NCT05972551进行中（未招募）3 期
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
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- Amgen
- 入组人数
- 111
- 试验地点
- 98
- 主要终点
- Change from Baseline in Lumbar Spine BMD Z-score at 12 Months, as assessed by DXA
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.
- •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.
排除标准
- •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.
研究组 & 干预措施
Romosozumab
Experimental
Participants will receive romosozumab once a month (QM) for 12 months.
干预措施: Romosozumab (Drug)
Standard of Care Bisphosphonate
Active Comparator
Participants will receive bisphosphonates per local standard of care treatment regimens, as determined by the investigator for 12 months.
干预措施: Bisphosphonate (Drug)
结局指标
主要结局
Change from Baseline in Lumbar Spine BMD Z-score at 12 Months, as assessed by DXA
时间窗: Baseline and 12 months
Number of Clinical Fractures
时间窗: 12 months
Clinical fractures include clinical vertebral fractures and nonvertebral fractures.
Number of Any Fractures
时间窗: 12 months
Fractures include new and worsening vertebral compression fractures, whether clinically silent or manifest, and nonvertebral fractures.
次要结局
- Change from Baseline in lumbar spine BMD Z-score at 6 months and 12 months, as assessed by DXA(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(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(Baseline, 6 months, and 12 months)
- Number of Participants with Any Fractures(12 months)
- Number of Participants with Clinical Fractures(12 months)
- Number of Participants with New or Worsening Vertebral Fractures(12 months)
- Number of Participants with Nonvertebral Fractures(12 months)
- Number of Participants with Long Bone Fractures(12 months)
- Number of New or Worsening Vertebral Fractures(12 months)
- Number of Nonvertebral Fractures(12 months)
- Number of Long Bone Fractures(12 months)
- Change from Baseline in Child Health Questionnaire - Parent Version (CHQ-PF-50) Physical Summary Score(Baseline and 12 months)
- Change from Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Score(Baseline and 12 months)
- Change from Baseline in the Wong-Baker Faces Pain Rating Scale(Baseline and 12 months)
- Serum Concentration of Romosozumab(Day 1 to Month 12)
- Number of Participants who Experience Treatment-emergent Adverse Events (TEAEs) at 12 Months(12 months)
- Number of Participants who Experience TEAEs from Month 12 to Month 15(Month 12 to Month 15)
- Number of Participants with Anti-drug Antibodies (ADA) to Romosozumab(Up to 15 months)
- Number of Participants who Experience TEAEs at 15 Months(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(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(Baseline and 12 months)
研究者
研究点 (98)
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