years old.
  • Participants must have a diagnosis of Osteogenesis Imperfecta (OI), which is a condition that affects bones, making them fragile and more likely to break. The specific types included are type I, III, or IV.
  • Participants should show typical signs of OI, such as certain facial features, voice characteristics, blue sclera (the white part of the eyes appearing blue), dentinogenesis imperfecta (a condition affecting teeth), and typical bone features seen in X-rays.
  • Participants should not have other unrelated conditions like blindness, mental retardation, nerve problems, or craniosynostosis (a condition where the skull bones fuse too early).
  • Participants need to have a lumbar spine Z-score of -1.0 or lower. This score is a measure used to compare bone density to what is expected for someone of the same age and gender.
  • Participants must have experienced at least one of the following:
    • Three or more bone fractures in the last two years.
    • One or more fractures in bones other than the spine in the last two years, along with at least one fracture in the spine.
    • Two or more fractures in the spine.

Who cannot join the trial?

10 criteria

  • Patients who have a different condition than Osteogenesis Imperfecta, which is a genetic disorder affecting bones.
  • Patients who are not within the specified age range for the study.
  • Patients who are not willing or able to follow the study procedures.
  • Patients who have other medical conditions that might interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had recent major surgery or are planning to have surgery during the study period.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of allergic reactions to the study medications.
  • Patients who have used certain medications that might affect the study results.
  • Patients who have a history of substance abuse or other factors that might affect their ability to participate in the study.
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Investigated drugs

  • Romosozumab

    is a medication used in this trial to help strengthen bones. It works by increasing bone formation and decreasing bone breakdown, which can help reduce the risk of fractures. This medication is being tested to see how effective it is in children and adolescents with a condition called Osteogenesis Imperfecta, which makes bones fragile and more likely to break.

  • Bisphosphonates

    are a group of medications that help prevent the loss of bone density. They work by slowing down the process that breaks down bone, which can help maintain or increase bone strength. In this trial, bisphosphonates are being compared to romosozumab to see which is more effective in reducing fractures in young patients with Osteogenesis Imperfecta.

What is already known about the treatment

Romosozumab – This medication is administered via injection and is currently being studied in clinical trials for its effectiveness in treating Osteogenesis Imperfecta, a condition characterized by fragile bones. Romosozumab is a monoclonal antibody that works by inhibiting a protein called sclerostin, which results in increased bone formation and decreased bone resorption, thereby strengthening bones. It is classified as a bone density conservation agent. The medication is being compared to bisphosphonates, which are another class of drugs used to treat bone disorders, to assess its safety and efficacy in reducing fractures in children and adolescents with this condition.

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 hearing loss, dental problems, and a blue tint to the whites of their eyes. The condition is present from birth and affects individuals throughout their lives.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2023-503294-37-00Protocol code20200105Estimated enrolment122 patientsSponsorAmgen Inc.

sourced from the EU Clinical Trials Register and site verification

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