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NCT Number: NCT05972551

Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta

The primary objective of this study is to evaluate the effect of romosozumab treatment for 12-months compared with bisphosphonate(s) on the number of clinical fractures at 12-months; the number of any fractures at 12-months and change in lumbar spine bone mineral density (BMD) Z-score at 6-months.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Osteogenesis Imperfecta Bone Diseases Bone Diseases, Developmental Collagen Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias Skin and Connective Tissue Diseases

Age range

5 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Monash Childrens Hospital, Clayton, Victoria, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Romosozumab

Drug

Subcutaneous (SC) injection

Other names: EVENITY®

Bisphosphonate

Drug

Administration determined by investigator according to the local standard of care

Primary outcomes

  1. Number of Clinical Fractures

    Time frame: 12 months

    Clinical fractures include clinical vertebral fractures and nonvertebral fractures.

  2. Number of Any Fractures

    Time frame: 12 months

    Fractures include new and worsening vertebral compression fractures, whether clinically silent or manifest, and nonvertebral fractures.

  3. Change from Baseline in Lumbar Spine BMD Z-score at 12 Months, as assessed by DXA

    Time frame: Baseline and 12 months

Secondary outcomes

  1. Change from Baseline in lumbar spine BMD Z-score at 6 months and 12 months, as assessed by DXA

    Time frame: Baseline, 6 months, and 12 months

  2. Change from Baseline in Total Hip BMD Z-score at 6 Months and at 12 Months, as assessed by DXA

    Time frame: Baseline, 6 months, and 12 months

  3. Change from Baseline in Femoral Neck BMD Z-score at 6 Months and at 12 Months, as assessed by DXA

    Time frame: Baseline, 6 months, and 12 months

  4. Number of Participants with Any Fractures

    Time frame: 12 months

  5. Number of Participants with Clinical Fractures

    Time frame: 12 months

  6. Number of Participants with New or Worsening Vertebral Fractures

    Time frame: 12 months

  7. Number of Participants with Nonvertebral Fractures

    Time frame: 12 months

  8. Number of Participants with Long Bone Fractures

    Time frame: 12 months

  9. Number of New or Worsening Vertebral Fractures

    Time frame: 12 months

  10. Number of Nonvertebral Fractures

    Time frame: 12 months

  11. Number of Long Bone Fractures

    Time frame: 12 months

  12. Change from Baseline in Child Health Questionnaire - Parent Version (CHQ-PF-50) Physical Summary Score

    Time frame: Baseline and 12 months

    The CHQ-PF-50 measures how a child's condition affects their ability to function in daily life. The CHQ-PF-50 measures 50 items in the following domains: physical functioning, role/social limitations - physical, general health perceptions, bodily pain/discomfort, family activities, role/social limitations - emotional/behavioral, parent impact - time, parent impact - emotion, self-esteem, mental health, behavior, family cohesion, change in health.

    Each item is rated on a scale from "without any difficulty" to "unable to do". Total scores for each item are transformed to 0 - 100 scale, with lower scores indicating worse health states. Higher change from baseline scores indicate better or more positive health states.

  13. Change from Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Score

    Time frame: Baseline and 12 months

    The CHAQ measures how a child's condition affects their ability to function in daily life. The CHAQ measures 50 items in the following domains: physical functioning, role/social limitations - physical, general health perceptions, bodily pain/discomfort, family activities, role/social limitations emotional/behavioral, self-esteem, mental health, behavior, family cohesion, change in health.

    Each item is rated on a scale from "without any difficulty" to "unable to do". Total scores for each item are transformed to 0 - 100 scale, with lower scores indicating worse health states. Higher change from baseline scores indicate better or more positive health states.

  14. Change from Baseline in the Wong-Baker Faces Pain Rating Scale

    Time frame: Baseline and 12 months

    The Wong-Baker Faces Pain Rating Scale is a horizontal pain scale that consists of six hand-drawn faces that range from a smiling "no hurt" face with a score of 0 to a crying "hurts worst" face with a score of 10. Greater change from baseline scores indicate greater pain experienced by the participant.

  15. Serum Concentration of Romosozumab

    Time frame: Day 1 to Month 12

  16. Number of Participants who Experience Treatment-emergent Adverse Events (TEAEs) at 12 Months

    Time frame: 12 months

    Any clinically signification change from baseline in laboratory values and vital signs after first dose will be recorded as a TEAE.

  17. Number of Participants who Experience TEAEs from Month 12 to Month 15

    Time frame: Month 12 to Month 15

    Any clinically signification change from baseline in laboratory values and vital signs after first dose will be recorded as a TEAE.

  18. Number of Participants with Anti-drug Antibodies (ADA) to Romosozumab

    Time frame: Up to 15 months

  19. Number of Participants who Experience TEAEs at 15 Months

    Time frame: 15 months

    Any clinically signification change from baseline in laboratory values and vital signs after first dose will be recorded as a TEAE.

  20. Number of Participants with a Narrowing from Baseline to 6 Months in the Intracranial Nerve Tract in the Cranium and Vault of the Skull

    Time frame: Baseline and 6 months

    Measured in a subset of participants who receive cranial nerve computerized tomography (CT) scans.

  21. Number of Participants with a Narrowing from Baseline to 12 Months in the Intracranial Nerve Tract in the Cranium and Vault of the Skull

    Time frame: Baseline and 12 months

    Measured in a subset of participants who receive cranial nerve CT scans.

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Aug 2, 2023
Registry last updated
May 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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