A Phase 3 Study of Romosozumab Vs Bisphosphonates in OI

  • Research type

    Research Study

  • Full title

    A Phase 3, Open-label, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Romosozumab Compared with Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta



  • Contact name

    Matthew Rodaway

  • Contact email


  • Sponsor organisation

    Amgen Inc.

  • Clinicaltrials.gov Identifier


  • Research summary

    The purpose of this study is to compare the study drug to a standard treatment used for osteogenesis imperfecta in children (5 to less than 12 years of age) and adolescents (12 to less than 18 years of age). Osteogenesis imperfecta (OI) is a genetic condition that prevents the body from building strong bones in children, characterized by bones that break easily. There are no approved medicinal products for the treatment of OI in children in the UK. Participants agree to be put into a treatment group by chance (“randomised”), like flipping a coin. The participants, study doctor, and the study staff will know which study drug each participant is given. The study may last up to about 15 months for each participant, including a screening period of up to 35 days, a 12-months treatment period, and a 3-month safety follow-up period.
    Children (5 to less than 12 years of age) and adolescents (12 to less than 18 years of age) with OI can participate in this study. Participants need to have history consistent with type I, III, or IV OI (types of OI). Participants must have also had 3 or more broken bones within the previous 2 years or 2 or more broken backbone or at least 1 or more in broken backbone and 1 or more in nonvertebral broken bones (not in the spine/skull) within last 2 years.
    Participants randomised to romosozumab group will be given romosozumab as an injection just under the skin monthly for 12 months. Participants randomised to comparator group will receive bisphosphonates per local standard of care.
    Participants in this study will undergo procedures including blood tests, x-rays, DEXA scans and questionnaires. If the participants agree to participate in the sub-study, CT scans will also be performed. This sub-study is optional.

  • REC name

    East of Scotland Research Ethics Service REC 2

  • REC reference


  • Date of REC Opinion

    2 Feb 2024

  • REC opinion

    Further Information Favourable Opinion