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Evaluating Romosozumab versus Bisphosphonates for Osteogenesis Imperfecta in Children and Adolescents

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

Phase 3 Interventional Amgen · NCT05972551

This study is testing whether a new treatment called romosozumab is better than bisphosphonates for helping children and teens with osteogenesis imperfecta have fewer fractures and stronger bones.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment106 (estimated)
Ages5 Years to 17 Years
SexAll
SponsorAmgen Industry-sponsored
Drugs / interventionsromosozumab
Locations65 sites (Birmingham, Alabama and 64 other locations)
Trial IDNCT05972551 on ClinicalTrials.gov

What this trial studies

This study aims to assess the efficacy and safety of romosozumab compared to bisphosphonates in treating children and adolescents with osteogenesis imperfecta over a 12-month period. Participants will be monitored for the number of clinical fractures and changes in lumbar spine bone mineral density. The study includes ambulatory children and adolescents aged 5 to under 18 years who have a clinical diagnosis of osteogenesis imperfecta. The primary focus is on evaluating fracture rates and bone density improvements.

Who should consider this trial

Good fit: Ideal candidates are ambulatory children and adolescents aged 5 to under 18 years diagnosed with type I, III, or IV osteogenesis imperfecta.

Not a fit: Patients with osteogenesis imperfecta types other than I, III, or IV, or those who are non-ambulatory may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce fracture rates and improve bone health in children and adolescents with osteogenesis imperfecta.

How similar studies have performed: Previous studies have shown promising results with romosozumab in other populations, but this specific approach in children with osteogenesis imperfecta is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.

OR

* Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
* Ambulatory male and female children and adolescents, age 5 to \<18 years, including ambulatory with assistance as defined in the pediatric osteogenesis imperfecta (OI) population.
* Clinical diagnosis of OI, defined as clinical history consistent with type I, III, or IV OI as determined by presence of expected phenotype (examples include: facial shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic features, fracture pattern) and lack of additional features unrelated to type I, III, or IV OI (eg, blindness, mental retardation, neuropathy, and craniosynostosis).

  o If familial, also must be autosomal dominant.
* Meets at least one of the following:

  * 3 or more fractures within the previous 2 years, or
  * 1 or more nonvertebral fracture(s) within the previous 2 years and at least 1 prevalent vertebral fracture, or
  * 2 or more prevalent vertebral fractures.

Exclusion Criteria:

Disease Related

* History of an electrophoresis pattern inconsistent with type I, III or IV OI.
* History of known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1A1/COL1A2) causing OI or other metabolic bone disease.
* History of congenital dislocation of the radial head, interosseous membrane calcification, or exuberant callus formation.

Where this trial is running

Birmingham, Alabama and 64 other locations

+15 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Osteogenesis ImperfectaRomosozumabEVENITY®Bisphosphonates
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.