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Recruitment Complete
Sponsor ID
20200105

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

Sponsor ID
20200105
Clinicaltrials.gov ID
NCT05972551
EUCTIS ID
N/A
EudraCT ID
2023-503294-37
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Study Details

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.

Additional Study Details

Phase
Phase 3
Product
romosozumab
Type
Interventional
Masking
None (Open Label)
Enrollment
111
Additional Study Details

Protocol Summary

Primary Outcome Measures

Number of Clinical Fractures

Timeframe: 12 months

Number of Any Fractures

Timeframe: 12 months

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

Timeframe: Baseline and 12 months

Secondary Outcome Measures

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

Timeframe: Baseline, 6 months, and 12 months

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

Timeframe: Baseline, 6 months, and 12 months

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

Timeframe: Baseline, 6 months, and 12 months

Number of Participants with Any Fractures

Timeframe: 12 months

Number of Participants with Clinical Fractures

Timeframe: 12 months

Number of Participants with New or Worsening Vertebral Fractures

Timeframe: 12 months

Number of Participants with Nonvertebral Fractures

Timeframe: 12 months

Number of Participants with Long Bone Fractures

Timeframe: 12 months

Number of New or Worsening Vertebral Fractures

Timeframe: 12 months

Number of Nonvertebral Fractures

Timeframe: 12 months

Number of Long Bone Fractures

Timeframe: 12 months

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

Timeframe: Baseline and 12 months

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

Timeframe: Baseline and 12 months

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

Timeframe: Baseline and 12 months

Serum Concentration of Romosozumab

Timeframe: Day 1 to Month 12

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

Timeframe: 12 months

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

Timeframe: Month 12 to Month 15

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

Timeframe: Up to 15 months

Number of Participants who Experience TEAEs at 15 Months

Timeframe: 15 months

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

Timeframe: Baseline and 6 months

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

Timeframe: Baseline and 12 months

Locations

Location
Status
Contact Us
Location
Monash Childrens Hospital
Clayton, VIC, Australia, 3168
Status
Recruiting
Location
Kepler Universitaetsklinikum GmbH
Linz, Austria, 4020
Status
Recruiting
Location
Centre Hospitalier Universitaire Sainte Justine
Montreal, QC, Canada, H3T 1C5
Status
Recruiting
Location
Hopital Necker Enfants Malades
Paris Cedex 15, France, 75743
Status
Recruiting
Location
Semmelweis Egyetem
Budapest, Hungary, 1094
Status
Recruiting
Location
Okayama University Hospital
Okayama-shi, Okayama, Japan, 700-8558
Status
Recruiting
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Study Info
Age
5 - 17 Years
Condition
  • Osteogenesis Imperfecta
    • Sex
    All
    Trial Dates
    Apr 2024 - May 2027
    Inclusion and 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: o 3 or more fractures within the previous 2 years, or o 1 or more nonvertebral fracture(s) within the previous 2 years and at least 1 prevalent vertebral fracture, or o 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.